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TARGETED GENOMIC INTEGRATION TO RESTORE NEUROFIBROMIN CODING SEQUENCE IN NEUROFIBROMATOSIS TYPE 1 (NF1)
TARGETED GENOMIC INTEGRATION TO RESTORE NEUROFIBROMIN CODING SEQUENCE IN NEUROFIBROMATOSIS TYPE 1 (NF1)
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机译:针对神经纤维瘤病症1(NF1)的恢复神经纤维素编码序列的靶向基因组酰化
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摘要
Disclosed herein are systems and methods for the treatment of NF1 in a subject. The CRISPR/Cas-based genome editing systems may include a polynucleotide sequence encoding a guide RNA (gRNA) targeting a fragment of a mutant NF1 gene, a polynucleotide sequence encoding a Cas protein or a fusion protein comprising the Cas protein, and a polynucleotide sequence encoding a donor sequence comprising a fragment of a wild-type NF1 gene.
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