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

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(2015) Anca M Paşca et al.   NATURE METHODS

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(2015) Jennifer B Treweek et al.   Nature Protocols

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(2015) Juan Carlos Izpisua Belmonte et al.   NEURON

Single-Cell Phenotyping within Transparent Intact Tissue through Whole-Body Clearing

(2014) Bin Yang et al.   CELL

Current status of haemophilia gene therapy

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Intrajugular Vein Delivery of AAV9-RNAi Prevents Neuropathological Changes and Weight Loss in Huntington's Disease Mice

(2014) Brett D Dufour et al.   MOLECULAR THERAPY

Adeno-Associated Virus Vectors and Neurological Gene Therapy

(2014) David S. Ojala et al.   NEUROSCIENTIST

Gene Therapy for the Nervous System: Challenges and New Strategies

(2014) Casey A. Maguire et al.   Neurotherapeutics

Widespread spinal cord transduction by intrathecal injection of rAAV delivers efficacious RNAi therapy for amyotrophic lateral sclerosis

(2013) Hongyan Wang et al.   HUMAN MOLECULAR GENETICS

Recombinant AAV as a Platform for Translating the Therapeutic Potential of RNA Interference

(2013) Florie Borel et al.   MOLECULAR THERAPY

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

(2013) Leszek Lisowski et al.   NATURE

A bright monomeric green fluorescent protein derived from Branchiostoma lanceolatum

(2013) Nathan C Shaner et al.   NATURE METHODS

Functional labeling of neurons and their projections using the synthetic activity–dependent promoter E-SARE

(2013) Takashi Kawashima et al.   NATURE METHODS

Permanent Genetic Access to Transiently Active Neurons via TRAP: Targeted Recombination in Active Populations

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Capsid Serotype and Timing of Injection Determines AAV Transduction in the Neonatal Mice Brain

(2013) Paramita Chakrabarty et al.   PLoS One

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

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(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Neutralizing antibodies against adeno-associated viruses in inflammatory bowel disease patients: Implications for gene therapy

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Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates

(2011) Steven J Gray et al.   MOLECULAR THERAPY

Engineering Liver-detargeted AAV9 Vectors for Cardiac and Musculoskeletal Gene Transfer

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

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

(2011) Adam K Bevan et al.   MOLECULAR THERAPY

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

(2010) Daniel Gaudet et al.   ATHEROSCLEROSIS SUPPLEMENTS

Heart-targeted adeno-associated viral vectors selected by in vivo biopanning of a random viral display peptide library

(2010) Y Ying et al.   GENE THERAPY

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

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

(2010) Jun Xie et al.   MOLECULAR THERAPY

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

(2010) F. Sonntag et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Monosynaptic circuit tracing in vivo through Cre-dependent targeting and complementation of modified rabies virus

(2010) N. R. Wall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

(2009) E Ayuso et al.   GENE THERAPY

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Directed evolution of adeno-associated virus to an infectious respiratory virus

(2009) K. J. D. A. Excoffon et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Improved Splicing of Adeno-Associated Viral (AAV) Capsid Protein-Supplying Pre-mRNAs Leads to Increased Recombinant AAV Vector Production

(2008) K. David Farris et al.   HUMAN GENE THERAPY

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

(2008) D. Grimm et al.   JOURNAL OF VIROLOGY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE