Brian K. Kaspar

Publications

• Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA - a Dose Response Study in Mice and Nonhuman Primates
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2014  |   Pubmed ID: 25358252
• A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2014  |   Pubmed ID: 25322757
• Making Sense of Pain: Are Pluripotent Stem Cell-derived Sensory Neurons a New Tool for Studying Pain Mechanisms?
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Aug, 2014  |   Pubmed ID: 25082088
• Microglia Induce Motor Neuron Death Via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis
  Neuron. Mar, 2014  |   Pubmed ID: 24607225
• Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept
  Annals of Clinical and Translational Neurology. Jan, 2014  |   Pubmed ID: 24511555
• Direct Conversion of Patient Fibroblasts Demonstrates Non-cell Autonomous Toxicity of Astrocytes to Motor Neurons in Familial and Sporadic ALS
  Proceedings of the National Academy of Sciences of the United States of America. Jan, 2014  |   Pubmed ID: 24379375
• AAV1.NT-3 Gene Therapy for Charcot-marie-tooth Neuropathy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Mar, 2014  |   Pubmed ID: 24162799
• Active and Passive Immunization Strategies Based on the SDPM1 Peptide Demonstrate Pre-clinical Efficacy in the APPswePSEN1dE9 Mouse Model for Alzheimer's Disease
  Neurobiology of Disease. Feb, 2014  |   Pubmed ID: 24021662
• Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Dec, 2013  |   Pubmed ID: 24008656
• Systemic Delivery of MeCP2 Rescues Behavioral and Cellular Deficits in Female Mouse Models of Rett Syndrome
  The Journal of Neuroscience : the Official Journal of the Society for Neuroscience. Aug, 2013  |   Pubmed ID: 23966684
• Micro-dystrophin and Follistatin Co-delivery Restores Muscle Function in Aged DMD Model
  Human Molecular Genetics. Dec, 2013  |   Pubmed ID: 23863459
• Neural Stem Cells As a Therapeutic Approach for Amyotrophic Lateral Sclerosis
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Mar, 2013  |   Pubmed ID: 23449106
• Transplantation of Gene-corrected Motor Neurons As a Therapeutic Strategy for Spinal Muscular Atrophy
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Mar, 2013  |   Pubmed ID: 23449105
• Gene Delivery Improvement for Treating the Lysosomal Storage Disorder Metachromatic Leukodystrophy
  Human Gene Therapy. Aug, 2012  |   Pubmed ID: 22900577
• Targeted Delivery of Self-complementary Adeno-associated Virus Serotype 9 to the Brain, Using Magnetic Resonance Imaging-guided Focused Ultrasound
  Human Gene Therapy. Nov, 2012  |   Pubmed ID: 22838844
• Gene Therapy for Muscular Dystrophy: Lessons Learned and Path Forward
  Neuroscience Letters. Oct, 2012  |   Pubmed ID: 22609847
• Aging Brain Microenvironment Decreases Hippocampal Neurogenesis Through Wnt-mediated Survivin Signaling
  Aging Cell. Jun, 2012  |   Pubmed ID: 22404871
• Knee Extensor Strength Exhibits Potential to Predict Function in Sporadic Inclusion-body Myositis
  Muscle & Nerve. Feb, 2012  |   Pubmed ID: 22246869
• Adeno-associated Virus Serotype 9 Transduction in the Central Nervous System of Nonhuman Primates
  Human Gene Therapy. Apr, 2012  |   Pubmed ID: 22201473
• Transforming Growth Factor α Transforms Astrocytes to a Growth-supportive Phenotype After Spinal Cord Injury
  The Journal of Neuroscience : the Official Journal of the Society for Neuroscience. Oct, 2011  |   Pubmed ID: 22016551
• Mdx(⁵cv) Mice Manifest More Severe Muscle Dysfunction and Diaphragm Force Deficits Than Do Mdx Mice
  The American Journal of Pathology. Nov, 2011  |   Pubmed ID: 21893021
• Disease Progression in a Mouse Model of Amyotrophic Lateral Sclerosis: the Influence of Chronic Stress and Corticosterone
  FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology. Dec, 2011  |   Pubmed ID: 21876068
• Astrocytes from Familial and Sporadic ALS Patients Are Toxic to Motor Neurons
  Nature Biotechnology. Sep, 2011  |   Pubmed ID: 21832997
• Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Nov, 2011  |   Pubmed ID: 21811247
• Rapid and Efficient Generation of Functional Motor Neurons from Human Pluripotent Stem Cells Using Gene Delivered Transcription Factor Codes
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Oct, 2011  |   Pubmed ID: 21772256
• A Role for Glia in the Progression of Rett's Syndrome
  Nature. Jul, 2011  |   Pubmed ID: 21716289
• Secretin Deficiency Causes Impairment in Survival of Neural Progenitor Cells in Mice
  Human Molecular Genetics. Mar, 2011  |   Pubmed ID: 21159798
• AAV4-mediated Expression of IGF-1 and VEGF Within Cellular Components of the Ventricular System Improves Survival Outcome in Familial ALS Mice
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Dec, 2010  |   Pubmed ID: 20859261
• Intramuscular AAV Delivery of NT-3 Alters Synaptic Transmission to Motoneurons in Adult Rats
  The European Journal of Neuroscience. Sep, 2010  |   Pubmed ID: 20849530
• Early Heart Failure in the SMNDelta7 Model of Spinal Muscular Atrophy and Correction by Postnatal ScAAV9-SMN Delivery
  Human Molecular Genetics. Oct, 2010  |   Pubmed ID: 20639395
• TrkB and TrkC Agonist Antibodies Improve Function, Electrophysiologic and Pathologic Features in Trembler J Mice
  Experimental Neurology. Aug, 2010  |   Pubmed ID: 20553714
• Could Gene Therapy Be the Future for Muscular Dystrophy?
  Therapy. May, 2010  |   Pubmed ID: 20543898
• Acetylcholine Negatively Regulates Development of the Neuromuscular Junction Through Distinct Cellular Mechanisms
  Proceedings of the National Academy of Sciences of the United States of America. Jun, 2010  |   Pubmed ID: 20498043
• Rescue of the Spinal Muscular Atrophy Phenotype in a Mouse Model by Early Postnatal Delivery of SMN
  Nature Biotechnology. Mar, 2010  |   Pubmed ID: 20190738
• Follistatin Gene Delivery Enhances Muscle Growth and Strength in Nonhuman Primates
  Science Translational Medicine. Nov, 2009  |   Pubmed ID: 20368179
• Over the Barrier and Through the Blood: to CNS Delivery We Go
  Cell Cycle (Georgetown, Tex.). Dec, 2009  |   Pubmed ID: 19949299
• AAV As a Gene Transfer Vector for the Treatment of Neurological Disorders: Novel Treatment Thoughts for ALS
  Current Gene Therapy. Oct, 2009  |   Pubmed ID: 19860657
• Two Factor Reprogramming of Human Neural Stem Cells into Pluripotency
  PloS One. 2009  |   Pubmed ID: 19763260
• Gene Therapy and Informed Consent Decision Making: Nursing Research Directions
  Biological Research for Nursing. Jul, 2009  |   Pubmed ID: 19398415
• Directed Evolution of Adeno-associated Virus to an Infectious Respiratory Virus
  Proceedings of the National Academy of Sciences of the United States of America. Mar, 2009  |   Pubmed ID: 19237554
• Inhibition of Myostatin with Emphasis on Follistatin As a Therapy for Muscle Disease
  Muscle & Nerve. Mar, 2009  |   Pubmed ID: 19208403
• Intravascular AAV9 Preferentially Targets Neonatal Neurons and Adult Astrocytes
  Nature Biotechnology. Jan, 2009  |   Pubmed ID: 19098898
• Mesenchymal Stem Cells As Trojan Horses for GDNF Delivery in ALS
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Dec, 2008  |   Pubmed ID: 19023270
• Delivery of AAV-IGF-1 to the CNS Extends Survival in ALS Mice Through Modification of Aberrant Glial Cell Activity
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Jun, 2008  |   Pubmed ID: 18388910
• Long-term Enhancement of Skeletal Muscle Mass and Strength by Single Gene Administration of Myostatin Inhibitors
  Proceedings of the National Academy of Sciences of the United States of America. Mar, 2008  |   Pubmed ID: 18334646
• Physical Activity and Neuroprotection in Amyotrophic Lateral Sclerosis
  Neuromolecular Medicine. 2008  |   Pubmed ID: 18286388
• Gene-targeted Therapies for the Central Nervous System
  Archives of Neurology. Apr, 2008  |   Pubmed ID: 18268183
• Gene Therapy for Duchenne Muscular Dystrophy: Expectations and Challenges
  Archives of Neurology. Sep, 2007  |   Pubmed ID: 17846262
• Construction of Diverse Adeno-associated Viral Libraries for Directed Evolution of Enhanced Gene Delivery Vehicles
  Nature Protocols. 2006  |   Pubmed ID: 17406299
• Gene Transfer Demonstrates That Muscle is Not a Primary Target for Non-cell-autonomous Toxicity in Familial Amyotrophic Lateral Sclerosis
  Proceedings of the National Academy of Sciences of the United States of America. Dec, 2006  |   Pubmed ID: 17164329
• Directed Evolution of Adeno-associated Virus Yields Enhanced Gene Delivery Vectors
  Nature Biotechnology. Feb, 2006  |   Pubmed ID: 16429148
• In Vivo Evidence for Radial Migration of Neurons by Long-distance Somal Translocation in the Developing Ferret Visual Cortex
  Cerebral Cortex (New York, N.Y. : 1991). Nov, 2006  |   Pubmed ID: 16357334
• Synergy of Insulin-like Growth Factor-1 and Exercise in Amyotrophic Lateral Sclerosis
  Annals of Neurology. May, 2005  |   Pubmed ID: 15852403
• Virus-delivered Small RNA Silencing Sustains Strength in Amyotrophic Lateral Sclerosis
  Annals of Neurology. May, 2005  |   Pubmed ID: 15852369
• Myocardial Gene Transfer and Long-term Expression Following Intracoronary Delivery of Adeno-associated Virus
  The Journal of Gene Medicine. Mar, 2005  |   Pubmed ID: 15515115
• IGF-I Instructs Multipotent Adult Neural Progenitor Cells to Become Oligodendrocytes
  The Journal of Cell Biology. Jan, 2004  |   Pubmed ID: 14709544
• Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model
  Science (New York, N.Y.). Aug, 2003  |   Pubmed ID: 12907804
• A Conditional Deletion of the NR1 Subunit of the NMDA Receptor in Adult Spinal Cord Dorsal Horn Reduces NMDA Currents and Injury-induced Pain
  The Journal of Neuroscience : the Official Journal of the Society for Neuroscience. Jun, 2003  |   Pubmed ID: 12832526
• Sonic Hedgehog Regulates Adult Neural Progenitor Proliferation in Vitro and in Vivo
  Nature Neuroscience. Jan, 2003  |   Pubmed ID: 12469128
• The Adult Substantia Nigra Contains Progenitor Cells with Neurogenic Potential
  The Journal of Neuroscience : the Official Journal of the Society for Neuroscience. Aug, 2002  |   Pubmed ID: 12151543
• Adeno-associated Virus Effectively Mediates Conditional Gene Modification in the Brain
  Proceedings of the National Academy of Sciences of the United States of America. Feb, 2002  |   Pubmed ID: 11842206
• Targeted Retrograde Gene Delivery for Neuronal Protection
  Molecular Therapy : the Journal of the American Society of Gene Therapy. Jan, 2002  |   Pubmed ID: 11786045