CRISPR deletion of the C9ORF72 promoter in ALS/FTD patient motor neurons abolishes production of dipeptide repeat proteins and rescues neurodegeneration

ACTA NEUROPATHOLOGICA

卷 140, 期 1, 页码 81-84

SPRINGER

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Clinical Neurology Neurosciences Pathology

1. NIH [R37NS057553, R01NS101986, R01NS093097, R21NS112766]
2. MDA Foundation
3. Target ALS Foundation
4. Frick Foundation for ALS Research
5. Alzheimer's Association [2016-NIRG-396129]
6. ALS Association
7. National Key R&D Program of China [2017YFC1310300]