Likhite, Shibi

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

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

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

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