Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems

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

Comparison of Adeno-Associated Viral Vector Serotypes for Spinal Cord and Motor Neuron Gene Delivery

(2011) Brooke R. Snyder et al.   HUMAN GENE THERAPY

Optimizing Promoters for Recombinant Adeno-Associated Virus-Mediated Gene Expression in the Peripheral and Central Nervous System Using Self-Complementary Vectors

(2011) Steven J. Gray et al.   HUMAN GENE THERAPY

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

Pleiotropic Genetic Syndromes with Developmental Abnormalities Associated with Obesity

(2011) E. Kousta et al.   JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

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

A Cell-penetrating Peptide Suppresses Inflammation by Inhibiting NF-κB Signaling

(2011) Yu Fu Wang et al.   MOLECULAR THERAPY

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

(2011) Nagesh Pulicherla et al.   MOLECULAR THERAPY

Stable Human FIX Expression After 0.9G Intrauterine Gene Transfer of Self-complementary Adeno-associated Viral Vector 5 and 8 in Macaques

(2011) Citra NZ Mattar 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

Correction of Neurological Disease of Mucopolysaccharidosis IIIB in Adult Mice by rAAV9 Trans-Blood–Brain Barrier Gene Delivery

(2011) Haiyan Fu et al.   MOLECULAR THERAPY

In uterogene transfer to the mouse nervous system: Figure 1

(2010) Ahad A. Rahim et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

(2010) A Geisler 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

Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer

(2010) Chantelle McIntyre et al.   JOURNAL OF GENE MEDICINE

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

(2010) Jun Xie 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

Prion-like transmission of protein aggregates in neurodegenerative diseases

(2010) Patrik Brundin et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Seeding induced by α-synuclein oligomers provides evidence for spreading of α-synuclein pathology

(2009) Karin M. Danzer et al.   JOURNAL OF NEUROCHEMISTRY

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

(2009) Robert F. Wynn et al.   JOURNAL OF PEDIATRICS

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

(2009) Sandra Duque et al.   MOLECULAR THERAPY

AAV2/8-mediated Correction of OTC Deficiency Is Robust in Adult but Not Neonatal Spfash Mice

(2009) Sharon C Cunningham et al.   MOLECULAR THERAPY

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

(2009) Aravind Asokan et al.   NATURE BIOTECHNOLOGY

Prions hijack tunnelling nanotubes for intercellular spread

(2009) Karine Gousset et al.   NATURE CELL BIOLOGY

Inclusion formation and neuronal cell death through neuron-to-neuron transmission of  -synuclein

(2009) P. Desplats et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

First trimester embryo-fetoscopic and ultrasound-guided fetal blood sampling for ex vivo viral transduction of cultured human fetal mesenchymal stem cells

(2008) Jerry Chan et al.   HUMAN REPRODUCTION

Assembly-dependent endocytosis and clearance of extracellular a-synuclein

(2008) He-Jin Lee et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease

(2008) Ulrich Matzner et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Abetalipoproteinemia: two case reports and literature review

(2008) Rola Zamel et al.   Orphanet Journal of Rare Diseases