Treating Cystic Fibrosis with mRNA Therapy or CRISPR

-The potential for treating cystic fibrosis (CF) using mRNA therapies or CRISPR gene editing is possible regardless of the causative mutation. CF clinical trials showing that a genotype-agnostic gene therapy for CF is possible are reviewed in the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free on the Human Gene Therapy website through November 4, 2020.

“Treating CF by delivering mRNA that encodes CFTR has the potential to work in any CF patient, independent of the underlying mutation,” state James Dahlman, Georgia Institute of Technology, and coauthors. “Another potential treatment is utilizing mRNA encoding nucleases such as CRISPRCas9 accompanied by gRNA and using them to edit DNA in target cells.”

Challenges remain to be able to utilize these approaches successfully. First among them is the need to identify drug delivery systems that can reach pulmonary epithelial cells at low doses.

“CF was the first disease target in humans for several vector platforms, including rAAV and rAd. It is gratifying to see these newer technologies applied to CF, particularly to the 5% of patients whose mutations are resistant to CFTR modulator drugs,” according to Editor-in-Chief of Human Gene Therapy Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School.

About the Journal

Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. For complete information and a sample issue, please visit the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers is known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 90 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

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