Cystic Fibrosis (CF)

Adeno-associated viruses (AAVs) exhibit many advantages in the gene therapies of some human genetic diseases. With the efforts of our experienced scientists, Creative Biolabs provides AAV vector design service for treating and underlying mechanism discovery of cystic fibrosis (CF) for clients worldwide.

Current Treatment of Cystic Fibrosis

CF is an autosomal recessive monogenetic disease that is caused by a mutation in cystic fibrosis transmembrane conductance regulator (CFTR), a gene responsible for the production of sweat, digestive fluids, and mucus. The CFTR is a chloride channel protein regulating the generation and maintenance of a fluid layer on mucosal membranes covering airways, gastrointestinal tract, liver, pancreas, and epididymis. Thus, CF caused by CFTR mutations mainly affects lungs, livers and digestive system leading to symptoms as chronic airway infection, frequent lung infections, gastrointestinal dysfunction, pancreatic insufficiency, etc.

CF is a life-limiting disease that differs widely in types and severity from patient to patient. There is no effective cure for CF at present, patients usually treated by receiving combined symptomatic therapy for a lifetime, including antibiotics, CFTR modulators, pancreatic enzyme supplements, inhaled medicines and airway clearance to help build up the lung function. New effectively therapeutic approaches, such as gene therapy and phage therapy are explored and developed to keep long-lasting efficacy of CF treatment.

AAV Vector-Based Gene Therapy for Cystic Fibrosis

As a monogenetic disorder (loss function of CFTR protein), CF makes an ideal candidate for gene therapy. Human CFTR cDNA delivery into cells by AAV vectors to reverse the pathological phenotype caused by protein dysfunction is a preferred choice. Moreover, recombinant AAV vectors are able to integrate loaded correct human CFTR cDNA into the host cell chromosomes, enabling the long-term expression of the CFTR gene and long-lasting therapeutic effects. Current AAV vector-based gene therapies for CF treatment mainly focus on the transfer of correct DNA to the epithelial cells in the airways or lungs since most CF patients die of progressive lung disease. Several gene therapy products are researched in preclinical development for CF treatment, and the current challenge for CF gene therapy is to transfer these products from the bench side into clinical application. With the advanced molecular strategies of AAV design, modification, engineering, and manufacturing, CF treatment will be conquered by AAV vector-based gene therapy.

Figure 1. Viral vector-based gene therapy for cystic fibrosis.

Creative Biolabs provides AAV vector design services for research and clinical applications of CF, utilizing novel strategies to construct vectors with characteristics of high efficacy, long-lasting and low-toxicity. You are welcomed to contact us for more information or get your customized AAV vector design service.