Systemic Delivery of Tyrosine-Mutant AAV Vectors Results in Robust Transduction of Neurons in Adult Mice

Tyrosine Cross-Linking Reveals Interfacial Dynamics in Adeno-Associated Viral Capsids during Infection

(2012) Eric D. Horowitz et al.   ACS Chemical Biology

Properties of the Adeno-Associated Virus Assembly-Activating Protein

(2012) M. Naumer et al.   JOURNAL OF VIROLOGY

Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2

(2012) Yu Miyazaki et al.   NATURE MEDICINE

Adeno-associated virus (AAV) gene therapy for neurological disease

(2012) Marc S. Weinberg et al.   NEUROPHARMACOLOGY

Limitations of Encapsidation of Recombinant Self-Complementary Adeno-Associated Viral Genomes in Different Serotype Capsids and Their Quantitation

(2012) Yuan Wang et al.   Human Gene Therapy Methods

Restricted Transgene Expression in the Brain with Cell-Type Specific Neuronal Promoters

(2012) Aurélie Delzor et al.   Human Gene Therapy Methods

Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system

(2011) Ahad A. Rahim et al.   FASEB JOURNAL

Enhanced gene delivery to the neonatal retina through systemic administration of tyrosine-mutated AAV9

(2011) D Dalkara et al.   GENE THERAPY

Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs

(2011) T Federici et al.   GENE THERAPY

Development of optimized AAV3 serotype vectors: mechanism of high-efficiency transduction of human liver cancer cells

(2011) B Cheng et al.   GENE THERAPY

Decreasing Disease Severity in Symptomatic, Smn−/−;SMN2+/+, Spinal Muscular Atrophy Mice Following scAAV9-SMN Delivery

(2011) Jacqueline J. Glascock et al.   HUMAN GENE THERAPY

Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

(2011) Yadong Zhang et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates

(2011) Lluis Samaranch et al.   HUMAN GENE THERAPY

Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis

(2011) Cristy A. Ku et al.   HUMAN MOLECULAR GENETICS

Genetic therapy for the nervous system

(2011) W. J. Bowers et al.   HUMAN MOLECULAR GENETICS

An efficient method for the long-term and specific expression of exogenous cDNAs in cultured Purkinje neurons

(2011) Wolfgang Wagner et al.   JOURNAL OF NEUROSCIENCE METHODS

Neuroinflammation in amyotrophic lateral sclerosis: role of glial activation in motor neuron disease

(2011) Thomas Philips et al.   LANCET NEUROLOGY

AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

(2011) Peter A LeWitt et al.   LANCET NEUROLOGY

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

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

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

(2011) Hongwei Zhang et al.   MOLECULAR THERAPY

High-Efficiency Transduction of Fibroblasts and Mesenchymal Stem Cells by Tyrosine-Mutant AAV2 Vectors for Their Potential Use in Cellular Therapy

(2010) Mengxin Li et al.   HUMAN GENE THERAPY

Adeno-Associated Virus Serotype 6 Capsid Tyrosine-to-Phenylalanine Mutations Improve Gene Transfer to Skeletal Muscle

(2010) Chunping Qiao et al.   HUMAN GENE THERAPY

High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

(2010) David M Markusic et al.   MOLECULAR THERAPY

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

A Phase I Study of Aromatic L-Amino Acid Decarboxylase Gene Therapy for Parkinson's Disease

(2010) Shin-ichi Muramatsu et al.   MOLECULAR THERAPY

Novel Properties of Tyrosine-mutant AAV2 Vectors in the Mouse Retina

(2010) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Astrocytes and therapeutics for Parkinson’s disease

(2010) Phillip M. Rappold et al.   Neurotherapeutics

Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

(2009) C. W. Christine et al.   NEUROLOGY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY

High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors

(2008) Hilda Petrs-Silva et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

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

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA