Kaspar, Brian K.

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

Mendell, J. R., Al-Zaidy, S., Shell, R., Arnold, W. D., Rodino-Klapac, L. R., Prior, T. W., Lowes, L., Alfano, L., Berry, K., Church, K., et al. (2017) · New England Journal of Medicine

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 2,381

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

Foust, K. D., Nurre, E., Montgomery, C. L., Hernandez, A., Chan, C. M., & Kaspar, B. K. (2009) · Nature Biotechnology

Virus-based gene therapy researchBarrier Structure and Function Studies

Cited by 1,456

Retrograde Viral Delivery of IGF-1 Prolongs Survival in a Mouse ALS Model

Kaspar, B. K., Lladó, J., Sherkat, N., Rothstein, J. D., & Gage, F. H. (2003) · Science

Amyotrophic Lateral Sclerosis ResearchNeurogenetic and Muscular Disorders Research

Cited by 872

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

Haidet-Phillips, A. M., Hester, M. E., Miranda, C. J., Meyer, K., Braun, L., Frakes, A., Song, S., Likhite, S., Murtha, M. J., Foust, K. D., et al. (2011) · Nature Biotechnology

Amyotrophic Lateral Sclerosis ResearchAlzheimer's disease research and treatments

Cited by 782

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Foust, K. D., Wang, X., McGovern, V. L., Braun, L., Bevan, A. K., Haidet, A. M., Le, T. T., Morales, P. R., Rich, M. M., Burghes, A. H. M., et al. (2010) · Nature Biotechnology

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 688

Microglia Induce Motor Neuron Death via the Classical NF-κB Pathway in Amyotrophic Lateral Sclerosis

Frakes, A. E., Ferraiuolo, L., Haidet-Phillips, A. M., Schmelzer, L., Braun, L., Miranda, C. J., Ladner, K. J., Bevan, A. K., Foust, K. D., Godbout, J. P., et al. (2014) · Neuron

Amyotrophic Lateral Sclerosis ResearchNeuroinflammation and Neurodegeneration Mechanisms

Cited by 638

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

Maheshri, N., Koerber, J. T., Kaspar, B. K., & Schaffer, D. V. (2006) · Nature Biotechnology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 538

A role for glia in the progression of Rett’s syndrome

Lioy, D. T., Garg, S. K., Monaghan, C. E., Raber, J., Foust, K. D., Kaspar, B. K., Hirrlinger, P. G., Kirchhoff, F., Bissonnette, J. M., Ballas, N., et al. (2011) · Nature

Genetics and Neurodevelopmental DisordersNeuroscience and Neuropharmacology Research

Cited by 496

The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy

Webster, C. P., Smith, E. F., Bauer, C. S., Moller, A., Hautbergue, G. M., Ferraiuolo, L., Myszczynska, M. A., Higginbottom, A., Walsh, M. J., Whitworth, A. J., et al. (2016) · The EMBO Journal

Amyotrophic Lateral Sclerosis ResearchAutophagy in Disease and Therapy

Cited by 404

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

Meyer, K., Ferraiuolo, L., Miranda, C. J., Likhite, S., McElroy, S., Renusch, S., Ditsworth, D., Lagier-Tourenne, C., Smith, R. A., Ravits, J., et al. (2014) · Proceedings of the National Academy of Sciences

Amyotrophic Lateral Sclerosis ResearchPluripotent Stem Cells Research

Cited by 361

Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

Bevan, A. K., Duque, S., Foust, K. D., Morales, P. R., Braun, L., Schmelzer, L., Chan, C. M., McCrate, M., Chicoine, L. G., Coley, B. D., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 337

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

Samaranch, L., Salegio, E. A., San Sebastian, W., Kells, A. P., Foust, K. D., Bringas, J. R., Lamarre, C., Forsayeth, J., Kaspar, B. K., & Bankiewicz, K. S. (2012) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 299

Improving Single Injection CSF Delivery of AAV9-mediated Gene Therapy for SMA: A Dose–response Study in Mice and Nonhuman Primates

Meyer, K., Ferraiuolo, L., Schmelzer, L., Braun, L., McGovern, V., Likhite, S., Michels, O., Govoni, A., Fitzgerald, J., Morales, P., et al. (2015) · Molecular Therapy

Neurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

Cited by 286

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

Rodino‐Klapac, L. R., Haidet, A. M., Kota, J., Handy, C., Kaspar, B. K., & Mendell, J. R. (2009) · Muscle & Nerve

Muscle Physiology and DisordersNeurogenetic and Muscular Disorders Research

Cited by 194

AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort

Al-Zaidy, S. A., Kolb, S. J., Lowes, L., Alfano, L. N., Shell, R., Church, K. R., Nagendran, S., Sproule, D. M., Feltner, D. E., Wells, C., et al. (2019) · Journal of Neuromuscular Diseases

Neurogenetic and Muscular Disorders ResearchLysosomal Storage Disorders Research

Cited by 185

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

Hester, M. E., Murtha, M. J., Song, S., Rao, M., Miranda, C. J., Meyer, K., Tian, J., Boulting, G., Schaffer, D. V., Zhu, M. X., et al. (2011) · Molecular Therapy

Pluripotent Stem Cells ResearchNeurogenesis and neuroplasticity mechanisms

Cited by 183

PEG conjugation moderately protects adeno‐associated viral vectors against antibody neutralization

Lee, G. K., Maheshri, N., Kaspar, B., & Schaffer, D. V. (2005) · Biotechnology and Bioengineering

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 169

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

Kaspar, B. K., Roth, D. M., Chin Lai, N., Drumm, J. D., Erickson, D. A., McKirnan, M. D., & Hammond, H. K. (2005) · The Journal of Gene Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 59

Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease

Cain, J. T., Likhite, S., White, K. A., Timm, D. J., Davis, S. S., Johnson, T. B., Dennys-Rivers, C. N., Rinaldi, F., Motti, D., Corcoran, S., et al. (2019) · Molecular Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 53

rAAV Gene Therapy in a Canavan’s Disease Mouse Model Reveals Immune Impairments and an Extended Pathology Beyond the Central Nervous System

Ahmed, S. S., Schattgen, S. A., Frakes, A. E., Sikoglu, E. M., Su, Q., Li, J., Hampton, T. G., Denninger, A. R., Kirschner, D. A., Kaspar, B., et al. (2016) · Molecular Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 24

Voluntary wheel running with and without follistatin overexpression improves NMJ transmission but not motor unit loss in late life of C57BL/6J mice

Chugh, D., Iyer, C. C., Bobbili, P., Blatnik, A. J., Kaspar, B. K., Meyer, K., Burghes, A. H., Clark, B. C., & Arnold, W. D. (2021) · Neurobiology of Aging

Muscle Physiology and DisordersNutrition and Health in Aging

Cited by 17