Gene Therapy for Cystic Fibrosis: Recent Advances and Future Prospects.

ABSTRACT: Gene replacement therapies are novel therapeutic approaches that seek to tackle hereditary diseases caused by a congenital deficiency in a particular gene, when a functional copy of a gene can be delivered to the cells and tissues using various delivery systems. To do this, viral particles carrying a functional copy of the gene of interest and various nonviral gene delivery systems, including liposomes, nanoparticles, etc., can be used. In this review, we discuss the state of current knowledge regarding the molecular mechanisms and types of genetic mutations that lead to cystic fibrosis and highlight recent developments in gene therapy that can be leveraged to correct these mutations and to restore the physiological function of the carrier protein transporting sodium and chlorine ions in t

SUBMITTER: Lomunova MA 

PROVIDER: S-EPMC10395777 | biostudies-literature | 2023 Apr-Jun

REPOSITORIES: biostudies-literature