AAV vectors: The Rubik’s cube of human gene therapy

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Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice

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A truncated reverse transcriptase enhances prime editing by split AAV vectors

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Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25

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Novel AAV44.9-Based Vectors Display Exceptional Characteristics for Retinal Gene Therapy

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Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

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AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice

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Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec,an AAV5-Mediated Gene Therapy for Hemophilia A

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AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease

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AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes

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AAV1.SERCA2a Gene Therapy Reverses Pulmonary Fibrosis by Blocking the STAT3/FOXM1 Pathway and Promoting the SNON/SKI Axis

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Local magnetic delivery of adeno-associated virus AAV2(quad Y-F)-mediated BDNF gene therapy restores hearing after noise injury

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Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial

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Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson’s disease

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AAV integration in human hepatocytes

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Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice

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Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders

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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

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Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning

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Non-viral Gene Therapy for Stargardt Disease with ECO/pRHO-ABCA4 Self-Assembled Nanoparticles

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