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Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons

Hedayati SS

[DOI] [PMC free article] [PubMed] [Google Scholar] 256.Slemmer JE

Priglinger et al

Unlike a long-acting compound that commits a subject to a week-long effect, the short 5-Amino-1MQ half life allows for more dynamic adjustments to the protocol