Adeno-associated virus vector as a platform for gene therapy delivery

Real-Time DNA Sequencing from Single Polymerase Molecules

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Non-viral vectors for gene-based therapy

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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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RNA targeting with CRISPR–Cas13

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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

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Effect of gene therapy on visual function in Leber’s congenital amaurosis

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

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Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

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Base editing: precision chemistry on the genome and transcriptome of living cells

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Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

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Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

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Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors

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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

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Clades of Adeno-Associated Viruses Are Widely Disseminated in Human Tissues

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

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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain

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Adenovirus-Associated Defective Virus Particles

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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

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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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Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

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Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector

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Site-specific integration by adeno-associated virus.

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Emerging issues in AAV-mediated in vivo gene therapy

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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

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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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Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses

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Site-specific integration by adeno-associated virus.

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Targeted integration of adeno‐associated virus (AAV) into human chromosome 19.

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Engineering adeno-associated viruses for clinical gene therapy

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Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors

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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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Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

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Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression

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Nucleotide sequence and organization of the adeno-associated virus 2 genome

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In Vitro and In Vivo Gene Therapy Vector Evolution via Multispecies Interbreeding and Retargeting of Adeno-Associated Viruses

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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

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AAV vector integration sites in mouse hepatocellular carcinoma

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Dystrophin Immunity in Duchenne's Muscular Dystrophy

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Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

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A Multifunctional AAV-CRISPR-Cas9 and Its Host Response

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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

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Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

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Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

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Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

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Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

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Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis

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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

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Antibody-based protection against HIV infection by vectored immunoprophylaxis

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Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors

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An essential receptor for adeno-associated virus infection

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Circular Intermediates of Recombinant Adeno-Associated Virus Have Defined Structural Characteristics Responsible for Long-Term Episomal Persistence in Muscle Tissue

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Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

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Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.

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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

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Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer

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Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

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Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

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

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Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance

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The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice

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Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

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Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2

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Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

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

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