Article

CRISPR-Cas9 Mediated Gene Correction of CFTR Mutations in Cystic Fibrosis: Evaluating Efficacy, Safety, and Long-Term Outcomes in Patient-Derived Lung Organoids

2023-05-19

Abstract excerpt

Cystic fibrosis (CF) is a genetic disorder caused by mutations in the *CFTR* gene, leading to severe respiratory and digestive problems. Current therapies including AAV gene therapy, small molecule modifiers, and RNA-based therapies only partially restore *CFTR* function, leaving a need for more effective therapies The aim of this review is to address CRISPR-1. Cas9-mediated genetic correction in patient derived l...

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Cystic Fibrosis Research Advances

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Literature Corpus work: 9cd3200d-2a0a-5eff-bc88-fe48df83a643
DOI: 10.70470/shifaa/2023/005

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