Genome Editing for CNS Disorders.

Front Neurosci · 2020 · PMC7642486 · PMID 33192264

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highly significantno p-value reported

Zeitler and coworkers recently generated a second-generation ZF-KRAB that preferentially recognizes pathogenic CAG repeats, and demonstrated highly significant mHTT suppression with wild-type allele preservation in patient derived-iPSCs ( Zeitler et al., 2019 ).

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