Retrograde Gene Delivery to Hypoglossal Motoneurons Using Adeno-Associated Virus Serotype 9

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

(2011) T Federici et al.   GENE THERAPY

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

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

Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

(2011) Terence R. Flotte 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

Pompe disease gene therapy

(2011) B. J. Byrne et al.   HUMAN MOLECULAR GENETICS

Pompe disease: Design, methodology, and early findings from the Pompe Registry

(2011) Barry J. Byrne et al.   MOLECULAR GENETICS AND METABOLISM

AAV9: Over the Fence and Into the Woods …

(2011) John R Forsayeth et al.   MOLECULAR THERAPY

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

Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice

(2011) Kai Qiu et al.   MOLECULAR THERAPY

The phrenic motor nucleus in the adult mouse

(2010) K. Qiu et al.   EXPERIMENTAL NEUROLOGY

Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

(2010) Cathryn S Mah et al.   MOLECULAR THERAPY

Acute Progression of Neuromuscular Findings in Infantile Pompe Disease

(2010) T. Andrew Burrow et al.   PEDIATRIC NEUROLOGY

Alglucosidase alfa: Long term use in the treatment of patients with Pompe disease

(2010) Michael Beck   Therapeutics and Clinical Risk Management

Oropharyngeal Dysphagia in Infants and Children with Infantile Pompe Disease

(2009) Harrison N. Jones et al.   DYSPHAGIA

Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6

(2009) C Towne et al.   GENE THERAPY

Efficient Retrograde Transport of Adeno-Associated Virus Type 8 to Spinal Cord and Dorsal Root Ganglion After Vector Delivery in Muscle

(2009) Hui Zheng et al.   HUMAN GENE THERAPY

Temporal Neuropathologic and Behavioral Phenotype of 6neo/6neoPompe Disease Mice

(2009) Richard L. Sidman et al.   JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY

AAV9: a potential blood-brain barrier buster

(2009) Fredric P Manfredsson et al.   MOLECULAR THERAPY

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

(2009) Sandra Duque et al.   MOLECULAR THERAPY

Emerging principles and neural substrates underlying tonic sleep-state-dependent influences on respiratory motor activity

(2009) R. L. Horner   PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Neural deficits contribute to respiratory insufficiency in Pompe disease

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

Clinical gene therapy using recombinant adeno-associated virus vectors

(2008) C Mueller et al.   GENE THERAPY

Cervical prephrenic interneurons in the normal and lesioned spinal cord of the adult rat

(2008) Michael A. Lane et al.   JOURNAL OF COMPARATIVE NEUROLOGY

Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain

(2008) Cassia N Cearley et al.   MOLECULAR THERAPY

Efficient Retrograde Neuronal Transduction Utilizing Self-complementary AAV1

(2008) Edmund R Hollis II et al.   MOLECULAR THERAPY

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

(2008) Wuping Li et al.   MOLECULAR THERAPY

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

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

(2008) Kevin D Foust et al.   NATURE BIOTECHNOLOGY