Research graph
References from Clinical immunogenicity in rAAV gene therapy: Insights and implications. Local targets link to admitted publications; unresolved targets remain external evidence.
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
10.1146/annurev-virology-031413-085355 · 2014 · External reference
Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
10.3390/cells12050785 · 2023 · External reference
AAV vectors: The Rubik's cube of human gene therapy
10.1016/j.ymthe.2022.09.015 · 2022 · External reference
Adeno-associated virus as a delivery vector for gene therapy of human diseases
10.1038/s41392-024-01780-w · 2024 · External reference
Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species
10.1016/j.cell.2021.08.028 · 2021 · External reference
Immunogenicity and toxicity of AAV gene therapy
10.3389/fimmu.2022.975803 · 2022 · External reference
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
10.1182/blood-2010-10-314518 · 2011 · External reference
CpG-depleted adeno-associated virus vectors evade immune detection
10.1172/jci68205 · 2013 · External reference
Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes
10.1089/hum.2023.018 · 2023 · External reference
CD8(+) T-cell responses to adeno-associated virus capsid in humans
10.1038/nm1549 · 2007 · External reference
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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
10.1038/nm1358 · 2006 · External reference
Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions
10.3389/fimmu.2014.00350 · 2014 · External reference
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
10.1056/nejmoa1108046 · 2011 · External reference
Humoral immune responses to AAV gene therapy in the ocular compartment
10.1111/brv.12718 · 2021 · External reference
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
10.1126/scitranslmed.3002865 · 2012 · External reference
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
10.1172/jci200316887 · 2003 · External reference
Tolerance, danger, and the extended family
10.1146/annurev.iy.12.040194.005015 · 1994 · External reference
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
10.1172/jci37607 · 2009 · External reference
Toll–Like Receptor 2–Mediated Innate Immune Response in Human Nonparenchymal Liver Cells Toward Adeno–Associated Viral Vectors
10.1002/hep.24625 · 2012 · External reference
Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction
10.1172/jci.insight.120474 · 2018 · External reference
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
10.3389/fimmu.2020.00670 · 2020 · External reference
Complement is an essential component of the immune response to adeno-associated virus vectors
10.1128/jvi.01990-07 · 2008 · External reference
Control of adaptive immunity by the innate immune system
10.1038/ni.3123 · 2015 · External reference
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8(+) T cells
10.1182/blood-2016-11-751040 · 2017 · External reference
Type I IFN Sensing by cDCs and CD4(+) T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8(+) T Cells
10.1016/j.ymthe.2019.11.011 · 2020 · External reference
TLR9-independent CD8(+) T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling
10.1016/j.ymthe.2023.11.029 · 2024 · External reference
Redundancy in Innate Immune Pathways That Promote CD8(+) T-Cell Responses in AAV1 Muscle Gene Transfer
10.3390/v16101507 · 2024 · External reference
Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence
2021 · External reference
Adeno-associated virus antibody profiles in newborns, children, and adolescents
10.1128/cvi.05107-11 · 2011 · External reference
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A
10.1089/hum.2021.287 · 2022 · External reference
Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors
10.1016/j.omtm.2019.05.014 · 2019 · External reference
Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements
10.1016/j.omtm.2024.101217 · 2024 · External reference
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
10.1016/j.omtm.2022.02.011 · 2022 · External reference
Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood
10.3389/fimmu.2022.999021 · 2022 · External reference
Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
10.1016/j.jhep.2020.11.001 · 2021 · External reference
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
10.1016/j.ymthe.2023.01.010 · 2023 · External reference
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
10.1182/blood-2013-01-306647 · 2013 · External reference
Overcoming innate immune barriers that impede AAV gene therapy vectors
10.1172/jci143780 · 2021 · External reference
T Cell-Mediated Immune Responses to AAV and AAV Vectors
10.3389/fimmu.2021.666666 · 2021 · External reference
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
10.1056/nejmoa1407309 · 2014 · External reference
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant
10.1056/nejmoa1708538 · 2017 · External reference
AAV5-Factor VIII Gene Transfer in Severe Hemophilia A
10.1056/nejmoa1708483 · 2017 · External reference
Gene therapy with adeno-associated virus vector 5–human factor IX in adults with hemophilia B
10.1182/blood-2017-09-804419 · 2018 · External reference
101HEMB01 Is a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Moderate/Severe Hemophilia B That Demonstrated Meaningful but Transient Expression of Human Factor IX (hFIX)
2017 · External reference
BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression
10.1182/blood.2019004625 · 2021 · External reference
Immune Responses in 101HEMB01, a Phase 1/2 Open-Label, Single Ascending Dose-Finding Trial of DTX101 (AAVrh10FIX) in Patients with Severe Hemophilia B
2017 · External reference
Unresolved reference
2018 · External reference
Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies
10.1182/bloodadvances.2024015184 · 2025 · External reference
BAX 335 Hemophilia B Gene Therapy Phase 1/2 Clinical Trial: Long-Term Safety and Efficacy Follow-up
10.1182/blood-2022-163297 · 2022 · External reference
Induction and role of regulatory CD4+CD25+ T cells in tolerance to the transgene product following hepatic in vivo gene transfer
10.1182/blood-2007-02-073304 · 2007 · External reference
Immune regulation and therapeutic application of T regulatory cells in liver diseases
10.3389/fimmu.2024.1371089 · 2024 · External reference
Liver-Mediated Adaptive Immune Tolerance
10.3389/fimmu.2019.02525 · 2019 · External reference
Nonredundant roles of IL-10 and TGF-beta in suppression of immune responses to hepatic AAV-factor IX gene transfer
10.1038/mt.2011.33 · 2011 · External reference
Liver Sinusoidal Endothelial Cells in the Regulation of Immune Responses and Fibrosis in Metabolic Dysfunction-Associated Fatty Liver Disease
10.3390/ijms26093988 · 2025 · External reference
Mechanisms underlying immunosuppression by regulatory cells
10.3389/fimmu.2024.1328193 · 2024 · External reference
Regulatory T-Cell Therapy in Liver Transplantation and Chronic Liver Disease
10.3389/fimmu.2021.719954 · 2021 · External reference
Liver induced transgene tolerance with AAV vectors
10.1016/j.cellimm.2017.12.002 · 2019 · External reference
Induction of factor VIII tolerance by hemophilia gene transfer to eradicate factor VIII inhibitors
10.1182/bloodadvances.2024013000 · 2025 · External reference
Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies
10.1172/jci173510 · 2024 · External reference
THROMBOTIC MICROANGIOPATHIES
10.1056/nejmra020528 · 2022 · External reference
The molecular biology of thrombotic microangiopathy
10.1038/sj.ki.5001535 · 2006 · External reference
Complement Activation and Thrombotic Microangiopathies
10.2215/cjn.05830519 · 2019 · External reference
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Complement System Part I - Molecular Mechanisms of Activation and Regulation
10.3389/fimmu.2015.00262 · 2015 · External reference
AAV-Associated Complement Activation in the Clinical Trial Setting
2020 · External reference
Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy
10.1182/bloodadvances.2021006419 · 2022 · External reference
Abstract 10727: Results from First-in-Human Clinical Trial of RP-A501 (AAV9:LAMP2B) Gene Therapy Treatment for Danon Disease
10.1161/circ.144.suppl_1.10727 · 2021 · External reference
First-in-human nuclease-free homologous recombination-dependent gene editing in pediatric patients with methylmalonic acidemia: results of a phase 1/2 study
10.1038/s41434-026-00609-1 · 2026 · External reference
Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
10.1016/j.jpeds.2020.11.054 · 2021 · External reference
10.1016/j.jtct.2022.11.015
10.1016/j.jtct.2022.11.015 · 2023 · External reference
Clinical Evaluation and Management of Thrombotic Microangiopathy
10.1002/art.42681 · 2024 · External reference
Complement and transplant-associated thrombotic microangiopathy: Current and future approaches
10.1002/hem3.70370 · 2026 · External reference
Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME
10.1016/j.ymthe.2026.05.016 · 2026 · External reference
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies
10.1016/j.ymthe.2025.12.067 · 2026 · External reference
rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis
10.3389/fimmu.2022.1001263 · 2022 · External reference
Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1
10.1016/j.jpeds.2020.05.044 · 2020 · External reference
High-dose AAV gene therapy deaths
10.1038/s41587-020-0642-9 · 2020 · External reference
Hepatotoxicity in Adeno-Associated Viral Vector Gene Therapy
10.1007/s11901-023-00624-5 · 2023 · External reference
Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease?
10.1111/joa.12083 · 2014 · External reference
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Safety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study
10.1016/j.jtha.2024.12.038 · 2025 · External reference
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
10.1056/nejmoa2104205 · 2021 · External reference
Optimizing liver health before and after gene therapy for hemophilia A
10.1182/bloodadvances.2024013059 · 2024 · External reference
Valoctocogene roxaparvovec gene therapy for hemophilia A
10.1056/nejmoa2113708 · 2022 · External reference
Two-year outcomes of valoctocogene roxaparvovec therapy for hemophilia A
10.1056/nejmoa2211075 · 2023 · External reference
Human liver sinusoidal endothelial cells but not hepatocytes contain factor VIII
10.1111/jth.12412 · 2014 · External reference
Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice
10.1038/mtm.2016.64 · 2016 · External reference
Induction of ER Stress by an AAV5 BDD FVIII Construct Is Dependent on the Strength of the Hepatic-Specific Promoter
10.1016/j.omtm.2020.07.005 · 2020 · External reference
Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A
10.1038/s41591-022-01751-0 · 2022 · External reference
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial
10.1016/s1474-4422(23)00313-7 · 2023 · External reference
Loss of Mtm1 causes cholestatic liver disease in a model of X-linked myotubular myopathy
10.1172/jci166275 · 2023 · External reference
Intrahepatic Cholestasis Is a Clinically Significant Feature Associated with Natural History of X-Linked Myotubular Myopathy (XLMTM): A Case Series and Biopsy Report
10.3233/jnd-210712 · 2022 · External reference
AAV8 gene therapy and dietary insults together precipitate cholestatic liver disease in a mouse model of X-linked myotubular myopathy
10.1126/scitranslmed.adr1483 · 2026 · External reference
Immunotherapy and Gene Therapy: New Challenges in the Diagnosis and Management of Drug-Induced Liver Injury
2021 · External reference
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method
10.1016/j.omtm.2021.05.012 · 2021 · External reference
Sirolimus for the treatment of steroid-refractory hepatotoxicity following AAV gene therapy in patients with Duchenne muscular dystrophy
10.1177/22143602261424971 · 2026 · External reference
Long COVID-19 Myocarditis and Various Heart Failure Presentations: A Case Series
10.3390/jcdd9120427 · 2022 · External reference
Dystrophin Immunity after Gene Therapy for Duchenne’s Muscular Dystrophy
10.1056/nejmc2212912 · 2023 · External reference
Immune transgene-dependent myocarditis in macaques after systemic administration of adeno-associated virus expressing human acid alpha-glucosidase
10.3389/fimmu.2023.1094279 · 2023 · External reference
2024 ACC Expert Consensus Decision Pathway on Strategies and Criteria for the Diagnosis and Management of Myocarditis: A Report of the American College of Cardiology Solution Set Oversight Committee
10.1016/j.jacc.2024.10.080 · 2025 · External reference
Gene therapy vector-related myocarditis
10.1016/j.ijcard.2023.131617 · 2024 · External reference
Drug development progress in duchenne muscular dystrophy
10.3389/fphar.2022.950651 · 2022 · External reference
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2023 · External reference
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2022 · External reference
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Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy
10.1007/s40264-021-01107-6 · 2021 · External reference
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2023 · External reference
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Unexpected Death of a Duchenne Muscular Dystrophy Patient in an N-of-1 Trial of rAAV9-delivered CRISPR-transactivator
2023 · External reference
410P GNT0004, Genethon's AAV8 vector-delivered microdystrophin gene therapy of Duchenne muscular dystrophy, first data of the phase I/II part of the GNT-016-MDYF all-in-one clinical trial in ambulant boys
10.1016/j.nmd.2024.07.290 · 2024 · External reference
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2019 · External reference
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2020 · External reference
Rocket Pharmaceuticals announces positive gene expression, clinical biomarker and preliminary functional data from Phase 1 trial of RP-A501 for the treatment of Danon disease
2020 · External reference
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Emerging Thrombotic Disorders Associated with Virus-Based Innovative Therapies: From VITT to AAV Gene Therapy–Related Thrombotic Microangiopathy
2024 · External reference
258P Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing≤ 17 kg and≤ 24 months old: phase 4 OFELIA study
10.1016/j.nmd.2024.07.475 · 2024 · External reference
Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment
10.1186/s13023-024-03326-3 · 2024 · External reference
Danon Disease phase 1 RP-A501 results: the first single-dose intravenous (IV) gene therapy with recombinant adeno-associated virus (AAV9:LAMP2B) for a monogenic cardiomyopathy European Society of Gene and Cell Therapy (ESGCT) Annual Congress
2023 · External reference
Demonstration of nuclease-free genome editing (GeneRide) in pediatric patients with methylmalonic acidemia
2024 · External reference
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Intrathecal gene therapy for giant axonal neuropathy
10.1056/nejmoa2307952 · 2024 · External reference
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
2024 · External reference
Real-world outcomes in patients with spinal muscular atrophy treated with onasemnogene abeparvovec monotherapy: findings from the RESTORE registry
10.3233/jnd-230122 · 2024 · External reference
10.1016/s1096-7192(21)00288-2
10.1016/s1096-7192(21)00288-2 · External reference
Results from the CANaspire Gene Therapy Trial for Canavan Disease: Safety, Biomarker, Imaging, and Clinical Outcome Data from the Completed Low-dose Cohort
2024 · External reference
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2023 · External reference
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FP. 28 IGNITE DMD phase I/II study of SGT-001 microdystrophin gene therapy for DMD: Long-term outcomes and expression update
10.1016/j.nmd.2022.07.234 · 2022 · External reference
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Onasemnogene abeparvovec: post-infusion efficacy and safety in patients with spinal muscular atrophy (SMA)—a fondazione policlinico gemelli IRCCS experience
10.1177/00185787231182562 · 2024 · 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
Use of Prophylactic Steroids to Mitigate Potential T-Cell Response in AAV8-Mediated hLDLR Gene Transfer in Subjects with Homozygous Familial Hypercholesterolemia
2020 · External reference
Diagnosis and Treatment of Acute Myocarditis: A Review
10.1001/jama.2023.3371 · 2023 · External reference
Current Treatment and Immunomodulation Strategies in Acute Myocarditis
10.1097/fjc.0000000000001542 · 2024 · External reference
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
10.1089/hum.2020.167 · 2020 · External reference
Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN
10.1089/hum.2018.015 · 2018 · External reference
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
10.1126/scitranslmed.aba9188 · 2020 · External reference
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
10.1056/nejmoa2005056 · 2020 · External reference
Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species
10.1016/j.omtm.2022.03.017 · 2022 · External reference
The influence of virus structure on antibody responses and virus serotype formation
10.1016/s0167-5699(96)10066-9 · 1996 · External reference
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies
10.1016/j.omtm.2024.101273 · 2024 · 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
Strategy to detect pre-existing immunity to AAV gene therapy
10.1038/gt.2017.95 · 2017 · External reference
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays
10.1208/s12248-019-0403-1 · 2020 · External reference
Etranacogene dezaparvovec in people with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: a 4-year subgroup analysis of the Health Outcomes with Padua Gene; Evaluation in Hemophilia B (HOPE-B) trial
10.1016/j.rpth.2025.103321 · 2026 · External reference
Etranacogene dezaparvovec in people with hemophilia B with preexisting adeno-associated virus 5 neutralizing antibodies: 4-year subgroup results from the HOPE-B trial
2026 · External reference
Unresolved reference
2021 · External reference
Systemic Toxicity of Recombinant Adeno-Associated Virus Gene Therapy Vectors
10.1177/01926233241298892 · 2024 · External reference
Structural and Functional Changes With the Aging Kidney
10.1053/j.ackd.2015.08.004 · 2016 · External reference
Age and immunity: What is “immunosenescence”
10.1016/j.exger.2017.10.024 · 2018 · External reference
Frailty in elderly people
10.1016/s0140-6736(12)62167-9 · 2013 · External reference
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
10.1038/gt.2010.157 · 2011 · External reference
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
10.1016/j.ymthe.2020.01.026 · 2020 · External reference
Effect of CpG Depletion of Vector Genome on CD8(+) T Cell Responses in AAV Gene Therapy
10.3389/fimmu.2021.672449 · 2021 · External reference
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
10.1016/s0140-6736(17)31868-8 · 2017 · External reference
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10.1056/nejmoa1706198 · 2017 · External reference
Can mTOR inhibitors reduce the risk of late kidney allograft failure?
2008 · External reference
Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches
10.1089/hum.2022.138 · 2022 · External reference
A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders
10.1016/j.ymthe.2024.07.016 · 2024 · External reference
First-in-human study of TN-201, an AAV9 gene replacement therapy in MYBPC3-associated hypertrophic cardiomyopathy: initial safety, pharmacodynamic, and imaging results from MyPEAK-1
10.1093/cvr/cvaf200 · 2025 · External reference
Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease
10.1056/nejmoa2412392 · 2025 · External reference
Progressive multifocal leukoencephalopathy in rituximab-treated rheumatic diseases: a rare event
10.1007/s13365-018-0615-7 · 2018 · External reference
Gene Therapy With Regulatory T Cells: A Beneficial Alliance
10.3389/fimmu.2018.00554 · 2018 · External reference
Immune Responses to Viral Gene Therapy Vectors
10.1016/j.ymthe.2020.01.001 · 2020 · External reference
Update on clinical gene therapy for hemophilia
10.1182/blood-2018-07-820720 · 2019 · External reference
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme
10.1172/jci.insight.139881 · 2020 · External reference
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
10.1038/s41591-020-0911-7 · 2020 · External reference
Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates
10.1016/j.omtm.2022.12.012 · 2023 · External reference
Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs
10.1016/j.omtm.2019.05.009 · 2019 · External reference
Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll
10.1016/j.ymthe.2020.07.006 · 2020 · External reference
Efficient Capsid Antigen Presentation From Adeno-Associated Virus Empty Virions In Vivo
10.3389/fimmu.2018.00844 · 2018 · External reference
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Emerging therapies for hemophilia: controversies and unanswered questions
10.12688/f1000research.12491.1 · 2018 · External reference
