Burghes, Arthur H M

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

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

Spinal muscular atrophy: why do low levels of survival motor neuron protein make motor neurons sick?

Burghes, A. H. M., & Beattie, C. E. (2009) · Nature Reviews Neuroscience

Neurogenetic and Muscular Disorders ResearchAmyotrophic Lateral Sclerosis Research

Cited by 723

RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN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

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

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

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