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A STEP into Darkness or Light?

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Adeno-Associated Viral Vectors for Retinal Gene Transfer and Treatment of Retinal Diseases

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Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

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Viral vector platforms within the gene therapy landscape

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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

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Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors

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Immune responses to AAV vectors: overcoming barriers to successful gene therapy

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Emerging issues in AAV-mediated in vivo gene therapy

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Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges

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Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

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Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID): A Phase 2 Trial of Intracoronary Gene Therapy of Sarcoplasmic Reticulum Ca ²⁺ -ATPase in Patients With Advanced Heart Failure

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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

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Gene Therapy

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Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

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Selection and evaluation of clinically relevant AAV variants in a xenograft liver model

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Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

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Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

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Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

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Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

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Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents

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Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

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Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

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In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

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Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery

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Immune responses to retinal gene therapy using adeno-associated viral vectors – implications for treatment success and safety

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Humoral immune response to AAV

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Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

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Unraveling the complex story of immune responses to AAV vectors trial after trial.

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Overcoming innate immune barriers that impede AAV gene therapy vectors

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Structural insight into the unique properties of adeno-associated virus serotype 9

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Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia

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Pharmaceutical development of AAV-based gene therapy products for the eye

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Results at 2 Years after gene therapy for RPE65-deficient leber congenital amaurosis and severe early-childhood–onset retinal dystrophy

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Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

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Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

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Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors

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A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

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Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

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Innate Immune Responses to AAV Vectors

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Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery

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rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis

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Neutralizing antibodies against AAV serotypes 1, 2, 6, and 9 in sera of commonly used animal models

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Tailoring the AAV vector capsid for gene therapy

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Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

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Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

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Efficient Serotype-Dependent Release of Functional Vector into the Culture Medium During Adeno-Associated Virus Manufacturing

Vandenberghe, L. H., Xiao, R., Lock, M., Lin, J., Korn, M., & Wilson, J. M. (2010) · Human Gene Therapy · doi:10.1089/hum.2010.107 · Cited by 151 · see citing works

AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression

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Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme

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Adeno-Associated Virus Gene Therapy for Liver Disease

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Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

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The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

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Novel adeno-associated viral vectors for retinal gene therapy.

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Capsid antibodies to different adeno-associated virus serotypes bind common regions

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Therapeutic hFIX activity achieved after single AAV5-hFIX treatment in hemophilia b patients and NHPs with pre-existing anti-AAV5 NABs

Majowicz, A., Nijmeijer, B., Lampen, M. H., Spronck, L., de Haan, M., Petry, H., van Deventer, S. J., Meyer, C., Tangelder, M., & Ferreira, V. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.05.009 · Cited by 126 · see citing works

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations

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Adeno-Associated Virus (AAV) Versus Immune Response

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