Towards personalized medicine for amyotrophic lateral sclerosis

Trends Endocrinol Metab. 2021 Jul 26:S1043-2760(21)00155-7. doi: 10.1016/j.tem.2021.07.002. Online ahead of print.ABSTRACTMohassel et al. provide unprecedented dichotomy of consequences on sphingolipid biosynthesis between pathogenic variants in the SPTLC1 gene, responsible for either amyotrophic lateral sclerosis (ALS) or hereditary sensory and autonomic neuropathy type 1 (HSAN1). Normalization of sphingolipid levels by siRNA selectively targeting the ALS mutant allele mRNA sheds light on new therapeutic approaches.PMID:34325980 | DOI:10.1016/j.tem.2021.07.002

Source: Trends in Endocrinology and Metabolism: TEM - July 30, 2021 Category: Endocrinology Authors: Julien Cassereau Philippe Corcia Pascal Reynier Source Type: research