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As a journalist, you can create a free Muck Rack account to customize your profile, list your contact preferences, and upload a portfolio of your best work.Articles
Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models
Abstract Arthrogryposis, Renal dysfunction and Cholestasis (ARC) syndrome is a rare inherited disorder caused by defects in the VPS33B trafficking protein, leading to impaired bile flow, progressive liver disease and early death. No effective treatments are currently available. Gene therapy offers a potential approach by restoring the missing VPS33B protein in liver cells.
Ex vivo primary liver sections recapitulate disease phenotype and therapeutic rescue for liver monogenic diseases
New Results doi: https://doi.org/10.1101/2023.03.23.533840 Abstract In academic research and the pharmaceutical industry, in vitro single cell line cultures and in vivo animal models are considered as gold standards in modelling diseases and assessing therapeutic efficacy.
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by <i>Asl</i> gene transfer
The AslNeo/Neo mice (B6.129S7-Asltm1Brle/J) were purchased from Jackson Laboratory (Bar Harbor, ME). For the initial experiments studying the neurological phenotype, WT and AslNeo/Neo littermates were maintained on standard rodent chow (Harlan 2018, Teklab Diets, Madison, WI; protein content 18%) with free access to water.
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