Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy
By J. Mendell, S. Al-Zaidy, R. Shell, W. Arnold, L. Rodino-Klapac, T. Prior, L. Lowes, L. Alfano, K. Berry, K. Church and 16 more. New England Journal of Medicine, 2017
1,912 citations recorded by the source corpus.
Subfields: Cellular and Molecular Neuroscience, Genetics, Neurology
View Paper: https://www.semanticscholar.org/paper/777b8bd73c02e9143f3a2cf08b3e6e7c3db2e2f7
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In Genetics
Citations in the corpus
- Emerging Issues in AAV-Mediated In Vivo Gene Therapy (2017, background): “One solution to the issue could be to administer an immunosuppression regimen up front for all subjects, as recently performed for other diseases.(29,68) However, this is not an ideal solution to the problem because not all individuals may have an immune response to the vector, and the timing of imm”
- Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN (2018, neutral): “14,32 The timing of these abnormalities could have different prognostic implications.”
- Gene therapy comes of age (2018, neutral): “Spinal muscular atrophy AAV9-SMN (107) Nationwide Children's”