AAV9 Vector: a Novel modality in gene therapy for spinal muscular atrophy
Author:
Funder
The Blazer Foundation
Publisher
Springer Science and Business Media LLC
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.nature.com/articles/s41434-019-0085-4.pdf
Reference76 articles.
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3. Farrar MA, Vucic S, Lin CS-Y, Park SB, Johnston HM, du Sart D, et al. Dysfunction of axonal membrane conductances in adolescents and young adults with spinal muscular atrophy. Brain 2011;134:3185–97.
4. Finkel RS, McDermott MP, Kaufmann P, Darras BT, Chung WK, Sproule DM, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 2014;83:810–7.
5. Sugarman EA, Nagan N, Zhu H, Akmaev VR, Zhou Z, Rohlfs EM, et al. Pan-ethnic carrier screening and prenatal diagnosis for spinal muscular atrophy: clinical laboratory analysis of >72,400 specimens. Eur J Hum Genet. 2012;20:27–32.
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