FIELD: biotechnology.
SUBSTANCE: invention relates to a composition, as well as to a method for editing a PMP22 gene having a target sequence in a cell using said composition. Said composition contains a Cas9 protein or a nucleic acid encoding a Cas9 protein and a guide RNA comprising crRNA and tracrRNA, or a nucleic acid encoding a guide RNA, where the Cas9 protein is derived from Streptococcus pyogenes or Campylobacter jejuni and where the target sequence is located in the TATA box of the PMP22 gene.
EFFECT: invention is effective for treating Charcot — Marie — Toute disease type 1A.
21 cl, 16 dwg, 9 tbl, 6 ex
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