Treatment of Cystic Fibrosis: From Gene- to Cell-Based Therapies.

ABSTRACT: Prognosis of patients with cystic fibrosis (CF) varies extensively despite recent advances in targeted therapies that improve CF transmembrane conductance regulator (CFTR) function. Despite being a multi-organ disease, extensive lung tissue destruction remains the major cause of morbidity and mortality. Progress towards a curative treatment strategy that implements a CFTR gene addition-technology to the patients' lungs has been slow and not yet developed beyond clinical trials. Improved delivery vectors are needed to overcome the body's defense system and ensure an efficient and consistent clinical response before gene therapy is suitable for clinical care. Cell-based therapy-which relies on functional modification of allogenic or autologous cells ex vivo, prior to transplant

SUBMITTER: Allan KM 

PROVIDER: S-EPMC8007963 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature