Article

Systemic in utero gene editing as a treatment for cystic fibrosis

2024-09-08

Abstract excerpt

In utero gene editing has the potential to modify disease causing genes in multiple developing tissues before birth, possibly allowing for normal organ development, disease improvement, and conceivably, cure. In cystic fibrosis (CF), a disease that arises from mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene, there are signs of multiorgan disease affecting the function of the res...

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Literature Corpus work: 5f441c6b-8b8f-5395-8ea8-929ab19e1783
DOI: 10.1101/2024.09.04.611031

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