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

Acta Neuropathol. 2020 Jul;140(1):81-84. doi: 10.1007/s00401-020-02154-6. Epub 2020 Apr 7.

¹ Department of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.
² Department of Neurology, Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai, China.
³ Neuromuscular and Movement Disorders, Biogen, Cambridge, MA, 02142, USA.
⁴ Department of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA. fen-biao.gao@umassmed.edu.
⁵ Department of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA. sandra.almeida@umassmed.edu.

No abstract available

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