Research graph
References from Ethical and regulatory aspects of gene therapy in neurodegenerative disorders. Local targets link to admitted publications; unresolved targets remain external evidence.
The promise of digital health: then, now, and the future
10.31478/202206e · 2022 · External reference
Bench, bedside, boardroom: negotiating translational gene therapy
10.1080/14636778.2017.1289468 · 2017 · External reference
Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: umbrella review
10.1016/j.healthpol.2022.10.007 · 2022 · External reference
Barriers for conducting clinical trials in developing countries- a systematic review
10.1186/s12939-018-0748-6 · 2018 · External reference
Regulations in Japan
2024 · External reference
Real-world evidence to support regulatory submissions: a landscape review and assessment of use cases
10.1111/cts.13903 · 2024 · External reference
10.3389/fmed.2024.1408636
10.3389/fmed.2024.1408636 · External reference
Post marketing surveillance of suspected adverse drug reactions through spontaneous reporting: current status, challenges and the future
10.1177/2042098620938595 · 2020 · External reference
Duchenne muscular dystrophy: disease mechanism and therapeutic strategies
2023 · External reference
Emerging technologies and their impact on regulatory science
10.1177/15353702211052280 · 2022 · External reference
International harmonization for cell and gene therapy products
10.1007/978-3-031-34567-8_14 · 2023 · External reference
The horizon of gene therapy in modern medicine: advances and challenges
10.1007/5584_2019_463 · 2020 · External reference
Bioethical issues in genome editing by CRISPR-Cas9 technology
10.3906/biy-1912-52 · 2020 · External reference
United States food and drug administration regulation of gene and cell therapies
10.1007/978-3-319-18618-4_1 · 2015 · External reference
A state-of-the-art review of registries in spinal muscular atrophy: a valuable resource for clinical research
2025 · External reference
Explainable artificial intelligence (XAI): concepts, taxonomies, opportunities and challenges toward responsible AI
10.1016/j.inffus.2019.12.012 · 2020 · External reference
Navigating challenges and opportunities in orphan medicines: a spotlight commentary on rare diseases
10.1002/bcp.70013 · 2025 · External reference
Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer
10.1002/emmm.201200245 · 2012 · External reference
Optimizing regulatory frameworks for gene therapies in rare diseases: challenges and solutions
10.1016/j.omtm.2024.101386 · 2024 · External reference
Role of the GH/IGF-1 axis in lifespan and healthspan: lessons from animal models
10.1016/j.ghir.2008.05.005 · 2008 · External reference
Strengthening health systems for access to gene therapy in rare genetic disorders
10.1016/j.omtm.2024.101220 · 2024 · External reference
Onasemnogene abeparvovec: a review in spinal muscular atrophy
10.1007/s40263-022-00941-1 · 2022 · External reference
Promoting inclusion in clinical trials—a rapid review of the literature and recommendations for action
10.1186/s13063-021-05849-7 · 2021 · External reference
Clinical potential of microdystrophin as a surrogate endpoint
10.1016/j.nmd.2022.12.007 · 2023 · External reference
Consequences of increased longevity for wealth, fertility, and population growth
10.1016/j.physa.2007.09.004 · 2008 · External reference
Recent advances in genome editing and bioinformatics: addressing challenges in genome editing implementation and genome sequencing
10.3390/ijms26073442 · 2025 · External reference
Advancing the future of equitable access to health care: recommendations from international health care leaders
10.1093/haschl/qxae094 · 2024 · External reference
Ethics by design for artificial intelligence
10.1007/s43681-023-00330-4 · 2024 · External reference
Bioethics of somatic gene therapy: what do we know so far?
10.1080/03007995.2023.2257600 · 2023 · External reference
Viral vector platforms within the gene therapy landscape
10.1038/s41392-021-00487-6 · 2021 · External reference
Challenges and recommendations for wearable devices in digital health: data quality, interoperability, health equity, fairness
10.1371/journal.pdig.0000104 · 2022 · External reference
A catholic perspective on human gene therapy
1993 · External reference
Innovation in chemistry, manufacturing, and controls—A regulatory perspective from industry
10.1016/j.xphs.2019.02.007 · 2019 · External reference
A roadmap for fostering timely regulatory and ethics approvals of international clinical trials in support of global health research systems
10.1016/s2214-109x(24)00515-1 · 2025 · External reference
AMA code of medical ethics’ opinions related to unrepresented patients
10.1001/amajethics.2019.600 · 2019 · External reference
Rethinking the knowledge-attitudes model and introducing belief in human evolution: examining antecedents of public acceptability of human gene editing
10.1080/13698575.2022.2123903 · 2022 · External reference
Stem cell therapeutics and gene therapy for neurologic disorders
10.1016/j.neurot.2024.e00427 · 2024 · External reference
Gene therapy for neurodegenerative disorders: advances, insights and prospects
10.1016/j.apsb.2020.01.015 · 2020 · External reference
Planning for progress: a US regulatory approach to advancing the clinical development of gene therapies
10.1016/j.ymthe.2022.05.005 · 2022 · External reference
Policy advocacy and nursing organizations: a scoping review
10.1177/15271544211050611 · 2021 · External reference
Ethics of human genome editing
10.1146/annurev-med-112717-094629 · 2019 · External reference
Gene therapy access: global challenges, opportunities, and views from Brazil, South Africa, and India
10.1016/j.ymthe.2022.04.002 · 2022 · External reference
Equitable access to gene therapy: a call to action for the American society of gene and cell therapy
10.1016/j.ymthe.2018.11.002 · 2018 · External reference
Therapeutic genome editing: prospects and challenges
10.1038/nm.3793 · 2015 · External reference
Assuring long-term safety of highly effective gene-modulating therapeutics for rare diseases
10.1172/jci152817 · 2021 · External reference
Bias in medical AI: implications for clinical decision-making
10.1371/journal.pdig.0000651 · 2024 · External reference
10.1007/978-3-030-05542-4_2
10.1007/978-3-030-05542-4_2 · External reference
The epigenetic impacts of social stress: how does social adversity become biologically embedded?
10.2217/epi-2016-0075 · 2016 · External reference
Amortization of gene replacement therapies: a health policy analysis exploring a mechanism for mitigating budget impact of high-cost treatments
10.1016/j.healthpol.2021.11.005 · 2022 · External reference
Neuroimaging applications for the delivery and monitoring of gene therapy for central nervous system diseases
10.1089/hum.2024.057 · 2024 · External reference
Real-world evidence: a primer
10.1007/s40290-022-00456-6 · 2023 · External reference
Online forums as a tool for broader inclusion of voices on health care communication experiences and serious illness care: mixed methods study
2023 · External reference
Prolonged lifespan with enhanced exploratory behavior in mice overexpressing the oxidized nucleoside triphosphatase hMTH1
10.1111/acel.12094 · 2013 · External reference
Public acceptability of gene therapy and gene editing for human use: a systematic review
10.1089/hum.2019.197 · 2020 · External reference
EU regulatory pathways for ATMPs: standard, accelerated and adaptive pathways to marketing authorisation
10.1016/j.omtm.2019.01.010 · 2019 · External reference
Advancing genome editing with artificial intelligence: opportunities, challenges, and future directions
2023 · External reference
Unresolved reference
2000 · External reference
Pharmacovigilance is everyone’s concern: let’s work it out together
10.1016/j.clinthera.2018.09.016 · 2018 · External reference
The potential of gene delivery for the treatment of traumatic brain injury
10.1186/s12974-024-03156-x · 2024 · External reference
Post-trial access to treatment for patients participating in clinical trials
10.4103/2229-3485.154003 · 2015 · External reference
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs
10.1016/j.omtm.2021.04.001 · 2021 · External reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
Gene therapy comes of age
10.1126/science.aan4672 · 2018 · External reference
Involving stakeholders as communication partners in research dissemination efforts
10.1007/s11606-021-07127-3 · 2022 · External reference
Supporting patient autonomy: the importance of clinician-patient relationships
10.1007/s11606-010-1292-2 · 2010 · External reference
Integration of artificial intelligence and genome editing system for determining the treatment of genetic disorders
10.4274/balkanmedj.galenos.2024.2024-080824 · 2024 · External reference
Challenges in accelerated approvals for gene therapies
10.1016/j.ymthe.2023.08.004 · 2023 · External reference
Processes of consent in research for adults with impaired mental capacity nearing the end of life: systematic review and transparent expert consultation (MORECare_Capacity statement)
10.1186/s12916-020-01654-2 · 2020 · External reference
Using patient perspectives to inform better clinical trial design and conduct: current trends and future directions
10.1007/s40290-022-00458-4 · 2023 · External reference
Twenty-five years of gene therapy for ADA-SCID: from bubble babies to an approved drug
10.1089/hum.2017.175 · 2017 · External reference
RESTORE: a prospective multinational registry of patients with genetically confirmed spinal muscular atrophy—rationale and study design
10.3233/jnd-190451 · 2020 · External reference
Unraveling the genetic landscape of neurological disorders: insights into pathogenesis, techniques for variant identification, and therapeutic approaches
10.3390/ijms25042320 · 2024 · External reference
The role of patient advocacy organizations in advancing human gene therapy
10.1089/hum.2015.29011.trf · 2015 · External reference
Expanded access to investigational drugs: balancing patient safety with potential therapeutic benefits
10.1080/13543784.2018.1430137 · 2018 · External reference
Regulatory pathways supporting expedited drug development and approval in ICH member countries
10.1007/s43441-022-00480-3 · 2023 · External reference
Advances and challenges in gene therapy for neurodegenerative diseases: a systematic review
10.3390/ijms252312485 · 2024 · External reference
Value-based pricing for emerging gene therapies: the economic case for a higher cost-effectiveness threshold
10.18553/jmcp.2019.18378 · 2019 · External reference
Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies
10.18553/jmcp.2021.27.5.674 · 2021 · External reference
Pharmaceutical manufacturing in the EU and the EU-US mutual recognition agreement
10.5731/pdajpst.2020.011437 · 2020 · External reference
The interplay of ethics and genetic technologies in balancing the social valuation of the human genome in UNESCO declarations
10.1038/s41431-024-01549-3 · 2024 · External reference
Gene therapy with voretigene neparvovec improves vision and partially restores electrophysiological function in pre-school children with leber congenital amaurosis
10.3390/biomedicines11010103 · 2023 · External reference
Gene therapy: advances, challenges and perspectives
10.1590/s1679-45082017rb4024 · 2017 · External reference
Surrogate consent for research involving adults with impaired decision making: survey of Institutional review board practices
10.1097/ccm.0b013e3181f26fe6 · 2010 · External reference
Gene therapy bio-safety: scientific and regulatory issues
10.1038/sj.gt.3302629 · 2005 · External reference
Gene therapy leaves a vicious cycle
10.3389/fonc.2019.00297 · 2019 · External reference
Understanding the challenges and ethical aspects of compassionate use of drugs in emergency situations
10.4103/ijp.ijp_665_20 · 2020 · External reference
Off-target effects in CRISPR/Cas9 gene editing
10.3389/fbioe.2023.1143157 · 2023 · External reference
Long-term follow-up after authorization of gene therapy: leveraging real-world data
10.1016/j.drudis.2025.104337 · 2025 · External reference
Artificial intelligence in health care: accountability and safety
10.2471/blt.19.237487 · 2020 · External reference
Cultural aspects related to informed consent in health research: a systematic review
10.1177/0969733015579312 · 2016 · External reference
Precision medicine—the golden gate for detection, treatment and prevention of Alzheimer’s disease
10.14283/jpad.2016.112 · 2016 · External reference
Considering global development? Insights from applications for FDA breakthrough therapy and EMA PRIME designations
10.1007/s43441-022-00475-0 · 2023 · External reference
Gene therapies development: slow progress and promising prospect
10.1080/20016689.2017.1265293 · 2017 · External reference
Assessing psychosocial readiness for gene therapy in sickle cell disease: a consensus statement
10.1182/blood-2023-190398 · 2023 · External reference
Considerable life extension and three views on the meaning of life
10.1017/s0963180110000599 · 2011 · External reference
When research becomes practice: the concept of the therapeutic misconception and challenges to consent in clinical trials
10.1111/imj.16015 · 2023 · External reference
Research with Alzheimer’s disease subjects: informed consent and proxy decision making
10.1111/j.1532-5415.1992.tb01995.x · 1992 · External reference
Enabling technologies for personalized and precision medicine
10.1016/j.tibtech.2019.12.021 · 2020 · External reference
Toward a global harmonization of service infrastructure in academic clinical trial units: an international survey
10.3389/fmed.2023.1252352 · 2023 · External reference
Understanding FDA regulatory requirements for investigational new drug applications for sponsor-investigators
10.2310/jim.0b013e3181afdb26 · 2009 · External reference
Community engaged leadership to advance health equity and build healthier communities
10.3390/socsci5010002 · 2016 · External reference
Controversies in treatment approaches: gene therapy, IVF, stem cells, and pharmacogenomics
2008 · External reference
Neurodegenerative diseases: regenerative mechanisms and novel therapeutic approaches
10.3390/brainsci8090177 · 2018 · External reference
Regulatory framework for advanced therapy medicinal products in Europe and United States
10.3389/fphar.2019.00921 · 2019 · External reference
Does the act on the safety of regenerative medicine in Japan ensure “safety”?: implications of low adverse event reporting
10.1016/j.stemcr.2023.10.012 · 2023 · External reference
Real-world evidence in new drug and biologics license application approvals during fiscal years 2020–2022
10.1002/cpt.3688 · 2025 · External reference
Overview of FDA’s expanded access program for investigational drugs
10.1177/2168479017694850 · 2017 · External reference
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union
10.1038/s41434-023-00437-7 · 2024 · External reference
Ethical perspectives of therapeutic human genome editing from multiple and diverse viewpoints: a scoping review
2022 · External reference
The importance of international collaboration for rare diseases research: a European perspective
10.1038/gt.2017.29 · 2017 · External reference
Informed consent process: a step further towards making it meaningful!
10.4103/picr.picr_147_16 · 2017 · External reference
Unresolved reference
2024 · External reference
Cognitive impairment and PD patients’ capacity to consent to research
10.1212/wnl.0b013e3182a05ba5 · 2013 · External reference
Facing the new diagnostic and treatment options of Alzheimer’s disease: the necessity of informed consent
10.1002/alz.14204 · 2025 · External reference
Development and use of gene therapy orphan drugs—Ethical needs for a broader cooperation between the pharmaceutical industry and society
10.3389/fmed.2020.608249 · 2020 · External reference
Growth signaling and longevity in mouse models
10.5483/bmbrep.2019.52.1.299 · 2019 · External reference
The ethics of informed consent in Alzheimer disease research
10.1038/nrneurol.2011.76 · 2011 · External reference
Ethics of gene and cell therapy development for neurologic disorders
10.1016/b978-0-323-90120-8.00002-2 · 2024 · External reference
Programming gene and engineered-cell therapies with synthetic biology
10.1126/science.aad1067 · 2018 · External reference
Eternal youth: a comprehensive exploration of gene, cellular, and pharmacological anti-aging strategies
10.3390/ijms25010643 · 2024 · External reference
The periodic safety update report as a pharmacovigilance tool
10.2165/00002018-200427080-00008 · 2004 · External reference
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States
10.1080/20016689.2023.2216518 · 2023 · External reference
Ethical issues in care and treatment of neuronal ceroid lipofuscinoses (NCL)-A personal view
10.3389/fneur.2021.692527 · 2021 · External reference
Understanding cultural values, norms and beliefs that may impact participation in genome‐editing related research: perspectives of local communities in Botswana
10.1111/dewb.12440 · 2025 · External reference
Unveiling the guidance heterogeneity for genome-informed drug treatment interventions among regulatory bodies and research consortia
10.1016/j.phrs.2019.104590 · 2020 · External reference
Postmarket surveillance of medical devices: a comparison of strategies in the US, EU, Japan, and China
10.1371/journal.pmed.1001519 · 2013 · External reference
Judicialization of zolgensma in the ministry of health: costs and clinical profile of patients
2024 · External reference
High-quality health systems in the sustainable development goals era: time for a revolution
10.1016/s2214-109x(18)30386-3 · 2018 · External reference
Clinical development of gene therapy: results and lessons from recent successes
10.1038/mtm.2016.34 · 2016 · External reference
A literature-based study of patient-centered care and communication in nurse-patient interactions: barriers, facilitators, and the way forward
10.1186/s12912-021-00684-2 · 2021 · External reference
A review of the common neurodegenerative disorders: current therapeutic approaches and the potential role of nanotherapeutics
10.3390/ijms23031851 · 2022 · External reference
Building trust: leadership reflections on community empowerment and engagement in a large urban initiative
10.1186/s12889-023-15860-z · 2023 · External reference
Clinical development of gene therapies: the first three decades and counting
10.1016/j.omtm.2020.10.004 · 2020 · External reference
Navigating equity in global access to genome therapy expanding access to potentially transformative therapies and benefiting those in need requires global policy changes
10.3389/fgene.2024.1381172 · 2024 · External reference
Evidence-informed capacity building for setting health priorities in low- and middle-income countries: a framework and recommendations for further research
10.12688/f1000research.10966.1 · 2017 · External reference
Biomarkers for Parkinson’s disease: how good are they?
10.1007/s12264-019-00433-1 · 2020 · External reference
Advanced therapy medicinal products in China: regulation and development
10.1002/mco2.251 · 2023 · External reference
Delivering the promise of gene therapy with nanomedicines in treating central nervous system diseases
10.1002/advs.202201740 · 2022 · External reference
Health technology for all: an equity-based paradigm shift opportunity
10.31478/202212a · 2022 · External reference
An ethical assessment of anti-aging medicine
10.1089/109454503322733045 · 2003 · External reference
Gene therapy for the nervous system: challenges and new strategies
10.1007/s13311-014-0299-5 · 2014 · External reference
Global collaboration in acute care clinical research: opportunities, challenges, and needs
10.1097/ccm.0000000000002211 · 2017 · External reference
Gene therapy for neurodegenerative diseases: slowing down the ticking clock
10.3389/fnins.2020.580179 · 2020 · External reference
Personalized medicine could transform healthcare (Review)
10.3892/br.2017.922 · 2017 · External reference
Understanding the influence and impact of stakeholder engagement in patient-centered outcomes research: a qualitative study
10.1007/s11606-021-07104-w · 2022 · External reference
Decision-making capacity evaluation in adult guardianship: a systematic review
10.1017/s1041610215001490 · 2016 · External reference
Assessing genetic risks—implications for health and social policy: response from the newborn screening committee of the council of regional networks for genetic services
10.1016/0925-6164(96)00133-8 · 1996 · External reference
Current clinical applications of in vivo gene therapy with AAVs
10.1016/j.ymthe.2020.12.007 · 2021 · External reference
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
10.1038/s41591-024-03304-z · 2025 · External reference
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: a phase 1/2a nonrandomized trial
10.1002/mus.27955 · 2024 · External reference
Ethical and regulatory challenges of AI technologies in healthcare: a narrative review
10.1016/j.heliyon.2024.e26297 · 2024 · External reference
Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy
10.1007/s10198-023-01639-x · 2024 · External reference
Regulatory systems and requirements for clinical trials of AAV-based gene therapies—perspectives from six Asian countries or regions: report from the 6th Asia Partnership Conference of Regenerative Medicine—April 20, 2023
10.1016/j.reth.2024.06.005 · 2024 · External reference
Revolutionizing personalized medicine: synergy with multi-omics data generation, main hurdles, and future perspectives
10.3390/biomedicines12122750 · 2024 · External reference
Next-generation strategies for gene-targeted therapies of central nervous system disorders: a workshop summary
10.1016/j.ymthe.2021.09.010 · 2021 · External reference
Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders
10.1186/s13023-019-1017-5 · 2019 · External reference
FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections
10.1038/d41573-023-00103-y · 2023 · External reference
Current challenges and potential solutions to the use of digital health technologies in evidence generation: a narrative review
10.3389/fdgth.2023.1203945 · 2023 · External reference
Addressing health literacy in patient decision aids: an update from the international patient decision aid standards
10.1177/0272989x211011101 · 2021 · External reference
Flexible and expedited regulatory review processes for innovative medicines and regenerative medical products in the US, the EU, and Japan
10.3390/ijms20153801 · 2019 · External reference
Regulatory challenges associated with conducting multicountry clinical trials in resource-limited settings
10.1097/qai.0000000000000037 · 2014 · External reference
Cost-of-illness studies of inherited retinal diseases: a systematic review
10.1186/s13023-024-03099-9 · 2024 · External reference
The iterative development of medicines through the european medicine agency’s adaptive pathway approach
10.3389/fmed.2019.00148 · 2019 · External reference
Ethical issues related to research on genome editing in human embryos
10.1016/j.csbj.2020.03.014 · 2020 · External reference
Human genetic technologies and islamic bioethics
2010 · External reference
The global role of patients, advocates and caregivers in rare diseases
10.2217/frd-2022-0003 · 2022 · External reference
Pricing Zolgensma—the world’s most expensive drug
10.1080/20016689.2021.2022353 · 2022 · External reference
Sharing and reuse of individual participant data from clinical trials: principles and recommendations
10.1136/bmjopen-2017-018647 · 2017 · External reference
Scientific advancements in gene therapies: opportunities for global regulatory convergence
10.3390/biomedicines13030758 · 2025 · External reference
Patient rights and ethics
2024 · External reference
Future-proofing genomic data and consent management: a comprehensive review of technology innovations
2014 · External reference
A systematic review of the psychological implications of genetic testing: a comparative analysis among cardiovascular, neurodegenerative and cancer diseases
10.3389/fgene.2018.00624 · 2018 · External reference
Issues and challenges for clinical research in international settings
2012 · External reference
Biobanks and personalized medicine
10.1111/cge.12370 · 2014 · External reference
Challenges of clinical trials in low- and middle-income countries
10.1192/s1749367600003039 · 2012 · External reference
The cultural lens of genomics
10.3912/ojin.vol13no01man05 · 2008 · External reference
Artificial intelligence (AI) in personalized medicine: AI-generated personalized therapy regimens based on genetic and medical history: short communication
10.1097/ms9.0000000000001320 · 2023 · External reference
Emerging roles and opportunities for rare disease patient advocacy groups
10.1177/26330040231164425 · 2023 · External reference
Ethical issues in genetic therapy
10.1111/j.1552-6909.1994.tb01911.x · 1994 · External reference
Unresolved reference
2008 · External reference
Managing the challenges of paying for gene therapy: strategies for market action and policy reform in the United States
10.57264/cer-2024-0118 · 2024 · External reference
Discussing treatment burden
2021 · External reference
Justice: a key consideration in health policy and systems research ethics
10.1136/bmjgh-2019-001942 · 2020 · External reference
Advances in manufacturing chimeric antigen receptor immune cell therapies
10.1007/s00281-024-01019-4 · 2024 · External reference
Genetic modification and genetic determinism
10.1186/1747-5341-1-9 · 2006 · External reference
Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant
10.1002/jcsm.13486 · 2024 · External reference
Therapeutic modulation of gene expression in the disease state: treatment strategies and approaches for the development of next-generation of the epigenetic drugs
10.3389/fbioe.2022.1035543 · 2022 · External reference
Prevailing public perceptions of the ethics of gene therapy
10.1089/hum.2014.030 · 2014 · External reference
Using digital technologies in clinical trials: current and future applications
10.1016/j.cct.2020.106219 · 2021 · External reference
Patient advocacy organizations, industry funding, and conflicts of interest
10.1001/jamainternmed.2016.8443 · 2017 · External reference
Genetic screening and genetic therapy
1998 · External reference
Risks and benefits of human germline genome editing: an ethical analysis
10.1007/s41649-018-0056-x · 2018 · External reference
Real-world safety data of the orphan drug onasemnogene abeparvovec (Zolgensma®) for the SMA rare disease: a pharmacovigilance study based on the EMA adverse event reporting system
10.3390/ph17030394 · 2024 · External reference
Addressing the value of gene therapy and enhancing patient access to transformative treatments
10.1016/j.ymthe.2018.10.017 · 2018 · External reference
Advance care planning and advance directives: an overview of the main critical issues
10.1007/s40520-021-02001-y · 2022 · External reference
The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations
10.1017/cts.2023.604 · 2023 · External reference
Incentives for orphan drug research and development in the United States
10.1186/1750-1172-3-33 · 2008 · External reference
Artificial intelligence (AI) applications in drug discovery and drug delivery: revolutionizing personalized medicine
10.3390/pharmaceutics16101328 · 2024 · External reference
Rules for processing genetic data for research purposes in view of the new EU general data protection regulation
10.1038/s41431-017-0045-7 · 2018 · External reference
The complexity of regulatory capture: diagnosis, causality, and remediation
2012 · External reference
Moving forward after two deaths in a gene therapy trial of myotubular myopathy” by Wilson and Flotte
10.1089/hum.2020.217 · 2020 · External reference
Immune responses to viral gene therapy vectors
10.1016/j.ymthe.2020.01.001 · 2020 · External reference
National Academies of Sciences, Engineering, and Medicine. Causes and consequences of medical product supply chain failures
2022 · External reference
Precision genome editing techniques in gene therapy: current state and future prospects
10.2174/0115665232279528240115075352 · 2024 · External reference
Ethical aspects of facilitating the recruitment of people with dementia for clinical trials: a call for further debate
10.1111/bcp.14968 · 2022 · External reference
The role of community advisory boards: involving communities in the informed consent process
10.2105/ajph.91.12.1938 · 2001 · External reference
Gene-based therapies for neurodegenerative diseases
10.1038/s41593-020-00778-1 · 2021 · External reference
The state of gene therapies: the FDA perspective
10.1038/mt.2012.51 · 2012 · External reference
Achievements and challenges of the Sakigake designation system in Japan
10.1111/bcp.14807 · 2021 · External reference
Cell and gene therapy products: navigating the regulatory landscape of paradigm approvals in the US (2020 to 2024)
10.1007/s12247-025-09973-8 · 2025 · External reference
Evolving drug regulatory landscape in China: a clinical pharmacology perspective
10.1111/cts.12987 · 2021 · External reference
Substituted judgment: the limitations of autonomy in surrogate decision making
10.1007/s11606-008-0688-8 · 2008 · External reference
Prebunking against misinformation in the modern digital age
2023 · External reference
CRISPR gene therapy: applications, limitations, and implications for the future
10.3389/fonc.2020.01387 · 2020 · External reference
Key concepts of clinical trials: a narrative review
10.3810/pgm.2011.09.2475 · 2011 · External reference
Drug delivery systems, CNS protection, and the blood brain barrier
10.1155/2014/869269 · 2014 · External reference
AI, opacity, and personal autonomy
10.1007/s13347-022-00577-5 · 2022 · External reference
Manufacturing and quality of advanced therapeutics: cellular therapies, CAR-T and gene therapies
2024 · External reference
Principles of clinical ethics and their application to practice
10.1159/000509119 · 2021 · External reference
Informed consent in clinical trials
10.1016/0168-8510(95)00805-5 · 1996 · External reference
Elucidating the role of artificial intelligence in drug development from the perspective of drug-target interactions
10.1016/j.jpha.2024.101144 · 2025 · External reference
Innate immune response to viral vectors in gene therapy
10.3390/v15091801 · 2023 · External reference
Solving neurodegeneration: common mechanisms and strategies for new treatments
10.1186/s13024-022-00524-0 · 2022 · External reference
Ethical issues in the treatment of late-stage Alzheimer’s disease
10.3233/jad-180865 · 2019 · External reference
Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the DA-CH-region: a population-based observational study
2024 · External reference
The estimated annual financial impact of gene therapy in the United States
10.1038/s41434-023-00419-9 · 2023 · External reference
Development and clinical translation of ex vivo gene therapy
10.1016/j.csbj.2022.06.015 · 2022 · External reference
Gene therapy breakthroughs in ALS: a beacon of hope for 20% of ALS patients
10.1186/s40035-025-00477-6 · 2025 · External reference
From bench to bedside: cutting-edge applications of base editing and prime editing in precision medicine
10.1186/s12967-024-05957-3 · 2024 · External reference
Ethical perspectives on treatment options with spinal muscular atrophy patients
10.1002/ana.26299 · 2022 · External reference
Regulatory reform outcomes and accelerated regulatory pathways for new prescription medicines in Australia
10.1007/s43441-022-00465-2 · 2023 · External reference
Standards and Evaluation of Healthcare Quality, Safety, and Person-Centered Care
2025 · External reference
Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥4 to <8 years) with Duchenne muscular dystrophy: 1-year interim results from study SRP-9001-103 (ENDEAVOR)
10.1002/ana.26755 · 2023 · External reference
Development of the first World Health Organization lentiviral vector standard: toward the production control and standardization of lentivirus-based gene therapy products
10.1089/hgtb.2017.078 · 2017 · External reference
AMA code of medical ethics’ opinions related to unrepresented patients
10.1001/amajethics.2019.600 · ExternalCitation · doi-reference
Patient advocacy organizations, industry funding, and conflicts of interest
10.1001/jamainternmed.2016.8443 · ExternalCitation · doi-reference
Delivering the promise of gene therapy with nanomedicines in treating central nervous system diseases
10.1002/advs.202201740 · ExternalCitation · doi-reference
Facing the new diagnostic and treatment options of Alzheimer’s disease: the necessity of informed consent
10.1002/alz.14204 · ExternalCitation · doi-reference
Ethical perspectives on treatment options with spinal muscular atrophy patients
10.1002/ana.26299 · ExternalCitation · doi-reference
Delandistrogene moxeparvovec gene therapy in ambulatory patients (aged ≥4 to <8 years) with Duchenne muscular dystrophy: 1-year interim results from study SRP-9001-103 (ENDEAVOR)
10.1002/ana.26755 · ExternalCitation · doi-reference
Navigating challenges and opportunities in orphan medicines: a spotlight commentary on rare diseases
10.1002/bcp.70013 · ExternalCitation · doi-reference
Real-world evidence in new drug and biologics license application approvals during fiscal years 2020–2022
10.1002/cpt.3688 · ExternalCitation · doi-reference
Telomerase gene therapy in adult and old mice delays aging and increases longevity without increasing cancer
10.1002/emmm.201200245 · ExternalCitation · doi-reference
Improved therapeutic approach for spinal muscular atrophy via ubiquitination‐resistant survival motor neuron variant
10.1002/jcsm.13486 · ExternalCitation · doi-reference
Advanced therapy medicinal products in China: regulation and development
10.1002/mco2.251 · ExternalCitation · doi-reference
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: a phase 1/2a nonrandomized trial
10.1002/mus.27955 · ExternalCitation · doi-reference
The horizon of gene therapy in modern medicine: advances and challenges
10.1007/5584_2019_463 · ExternalCitation · doi-reference
10.1007/978-3-030-05542-4_2
10.1007/978-3-030-05542-4_2 · ExternalCitation · doi-reference
International harmonization for cell and gene therapy products
10.1007/978-3-031-34567-8_14 · ExternalCitation · doi-reference
United States food and drug administration regulation of gene and cell therapies
10.1007/978-3-319-18618-4_1 · ExternalCitation · doi-reference
Advances in manufacturing chimeric antigen receptor immune cell therapies
10.1007/s00281-024-01019-4 · ExternalCitation · doi-reference
Special FDA designations for drug development: orphan, fast track, accelerated approval, priority review, and breakthrough therapy
10.1007/s10198-023-01639-x · ExternalCitation · doi-reference
Substituted judgment: the limitations of autonomy in surrogate decision making
10.1007/s11606-008-0688-8 · ExternalCitation · doi-reference
Supporting patient autonomy: the importance of clinician-patient relationships
10.1007/s11606-010-1292-2 · ExternalCitation · doi-reference
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10.1007/s11606-021-07104-w · ExternalCitation · doi-reference
Involving stakeholders as communication partners in research dissemination efforts
10.1007/s11606-021-07127-3 · ExternalCitation · doi-reference
Cell and gene therapy products: navigating the regulatory landscape of paradigm approvals in the US (2020 to 2024)
10.1007/s12247-025-09973-8 · ExternalCitation · doi-reference
Biomarkers for Parkinson’s disease: how good are they?
10.1007/s12264-019-00433-1 · ExternalCitation · doi-reference
Gene therapy for the nervous system: challenges and new strategies
10.1007/s13311-014-0299-5 · ExternalCitation · doi-reference
AI, opacity, and personal autonomy
10.1007/s13347-022-00577-5 · ExternalCitation · doi-reference
Onasemnogene abeparvovec: a review in spinal muscular atrophy
10.1007/s40263-022-00941-1 · ExternalCitation · doi-reference
Real-world evidence: a primer
10.1007/s40290-022-00456-6 · ExternalCitation · doi-reference
Using patient perspectives to inform better clinical trial design and conduct: current trends and future directions
10.1007/s40290-022-00458-4 · ExternalCitation · doi-reference
Advance care planning and advance directives: an overview of the main critical issues
10.1007/s40520-021-02001-y · ExternalCitation · doi-reference
Risks and benefits of human germline genome editing: an ethical analysis
10.1007/s41649-018-0056-x · ExternalCitation · doi-reference
Regulatory reform outcomes and accelerated regulatory pathways for new prescription medicines in Australia
10.1007/s43441-022-00465-2 · ExternalCitation · doi-reference
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10.1007/s43441-022-00475-0 · ExternalCitation · doi-reference
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10.1007/s43441-022-00480-3 · ExternalCitation · doi-reference
Ethics by design for artificial intelligence
10.1007/s43681-023-00330-4 · ExternalCitation · doi-reference
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10.1016/0168-8510(95)00805-5 · ExternalCitation · doi-reference
Assessing genetic risks—implications for health and social policy: response from the newborn screening committee of the council of regional networks for genetic services
10.1016/0925-6164(96)00133-8 · ExternalCitation · doi-reference
Ethics of gene and cell therapy development for neurologic disorders
10.1016/b978-0-323-90120-8.00002-2 · ExternalCitation · doi-reference
Gene therapy for neurodegenerative disorders: advances, insights and prospects
10.1016/j.apsb.2020.01.015 · ExternalCitation · doi-reference
Using digital technologies in clinical trials: current and future applications
10.1016/j.cct.2020.106219 · ExternalCitation · doi-reference
Pharmacovigilance is everyone’s concern: let’s work it out together
10.1016/j.clinthera.2018.09.016 · ExternalCitation · doi-reference
Ethical issues related to research on genome editing in human embryos
10.1016/j.csbj.2020.03.014 · ExternalCitation · doi-reference
Development and clinical translation of ex vivo gene therapy
10.1016/j.csbj.2022.06.015 · ExternalCitation · doi-reference
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10.1016/j.drudis.2025.104337 · ExternalCitation · doi-reference
Role of the GH/IGF-1 axis in lifespan and healthspan: lessons from animal models
10.1016/j.ghir.2008.05.005 · ExternalCitation · doi-reference
Amortization of gene replacement therapies: a health policy analysis exploring a mechanism for mitigating budget impact of high-cost treatments
10.1016/j.healthpol.2021.11.005 · ExternalCitation · doi-reference
Barriers for the evaluation of advanced therapy medicines and their translation to clinical practice: umbrella review
10.1016/j.healthpol.2022.10.007 · ExternalCitation · doi-reference
Ethical and regulatory challenges of AI technologies in healthcare: a narrative review
10.1016/j.heliyon.2024.e26297 · ExternalCitation · doi-reference
Explainable artificial intelligence (XAI): concepts, taxonomies, opportunities and challenges toward responsible AI
10.1016/j.inffus.2019.12.012 · ExternalCitation · doi-reference
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10.1016/j.jpha.2024.101144 · ExternalCitation · doi-reference
Stem cell therapeutics and gene therapy for neurologic disorders
10.1016/j.neurot.2024.e00427 · ExternalCitation · doi-reference
Clinical potential of microdystrophin as a surrogate endpoint
10.1016/j.nmd.2022.12.007 · ExternalCitation · doi-reference
EU regulatory pathways for ATMPs: standard, accelerated and adaptive pathways to marketing authorisation
10.1016/j.omtm.2019.01.010 · ExternalCitation · doi-reference
Clinical development of gene therapies: the first three decades and counting
10.1016/j.omtm.2020.10.004 · ExternalCitation · doi-reference
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs
10.1016/j.omtm.2021.04.001 · ExternalCitation · doi-reference
Strengthening health systems for access to gene therapy in rare genetic disorders
10.1016/j.omtm.2024.101220 · ExternalCitation · doi-reference
Optimizing regulatory frameworks for gene therapies in rare diseases: challenges and solutions
10.1016/j.omtm.2024.101386 · ExternalCitation · doi-reference
Unveiling the guidance heterogeneity for genome-informed drug treatment interventions among regulatory bodies and research consortia
10.1016/j.phrs.2019.104590 · ExternalCitation · doi-reference
Consequences of increased longevity for wealth, fertility, and population growth
10.1016/j.physa.2007.09.004 · ExternalCitation · doi-reference
Regulatory systems and requirements for clinical trials of AAV-based gene therapies—perspectives from six Asian countries or regions: report from the 6th Asia Partnership Conference of Regenerative Medicine—April 20, 2023
10.1016/j.reth.2024.06.005 · ExternalCitation · doi-reference
Does the act on the safety of regenerative medicine in Japan ensure “safety”?: implications of low adverse event reporting
10.1016/j.stemcr.2023.10.012 · ExternalCitation · doi-reference
Enabling technologies for personalized and precision medicine
10.1016/j.tibtech.2019.12.021 · ExternalCitation · doi-reference
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10.1016/j.xphs.2019.02.007 · ExternalCitation · doi-reference
Addressing the value of gene therapy and enhancing patient access to transformative treatments
10.1016/j.ymthe.2018.10.017 · ExternalCitation · doi-reference
Equitable access to gene therapy: a call to action for the American society of gene and cell therapy
10.1016/j.ymthe.2018.11.002 · ExternalCitation · doi-reference
Immune responses to viral gene therapy vectors
10.1016/j.ymthe.2020.01.001 · ExternalCitation · doi-reference
Current clinical applications of in vivo gene therapy with AAVs
10.1016/j.ymthe.2020.12.007 · ExternalCitation · doi-reference
Next-generation strategies for gene-targeted therapies of central nervous system disorders: a workshop summary
10.1016/j.ymthe.2021.09.010 · ExternalCitation · doi-reference
Gene therapy access: global challenges, opportunities, and views from Brazil, South Africa, and India
10.1016/j.ymthe.2022.04.002 · ExternalCitation · doi-reference
Planning for progress: a US regulatory approach to advancing the clinical development of gene therapies
10.1016/j.ymthe.2022.05.005 · ExternalCitation · doi-reference
Challenges in accelerated approvals for gene therapies
10.1016/j.ymthe.2023.08.004 · ExternalCitation · doi-reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · ExternalCitation · doi-reference
High-quality health systems in the sustainable development goals era: time for a revolution
10.1016/s2214-109x(18)30386-3 · ExternalCitation · doi-reference
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10.1016/s2214-109x(24)00515-1 · ExternalCitation · doi-reference
The use of N-of-1 trials to generate real-world evidence for optimal treatment of individuals and populations
10.1017/cts.2023.604 · ExternalCitation · doi-reference
Considerable life extension and three views on the meaning of life
10.1017/s0963180110000599 · ExternalCitation · doi-reference
Decision-making capacity evaluation in adult guardianship: a systematic review
10.1017/s1041610215001490 · ExternalCitation · doi-reference
FDA approves first gene therapy for Duchenne muscular dystrophy, despite internal objections
10.1038/d41573-023-00103-y · ExternalCitation · doi-reference
The importance of international collaboration for rare diseases research: a European perspective
10.1038/gt.2017.29 · ExternalCitation · doi-reference
The state of gene therapies: the FDA perspective
10.1038/mt.2012.51 · ExternalCitation · doi-reference
Clinical development of gene therapy: results and lessons from recent successes
10.1038/mtm.2016.34 · ExternalCitation · doi-reference
Therapeutic genome editing: prospects and challenges
10.1038/nm.3793 · ExternalCitation · doi-reference
The ethics of informed consent in Alzheimer disease research
10.1038/nrneurol.2011.76 · ExternalCitation · doi-reference
Viral vector platforms within the gene therapy landscape
10.1038/s41392-021-00487-6 · ExternalCitation · doi-reference
Rules for processing genetic data for research purposes in view of the new EU general data protection regulation
10.1038/s41431-017-0045-7 · ExternalCitation · doi-reference
The interplay of ethics and genetic technologies in balancing the social valuation of the human genome in UNESCO declarations
10.1038/s41431-024-01549-3 · ExternalCitation · doi-reference
The estimated annual financial impact of gene therapy in the United States
10.1038/s41434-023-00419-9 · ExternalCitation · doi-reference
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union
10.1038/s41434-023-00437-7 · ExternalCitation · doi-reference
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial
10.1038/s41591-024-03304-z · ExternalCitation · doi-reference
Gene-based therapies for neurodegenerative diseases
10.1038/s41593-020-00778-1 · ExternalCitation · doi-reference
Gene therapy bio-safety: scientific and regulatory issues
10.1038/sj.gt.3302629 · ExternalCitation · doi-reference
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10.1080/03007995.2023.2257600 · ExternalCitation · doi-reference
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10.1080/13543784.2018.1430137 · ExternalCitation · doi-reference
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10.1080/13698575.2022.2123903 · ExternalCitation · doi-reference
Bench, bedside, boardroom: negotiating translational gene therapy
10.1080/14636778.2017.1289468 · ExternalCitation · doi-reference
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10.1080/20016689.2017.1265293 · ExternalCitation · doi-reference
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10.1080/20016689.2021.2022353 · ExternalCitation · doi-reference
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States
10.1080/20016689.2023.2216518 · ExternalCitation · doi-reference
An ethical assessment of anti-aging medicine
10.1089/109454503322733045 · ExternalCitation · doi-reference
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10.1089/hgtb.2017.078 · ExternalCitation · doi-reference
Prevailing public perceptions of the ethics of gene therapy
10.1089/hum.2014.030 · ExternalCitation · doi-reference
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10.1089/hum.2015.29011.trf · ExternalCitation · doi-reference
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10.1089/hum.2017.175 · ExternalCitation · doi-reference
Public acceptability of gene therapy and gene editing for human use: a systematic review
10.1089/hum.2019.197 · ExternalCitation · doi-reference
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10.1089/hum.2020.217 · ExternalCitation · doi-reference
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10.1089/hum.2024.057 · ExternalCitation · doi-reference
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10.1093/haschl/qxae094 · ExternalCitation · doi-reference
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10.1097/ccm.0000000000002211 · ExternalCitation · doi-reference
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10.1097/ccm.0b013e3181f26fe6 · ExternalCitation · doi-reference
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10.1097/ms9.0000000000001320 · ExternalCitation · doi-reference
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10.1097/qai.0000000000000037 · ExternalCitation · doi-reference
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10.1111/acel.12094 · ExternalCitation · doi-reference
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10.1111/bcp.14807 · ExternalCitation · doi-reference
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10.1111/bcp.14968 · ExternalCitation · doi-reference
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10.1111/cge.12370 · ExternalCitation · doi-reference
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10.1111/cts.12987 · ExternalCitation · doi-reference
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10.1111/dewb.12440 · ExternalCitation · doi-reference
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10.1111/imj.16015 · ExternalCitation · doi-reference
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10.1111/j.1532-5415.1992.tb01995.x · ExternalCitation · doi-reference
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10.1111/j.1552-6909.1994.tb01911.x · ExternalCitation · doi-reference
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10.1126/science.aad1067 · ExternalCitation · doi-reference
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10.1126/science.aan4672 · ExternalCitation · doi-reference
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10.1136/bmjgh-2019-001942 · ExternalCitation · doi-reference
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10.1136/bmjopen-2017-018647 · ExternalCitation · doi-reference
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10.1155/2014/869269 · ExternalCitation · doi-reference
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10.1159/000509119 · ExternalCitation · doi-reference
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10.1172/jci152817 · ExternalCitation · doi-reference
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10.1177/15353702211052280 · ExternalCitation · doi-reference
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10.1177/26330040231164425 · ExternalCitation · doi-reference
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10.1192/s1749367600003039 · ExternalCitation · doi-reference
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10.12688/f1000research.10966.1 · ExternalCitation · doi-reference
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10.1371/journal.pdig.0000651 · ExternalCitation · doi-reference
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10.2174/0115665232279528240115075352 · ExternalCitation · doi-reference
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10.2217/epi-2016-0075 · ExternalCitation · doi-reference
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10.2217/frd-2022-0003 · ExternalCitation · doi-reference
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10.2471/blt.19.237487 · ExternalCitation · doi-reference
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10.3233/jad-180865 · ExternalCitation · doi-reference
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10.3389/fgene.2024.1381172 · ExternalCitation · doi-reference
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10.3389/fmed.2019.00148 · ExternalCitation · doi-reference
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10.3389/fmed.2020.608249 · ExternalCitation · doi-reference
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10.3389/fmed.2023.1252352 · ExternalCitation · doi-reference
10.3389/fmed.2024.1408636
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10.3389/fneur.2021.692527 · ExternalCitation · doi-reference
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10.3389/fonc.2019.00297 · ExternalCitation · doi-reference
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10.3389/fonc.2020.01387 · ExternalCitation · doi-reference
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10.3390/biomedicines13030758 · ExternalCitation · doi-reference
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10.3390/ijms20153801 · ExternalCitation · doi-reference
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10.3390/ijms23031851 · ExternalCitation · doi-reference
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