Research graph
References from Design and initial characterization of a novel mini-promoter for gene therapies targeting the central nervous system. Local targets link to admitted publications; unresolved targets remain external evidence.
Adeno-associated virus vector as a platform for gene therapy delivery
10.1038/s41573-019-0012-9 · 2019 · External reference
Mammalian Stable Cell Platforms for Recombinant Adeno-associated Virus (rAAV) Production: Development Strategies and their Impact on Viral Productivity
10.2174/1566523223666230331111336 · 2023 · External reference
Moving Forward After Two Deaths in a Gene Therapy Trial of Myotubular Myopathy
10.1089/hum.2020.182 · 2020 · External reference
Food and Drug Administration Lifts Clinical Hold on Pfizer Duchenne Muscular Dystrophy Gene Therapy Linked to Patient Death
10.1089/hum.2022.29211.bfs · 2022 · External reference
Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy
10.1182/bloodadvances.2021006419 · 2022 · External reference
Sarepta's DMD gene therapy falls flat
2021 · External reference
AAV Vectors: Are They Safe?
10.1089/hum.2020.187 · 2020 · External reference
AAV-DJ is superior to AAV9 for targeting brain and spinal cord, and de-targeting liver across multiple delivery routes in mice
10.1186/s12967-024-05599-5 · 2024 · External reference
Targeting Adeno-Associated Virus Vectors for Local Delivery to Fractures and Systemic Delivery to the Skeleton
10.1016/j.omtm.2019.08.010 · 2019 · External reference
Metronidazole-Induced Hepatitis in a Teenager With Xeroderma Pigmentosum and Trichothiodystrophy Overlap
10.1542/peds.2021-050360 · 2021 · External reference
Hepatic Failure following Metronidazole in Children with Cockayne Syndrome
2020 · External reference
Metronidazole-induced hepatotoxicity in a patient with xeroderma pigmentosum: A case report
2022 · External reference
Metronidazole Toxicity in Cockayne Syndrome: A Case Series
10.1542/peds.2015-0531 · 2015 · External reference
Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice
10.1038/s41434-023-00411-3 · 2024 · External reference
Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia
10.3390/jcm10112471 · 2021 · External reference
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
10.1056/nejmoa1407309 · 2014 · External reference
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10.1056/nejmoa1706198 · 2017 · External reference
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper
10.1016/j.omtm.2022.07.018 · 2022 · External reference
Lethal immunotoxicity in high-dose systemic AAV therapy
10.1016/j.ymthe.2023.10.015 · 2023 · External reference
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders
10.3390/ijms25021050 · 2024 · External reference
Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice
10.1186/1479-0556-6-13 · 2008 · External reference
Intravitreal Delivery of rAAV2-hSyn-hRS1 Results in Retinal Ganglion Cell-Specific Gene Expression and Retinal Improvement in the Rs1-KO Mouse
10.1089/hum.2023.209 · 2024 · External reference
AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo
10.1016/j.ymthe.2020.03.007 · 2020 · External reference
AAV-mediated liver-directed gene therapy
10.1007/978-1-61779-370-7_6 · 2011 · External reference
AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome
10.1089/hum.2018.020 · 2019 · External reference
Transcriptional regulation of the tissue-type plasminogen-activator gene in human endothelial cells: identification of nuclear factors that recognise functional elements in the tissue-type plasminogen-activator gene promoter
10.1046/j.1432-1327.1998.2580123.x · 1998 · External reference
New core promoter element in RNA polymerase II-dependent transcription: sequence-specific DNA binding by transcription factor IIB
10.1101/gad.12.1.34 · 1998 · External reference
Regulation of gene expression via the core promoter and the basal transcriptional machinery
10.1016/j.ydbio.2009.08.009 · 2010 · External reference
Homotypic clusters of transcription factor binding sites are a key component of human promoters and enhancers
10.1101/gr.104471.109 · 2010 · External reference
Battling Neurodegenerative Diseases with Adeno-Associated Virus-Based Approaches
10.3390/v12040460 · 2020 · External reference
Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle
10.1002/jgm.516 · 2004 · External reference
Comparison of the EF-1 alpha and the CMV promoter for engineering stable tumor cell lines using recombinant adeno-associated virus
2002 · External reference
Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury
10.1038/s41434-019-0075-6 · 2019 · External reference
A Novel Small NPC1 Promoter Enhances AAV-Mediated Gene Therapy in Mouse Models of Niemann-Pick Type C1 Disease
10.3390/cells12121619 · 2023 · External reference
Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor
10.1038/s44321-024-00037-x · 2024 · External reference
Quantitative analysis of the packaging capacity of recombinant adeno-associated virus
10.1089/hum.1996.7.17-2101 · 1996 · External reference
Cognitive Decline and Other Late-Stage Neurologic Complications in Cockayne Syndrome
10.1212/cpj.0000000000200309 · 2024 · External reference
The Genotype-Tissue Expression (GTEx) project
10.1038/ng.2653 · 2013 · External reference
Sequence features that drive human promoter function and tissue specificity
10.1101/gr.100370.109 · 2010 · External reference
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors
10.1016/j.omtm.2023.05.013 · 2023 · External reference
Design of a Novel Gene Therapy Construct to Achieve Sustained Brain-Derived Neurotrophic Factor Signaling in Neurons
10.1089/hum.2017.069 · 2018 · External reference
Receptor-ligand supplementation via a self-cleaving 2A peptide-based gene therapy promotes CNS axonal transport with functional recovery
10.1126/sciadv.abd2590 · 2021 · External reference
Split intein-mediated protein trans-splicing to express large dystrophins
10.1038/s41586-024-07710-8 · 2024 · External reference
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors
10.1089/hum.2010.245 · 2011 · External reference
Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial
10.1038/gt.2012.43 · 2013 · External reference
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy
10.1038/mt.2014.200 · 2015 · External reference
Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector
10.1016/0006-8993(95)01488-8 · 1996 · External reference
Recombinant AAV-mediated gene delivery to the central nervous system
10.1002/jgm.506 · 2004 · External reference
Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain
10.1038/sj.gt.3301221 · 2000 · External reference
Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors
10.1006/exnr.1997.6736 · 1998 · External reference
Better Targeting, Better Efficiency for Wide-Scale Neuronal Transduction with the Synapsin Promoter and AAV-PHP
2016 · External reference
Promoter analysis of RPE65, the gene encoding a 61-kDa retinal pigment epithelium-specific protein
1998 · External reference
Analysis of the VMD2 promoter and implication of E-box binding factors in its regulation
10.1074/jbc.m309881200 · 2004 · External reference
Vector platforms for gene therapy of inherited retinopathies
10.1016/j.preteyeres.2014.08.001 · 2014 · External reference
Use of the human EF-1alpha promoter for expression can significantly increase success in establishing stable cell lines with consistent expression: a study using the tetracycline-inducible system in human cancer cells
10.1093/nar/27.24.4775 · 1999 · External reference
A comparative analysis of constitutive promoters located in adeno-associated viral vectors
10.1371/journal.pone.0106472 · 2014 · External reference
Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter
10.1038/sj.gt.3301561 · 2001 · External reference
Assessment of the long-term transcriptional activity of a 550-bp-long human beta-actin promoter region
10.1016/j.plasmid.2012.07.003 · 2012 · External reference
Systematic comparison of constitutive promoters and the doxycycline-inducible promoter
10.1371/journal.pone.0010611 · 2010 · External reference
Generation of a synthetic mammalian promoter library by modification of sequences spacing transcription factor binding sites
10.1016/s0378-1119(02)00878-8 · 2002 · External reference
AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 disease
10.1172/jci146286 · 2022 · External reference
Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease
10.1089/hum.2016.013 · 2016 · External reference
Gene therapy for CNS disorders: modalities, delivery and translational challenges
10.1038/s41583-024-00829-7 · 2024 · External reference
Designing and optimizing AAV-mediated gene therapy for neurodegenerative diseases: from bench to bedside
10.1186/s12967-024-05661-2 · 2024 · External reference
Regulation of the hypoxia-inducible factor-1 alpha. ARNT is not necessary for hypoxic induction of HIF-1 alpha in the nucleus
10.1007/0-306-46825-5_9 · 2000 · External reference
Evaluation of promoters for use in tissue-specific gene delivery
2008 · External reference
CRISPR-Cas9: Transforming Functional Genomics, Precision Medicine, and Drug Development - Opportunities, Challenges, and Future Directions
2025 · External reference
Immune Responses to Viral Gene Therapy Vectors
10.1016/j.ymthe.2020.01.001 · 2020 · External reference
Gene Expression Signatures Associated With Immune and Virological Responses to Therapeutic Vaccination With Dendritic Cells in HIV-Infected Individuals
10.3389/fimmu.2019.00874 · 2019 · External reference
Promoter deletion analysis using a dual-luciferase reporter system
10.1007/978-1-62703-284-1_7 · 2013 · External reference
Does not hUTP14a promoter form a regulation feedback loop with P53?
2014 · External reference
High-efficiency transduction of liver cancer cells by recombinant adeno-associated virus serotype 3 vectors
10.3791/2538 · 2011 · External reference
Tumor antigen-loaded AAV vaccine drives protective immunity in a melanoma animal model
10.1016/j.omtm.2023.01.006 · 2023 · External reference
Rationally designed capsid and transgene cassette of AAV6 vectors for dendritic cell-based cancer immunotherapy
10.1038/icb.2013.74 · 2014 · External reference
Modifiers of Adeno-Associated Virus-Mediated Gene Expression in Implication for Serotype-Universal Neutralizing Antibody Assay
10.1089/hum.2020.074 · 2020 · External reference
Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo
10.1161/01.res.0000237661.18885.f6 · 2006 · External reference
Intrathecal substance P elicits a caudally-directed biting and scratching behavior in mice
10.1016/0006-8993(81)90203-1 · 1981 · External reference
Intrathecal delivery of AAV-NDNF ameliorates disease progression of ALS mice
10.1016/j.ymthe.2023.09.018 · 2023 · External reference
Intrathecal morphine in mice: a new technique
10.1016/0014-2999(80)90515-4 · 1980 · External reference
An optimized intracerebroventricular injection of CD4(+) T cells into mice
10.1016/j.xpro.2021.100725 · 2021 · External reference
Lipofuscin-like autofluorescence within microglia and its impact on studying microglial engulfment
10.1038/s41467-023-42809-y · 2023 · External reference
