Abstract
No public abstract is available from the verified source records.
Contact and support
Need help, have a question, or want to contact the ResearchHub team?
© 2026 ResearchHub. Built for responsible scholarly connection.
Privacy-aware community infrastructureNitish Gulve, Mariana Bego, Hitesh Gandhi, Zilehuma Khan, Olivia Tong, Sara Katebifar, Manoj Rajadhyaksha
Abstract
Authors
Institutions
No ROR-resolved institution is linked to this work yet.
Provenance
crossref
Confidence 100%
pubmed
Confidence 98%
No local reference links have been materialized yet.
No local citing links have been materialized yet.
AAV-Mediated Gene Therapy for Research and Therapeutic Purposes
10.1146/annurev-virology-031413-085355 · 2014
Various AAV Serotypes and Their Applications in Gene Therapy: An Overview
10.3390/cells12050785 · 2023
AAV vectors: The Rubik's cube of human gene therapy
10.1016/j.ymthe.2022.09.015 · 2022
Adeno-associated virus as a delivery vector for gene therapy of human diseases
10.1038/s41392-024-01780-w · 2024
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
Immunogenicity and toxicity of AAV gene therapy
10.3389/fimmu.2022.975803 · 2022
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
CpG-depleted adeno-associated virus vectors evade immune detection
10.1172/jci68205 · 2013
Complement Activation by Adeno-Associated Virus-Neutralizing Antibody Complexes
10.1089/hum.2023.018 · 2023
europepmc
Confidence 96%
unpaywall
Confidence 95%
datacite
Confidence 0%
CD8(+) T-cell responses to adeno-associated virus capsid in humans
10.1038/nm1549 · 2007
Unresolved referenced work
Kept as external metadata until matched
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
10.1038/nm1358 · 2006
Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions
10.3389/fimmu.2014.00350 · 2014
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
10.1056/nejmoa1108046 · 2011
Humoral immune responses to AAV gene therapy in the ocular compartment
10.1111/brv.12718 · 2021
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness
10.1126/scitranslmed.3002865 · 2012
Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
10.1172/jci200316887 · 2003
Tolerance, danger, and the extended family
10.1146/annurev.iy.12.040194.005015 · 1994
The TLR9-MyD88 pathway is critical for adaptive immune responses to adeno-associated virus gene therapy vectors in mice
10.1172/jci37607 · 2009
Toll–Like Receptor 2–Mediated Innate Immune Response in Human Nonparenchymal Liver Cells Toward Adeno–Associated Viral Vectors
10.1002/hep.24625 · 2012
Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction
10.1172/jci.insight.120474 · 2018
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors
10.3389/fimmu.2020.00670 · 2020
Complement is an essential component of the immune response to adeno-associated virus vectors
10.1128/jvi.01990-07 · 2008
Control of adaptive immunity by the innate immune system
10.1038/ni.3123 · 2015
Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8(+) T cells
10.1182/blood-2016-11-751040 · 2017
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
TLR9-independent CD8(+) T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling
10.1016/j.ymthe.2023.11.029 · 2024
Redundancy in Innate Immune Pathways That Promote CD8(+) T-Cell Responses in AAV1 Muscle Gene Transfer
10.3390/v16101507 · 2024
Adeno-Associated Virus (AAV)-Mediated Gene Therapy for Duchenne Muscular Dystrophy: The Issue of Transgene Persistence
2021
Adeno-associated virus antibody profiles in newborns, children, and adolescents
10.1128/cvi.05107-11 · 2011
Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A
10.1089/hum.2021.287 · 2022
Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors
10.1016/j.omtm.2019.05.014 · 2019
Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements
10.1016/j.omtm.2024.101217 · 2024
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations
10.1016/j.omtm.2022.02.011 · 2022
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
Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
10.1016/j.jhep.2020.11.001 · 2021
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy
10.1016/j.ymthe.2023.01.010 · 2023
Immune responses to AAV vectors: overcoming barriers to successful gene therapy
10.1182/blood-2013-01-306647 · 2013
Overcoming innate immune barriers that impede AAV gene therapy vectors
10.1172/jci143780 · 2021
T Cell-Mediated Immune Responses to AAV and AAV Vectors
10.3389/fimmu.2021.666666 · 2021
Emerging therapies for hemophilia: controversies and unanswered questions
10.12688/f1000research.12491.1 · doi-reference
Efficient Capsid Antigen Presentation From Adeno-Associated Virus Empty Virions In Vivo
10.3389/fimmu.2018.00844 · doi-reference
Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll
10.1016/j.ymthe.2020.07.006 · doi-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 · doi-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 · doi-reference
IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies
10.1038/s41591-020-0911-7 · doi-reference
Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme
10.1172/jci.insight.139881 · doi-reference
Update on clinical gene therapy for hemophilia
10.1182/blood-2018-07-820720 · doi-reference
Immune Responses to Viral Gene Therapy Vectors
10.1016/j.ymthe.2020.01.001 · doi-reference
Gene Therapy With Regulatory T Cells: A Beneficial Alliance
10.3389/fimmu.2018.00554 · doi-reference
Progressive multifocal leukoencephalopathy in rituximab-treated rheumatic diseases: a rare event
10.1007/s13365-018-0615-7 · doi-reference
Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease
10.1056/nejmoa2412392 · doi-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 · doi-reference
A systematic review of immunosuppressive protocols used in AAV gene therapy for monogenic disorders
10.1016/j.ymthe.2024.07.016 · doi-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 · doi-reference
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy
10.1056/nejmoa1706198 · doi-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 · doi-reference
Effect of CpG Depletion of Vector Genome on CD8(+) T Cell Responses in AAV Gene Therapy
10.3389/fimmu.2021.672449 · doi-reference
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?
10.1016/j.ymthe.2020.01.026 · doi-reference
Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver
10.1038/gt.2010.157 · doi-reference
Frailty in elderly people
10.1016/s0140-6736(12)62167-9 · doi-reference
Age and immunity: What is “immunosenescence”
10.1016/j.exger.2017.10.024 · doi-reference
Structural and Functional Changes With the Aging Kidney
10.1053/j.ackd.2015.08.004 · doi-reference
Systemic Toxicity of Recombinant Adeno-Associated Virus Gene Therapy Vectors
10.1177/01926233241298892 · doi-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 · doi-reference
Recommendations for the Development of Cell-Based Anti-Viral Vector Neutralizing Antibody Assays
10.1208/s12248-019-0403-1 · doi-reference
Strategy to detect pre-existing immunity to AAV gene therapy
10.1038/gt.2017.95 · doi-reference
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders
10.3390/ijms25021050 · doi-reference
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies
10.1016/j.omtm.2024.101273 · doi-reference
The influence of virus structure on antibody responses and virus serotype formation
10.1016/s0167-5699(96)10066-9 · doi-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 · doi-reference
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS
10.1056/nejmoa2005056 · doi-reference
MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates
10.1126/scitranslmed.aba9188 · doi-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 · doi-reference
Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology
10.1089/hum.2020.167 · doi-reference
Current Treatment and Immunomodulation Strategies in Acute Myocarditis
10.1097/fjc.0000000000001542 · doi-reference
Diagnosis and Treatment of Acute Myocarditis: A Review
10.1001/jama.2023.3371 · doi-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 · doi-reference
Onasemnogene abeparvovec: post-infusion efficacy and safety in patients with spinal muscular atrophy (SMA)—a fondazione policlinico gemelli IRCCS experience
10.1177/00185787231182562 · doi-reference
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 · doi-reference