[Gene therapy in mucoviscidosis]

Area of Science:

• * Molecular Biology
• * Genetics
• * Respiratory Medicine

Context:

• * Cystic fibrosis (CF) is a genetic disorder characterized by defective transepithelial ion transport.
• * Respiratory manifestations significantly impact patient morbidity and mortality.
• * Current treatments focus on managing symptoms, highlighting the need for novel therapeutic strategies.

Purpose:

• * To evaluate the efficacy and safety of gene therapy for treating cystic fibrosis.
• * To explore various gene delivery systems for CFTR gene transfer.
• * To assess the potential of gene therapy in correcting CFTR protein function in patients.

Summary:

• * Several gene delivery vectors, including recombinant adenovirus (Ad CFTR) and liposomes with CFTR DNA, have been tested in vitro and in vivo.
• * Gene transfer and expression of the CFTR gene were confirmed in CF patients following nasal and pulmonary administration.
• * Transient correction of defective chloride transport was observed in the nasal mucosa of some patients, demonstrating proof-of-concept.

Impact:

• * Gene therapy presents a viable alternative for addressing the underlying cause of cystic fibrosis.
• * Successful CFTR gene delivery and expression pave the way for improved therapeutic outcomes.
• * Ongoing research aims to enhance vector safety and long-term CFTR expression for sustained clinical benefit.