Therapeutic editing of hepatocyte genome in vivo

Journal of Hepatology
Marina Ruiz de Galarreta, Amaia Lujambio

Abstract

The recent development of gene editing platforms enables making precise changes in the genome of eukaryotic cells. Programmable nucleases, such as meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)-associated nucleases have revolutionized the way research is conducted as they facilitate the rapid production of mutant or knockout cellular and animal models. These same genetic tools can potentially be applied to cure or alleviate a variety of diseases, including genetic diseases that lack an efficient therapy. Thus, gene editing platforms could be used for correcting mutations that cause a disease, restoration of the expression of genes that are missing, or be used for the removal of deleterious genes or viral genomes. In the context of liver diseases, genome editing could be developed to treat not only hereditary monogenic liver diseases but also hepatitis B infection and diseases that have both genetic and non-genetic components. While the prospect of translating these therapeutic strategies to a clinical setting is highly appealing, there are numerous challenges that need to be addressed first. Safety, effi...Continue Reading

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Citations

Apr 13, 2018·Genes·Kristie BloomPatrick Arbuthnot
May 28, 2019·Frontiers in Plant Science·Muntazir MushtaqRomesh Kumar Salgotra
Nov 27, 2019·Cancers·Kenya KamimuraShuji Terai
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Jun 24, 2021·JHEP Reports : Innovation in Hepatology·Sheila MaestroGloria Gonzalez-Aseguinolaza
Oct 5, 2021·Technology in Cancer Research & Treatment·Wei LvWei Wei

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