Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy

Abstract excerpt

<h4>ABSTRACT</h4> Lipodystrophy is a rare disorder which can be life-threatening. Here individuals fail to develop or maintain appropriate adipose tissue stores. This typically causes severe metabolic complications, including hepatic steatosis and lipoatrophic diabetes. There is no cure for lipodystrophy, and treatment options remain very limited. Here we evaluate whether tissue-selective adeno-associated virus (...

Topics

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• Brain Diseases, Metabolic
• CRISPR-Cas Systems
• Disease Management
• Drug Evaluation, Preclinical
• Gene Editing
• Gene Transfer Techniques
• Liver Diseases
• Transduction, Genetic

Identifiers and source

Literature Corpus work

4e776f8b-c3ad-5833-a3e3-6be6b9b7158c

DOI

10.1101/2024.06.27.600757