Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species

Overcoming innate immune barriers that impede AAV gene therapy vectors

(2021) Manish Muhuri et al.   JOURNAL OF CLINICAL INVESTIGATION

Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening

(2020) Kathleen Börner et al.   MOLECULAR THERAPY

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV

(2020) Leon Morales et al.   MOLECULAR THERAPY

Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy

(2020) Jerry R. Mendell et al.   JAMA Neurology

Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning

(2020) Mathieu Nonnenmacher et al.   Molecular Therapy-Methods & Clinical Development

In Situ Modification of Tissue Stem and Progenitor Cell Genomes

(2019) Jill M. Goldstein et al.   Cell Reports

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

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor

(2018) Amanda M. Dudek et al.   JOURNAL OF VIROLOGY

Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy

(2018) Dongsheng Duan   MOLECULAR THERAPY

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

(2018) Juliette Hordeaux et al.   MOLECULAR THERAPY

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

(2017) David L. Mack et al.   MOLECULAR THERAPY

Long-term effects of systemic gene therapy in a canine model of myotubular myopathy

(2017) Matthew Elverman et al.   MUSCLE & NERVE

Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion

(2017) Longping Victor Tse et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

(2017) Niclas E. Bengtsson et al.   Nature Communications

A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy

(2017) Chady H. Hakim et al.   Molecular Therapy-Methods & Clinical Development

Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries

(2016) Jakob Körbelin et al.   MOLECULAR THERAPY

Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer

(2016) Chengwen Li et al.   MOLECULAR THERAPY

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy

(2016) Sourav R Choudhury et al.   MOLECULAR THERAPY

An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

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

(2016) Benjamin E Deverman et al.   NATURE BIOTECHNOLOGY

Cellular transduction mechanisms of adeno-associated viral vectors

(2016) Garrett Edward Berry et al.   Current Opinion in Virology

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

(2015) M. Tabebordbar et al.   SCIENCE

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

(2015) C. E. Nelson et al.   SCIENCE

Human skeletal muscle xenograft as a new preclinical model for muscle disorders

(2014) Y. Zhang et al.   HUMAN MOLECULAR GENETICS

MicroRNA-486–dependent modulation of DOCK3/PTEN/AKT signaling pathways improves muscular dystrophy–associated symptoms

(2014) Matthew S. Alexander et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene Therapy Prolongs Survival and Restores Function in Murine and Canine Models of Myotubular Myopathy

(2014) M. K. Childers et al.   Science Translational Medicine

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates

(2014) Darren A. Murrey et al.   Human Gene Therapy Clinical Development

In Vivo-Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous

(2013) D. Dalkara et al.   Science Translational Medicine

Creation of a cardiotropic adeno-associated virus: the story of viral directed evolution

(2013) Lin Yang et al.   Virology Journal

Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

(2012) M. A. DiMattia et al.   JOURNAL OF VIROLOGY

The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

(2011) Christie L. Bell et al.   JOURNAL OF CLINICAL INVESTIGATION

Successful Expansion but Not Complete Restriction of Tropism of Adeno-Associated Virus by In Vivo Biopanning of Random Virus Display Peptide Libraries

(2009) Stefan Michelfelder et al.   PLoS One

A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

(2009) L. Yang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Highly Efficient, Functional Engraftment of Skeletal Muscle Stem Cells in Dystrophic Muscles

(2008) Massimiliano Cerletti et al.   CELL

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY