Synthetic mRNA Gene Therapies and Hepatotropic Non-viral Vectors for the Treatment of Chronic HBV Infections

Kairuz, Dylan; Singh, Prashika; Smith, Tiffany S.; Arbuthnot, Patrick; Ely, Abdullah; Bloom, Kristie

doi:10.1007/978-3-031-08415-7_8

Cited by 1 publication

(1 citation statement)

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“…Similarly, strategies that enhance the translation and stability of endogenous or exogenous mRNA that were already used in other disease scenarios could be applied [58]. Finally, in the long term, there will be gene replacement techniques available (e.g., CRISPR/Cas9) that will allow for the replacement of MBOAT7 mutations that are associated with increased accumulation of intracellular free fatty acids and hepatic steatosis.…”

Section: Challenges and Recent Progress In Treating Complex Nafldmentioning

confidence: 99%

The Pivotal Role of the Membrane-Bound O-Acyltransferase Domain Containing 7 in Non-Alcoholic Fatty Liver Disease

Chandrasekaran,

Weiskirchen

2023

Livers

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Non-alcoholic fatty liver disease (NAFLD) is a common and prevalent disorder affecting 25 percent of the adults in the United States and 32 percent of adults globally. It is one of the common causes of chronic liver disease characterized by steatosis, which can lead to inflammation, fibrosis, and cirrhosis. NAFLD is strongly associated with obesity and insulin resistance. Multiple genetic variants have been consistently found to be associated with NAFLD; one of them is found in the TMC4-MBOAT7 loci. One variant (rs641738 C>T) within MBOAT7 encoding lysophosphatidyl inositol acyltransferase increases the risk for NAFLD development and triggers hepatic inflammation by regulating arachidonic acid levels. This review provides an overview of the MBOAT7 gene, pathogenesis of NAFLD, understanding the regulation of MBOAT7 and mechanistic link between MBOAT7 and NAFLD. It further summarizes pathophysiologically relevant in vivo and in vitro studies on MBOAT7 and challenges in treating complex NAFLD with recent progress made in the treatment of NAFLD. As such, this review provides useful information on MBOAT7 and NAFLD interrelation, which has the potential of deciphering novel therapeutic targets rather than well-known genetic variants such as PNPLA3 and TM6SF2.

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Section: Challenges and Recent Progress In Treating Complex Nafldmentioning

confidence: 99%