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With the ability to make precise edits ranging from single-nucleotide changes to large chromosomal rearrangements, CRISPR offers new possibilities for treating inherited blood diseases ( BCL11A gene, a transcriptional repressor of -globin and fetal hemoglobin (HbF) production

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In a model of amyotrophic lateral sclerosis (ALS) caused by C9orf72 gene mutations, the pathogenic poly-dipeptide GA50 directly binds to and inhibits SQOR, leading to excessive NLRP3 inflammasome activation in microglia and consequent mitochondrial dysfunction ( A growing body of preclinical work supports this strategy: boosting SQOR activity or reducing H 2 S can protect the nervous system

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