Brian K. Kaspar

Targeted retrograde gene delivery for neuronal protection.

Adeno-associated virus effectively mediates conditional gene modification in the brain.

The adult substantia nigra contains progenitor cells with neurogenic potential.

Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo.

A conditional deletion of the NR1 subunit of the NMDA receptor in adult spinal cord dorsal horn reduces NMDA currents and injury-induced pain.

Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model.

IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes.

Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus.

Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis.

Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis.

In vivo evidence for radial migration of neurons by long-distance somal translocation in the developing ferret visual cortex.

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors.

Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis.

Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene delivery vehicles.

Gene therapy for duchenne muscular dystrophy: expectations and challenges.

Gene-targeted therapies for the central nervous system.

Physical activity and neuroprotection in amyotrophic lateral sclerosis.

Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors.

Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity.

Mesenchymal stem cells as trojan horses for GDNF delivery in ALS.

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease.

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

Gene therapy and informed consent decision making: nursing research directions.

Two factor reprogramming of human neural stem cells into pluripotency.

AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS.

Over the barrier and through the blood: to CNS delivery we go.

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

Follistatin gene delivery enhances muscle growth and strength in nonhuman primates.

Acetylcholine negatively regulates development of the neuromuscular junction through distinct cellular mechanisms.

Could gene therapy be the future for muscular dystrophy?

TrkB and TrkC agonist antibodies improve function, electrophysiologic and pathologic features in Trembler J mice.

Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery.

Intramuscular AAV delivery of NT-3 alters synaptic transmission to motoneurons in adult rats.

AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice.

Secretin deficiency causes impairment in survival of neural progenitor cells in mice.

A role for glia in the progression of Rett's syndrome.

Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes.

Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.

Astrocytes from familial and sporadic ALS patients are toxic to motor neurons.

Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone.

mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice.

Transforming growth factor α transforms astrocytes to a growth-supportive phenotype after spinal cord injury.

Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates.

Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis.

Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling.

Gene therapy for muscular dystrophy: lessons learned and path forward.

Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound.

Gene delivery improvement for treating the lysosomal storage disorder metachromatic leukodystrophy.

Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy.

Neural stem cells as a therapeutic approach for amyotrophic lateral sclerosis.

Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model.

Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome.

Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease.

AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy.

Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS.

Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept.

Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis.

Making sense of pain: are pluripotent stem cell-derived sensory neurons a new tool for studying pain mechanisms?

A Phase I/IIa Follistatin Gene Therapy Trial for Becker Muscular Dystrophy.

Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA - a dose response study in mice and nonhuman primates.