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pro vyhledávání: '"Binh T. T. Chu"'
Autor:
Arthur H.M. Burghes, Caroline Hsieh, Jerry R. Mendell, Douglas M. Sproule, Binh T. T. Chu, Kevin D. Foust, Douglas E. Feltner, Wendy K. Chung, Francesco Muntoni, Mariacristina Scoto, Christopher R. Pierson, Miriam R Conces, Stephanie Perry, Gretchen Thomsen, Petra Kaufmann, Robert F. Hevner, Basam Barkho, Janet Do, Vicki L. McGovern
Publikováno v:
Nature Medicine. 27:1701-1711
Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec (formerly AVXS-101) is a gene therapy that restores SMN production via on
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ef6f0fd234d81724758ac7b79e4e9847
https://doi.org/10.1038/s41591-021-01483-7
https://doi.org/10.1038/s41591-021-01483-7
