On the Corner of Models and Cure: Gene Editing in Cystic Fibrosis

Frontiers in Pharmacology

Teresinha Leal¹  Claire Detry¹  Angélique Mottais¹  Marianne S. Carlon²  Marjolein Ensinck² 

[1] Institut de Recherche Expérimentale et Clinique, Louvain Centre for Toxicology and Applied Pharmacology, Université Catholique de Louvain, Brussels, Belgium;Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium;

关键词: cystic fibrosis;    CFTR;    gene therapy;    gene editing;    CF cell models;    CF animal models;   

DOI  :  10.3389/fphar.2021.662110

来源: DOAJ

【 摘 要 】

Cystic fibrosis (CF) is a severe genetic disease for which curative treatment is still lacking. Next generation biotechnologies and more efficient cell-based and in vivo disease models are accelerating the development of novel therapies for CF. Gene editing tools, like CRISPR-based systems, can be used to make targeted modifications in the genome, allowing to correct mutations directly in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene. Alternatively, with these tools more relevant disease models can be generated, which in turn will be invaluable to evaluate novel gene editing-based therapies for CF. This critical review offers a comprehensive description of currently available tools for genome editing, and the cell and animal models which are available to evaluate them. Next, we will give an extensive overview of proof-of-concept applications of gene editing in the field of CF. Finally, we will touch upon the challenges that need to be addressed before these proof-of-concept studies can be translated towards a therapy for people with CF.

【 授权许可】

Unknown