News Release 

Induced liver regeneration enhances CRISPR/Cas9-mediated gene repair

Mary Ann Liebert, Inc./Genetic Engineering News

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Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, November 9, 2020--Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver. This dietary induction of hepatocyte regeneration may be a viable clinical strategy to enhance gene repair in the liver, according to the peer-reviewed journal Human Gene Therapy. Click here to read the full-text article free through December 9, 2020.

The study was done in a mouse model of tyrosinemia type 1. "In neonatal mice, a gene correction frequency of ~10.8% of hepatocytes was achieved," said Qing-Shuo Zhang, Oregon Health & Science University and coauthors. "The efficiency in adult mice was significantly lower at ~1.6%."

Use of thyroid hormone T3 to temporarily induce hepatocyte division in the adult mice led to a significant increase in the gene correction efficiency to 3.5%.

"The promise of gene editing for human gene therapy is being realized initially with ex vivo manipulation of stem cells and lymphocytes and in small organ targets like the retina. If gene editing becomes efficient enough to correct genetic defects in vivo in the liver, it could then be used to treat a much wider variety of disorders. The work in this paper moves the field closer to that goal," 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.

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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. Complete tables of contents and a sample issue are available 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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