Article

Detection of Pre-Existing Immunity to Bacterial Cas9 Proteins in People with Cystic Fibrosis

2025-03-22

Abstract excerpt

Cystic fibrosis (CF) is caused by homozygous mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene, resulting in multi-organ dysfunction and decreased lifespan and quality of life. A durable cure for CF will likely require a gene therapy approach to correct CFTR. Rapid advancements in genome editing technologies such as CRISPR/Cas9 have already resulted in successful FDA approval for...

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Literature Corpus work: 3898bb43-89e5-5ea2-b12f-b3d9266c2410
DOI: 10.1101/2025.03.20.644396

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