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Genetic conversion of an SMN2 gene to SMN1: a novel approach to the treatment of spinal muscular atrophy |
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| Authors: | DiMatteo Darlise Callahan Stephanie Kmiec Eric B |
| Institution: | Department of Biological Sciences, University of Delaware, Newark, Delaware 19716, USA. |
| Abstract: | |
| Keywords: | Single-stranded oligonucleotides Gene alteration SMA |
| 本文献已被 ScienceDirect PubMed 等数据库收录！ | |