Therapeutic liver cell transplantation to treat a genetic liver defect

Abstract excerpt

For gene therapy of the liver, in vivo applications based on adeno-associated virus are the most advanced vectors despite limitations, including low efficacy and episomal loss, potential integration and safety issues, and high production costs. Alternative vectors and/or delivery routes are of high interest. The regenerative ability of the liver bears the potential for ex vivo therapy using liver cell transplant...

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

c45b110b-55c7-5b2b-8d5e-ee3b149e961b

DOI

10.1101/2024.06.26.600783