Efficient Gene Editing at Major CFTR Mutation Loci
Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Nuclease-mediated precise gene editing (PGE) represents a promising therapy for CF, for which an efficient strategy that is free of viral vector, d...
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Materiálatiipa: | Girji |
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Elsevier,
2019-06-01T00:00:00Z.
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