Year: 2025
Type: article
Abstract: Adeno-associated virus (AAV) vectors are the workhorse of in vivo gene delivery. However, the initial clinical studies of AAV rely largely on native tropism, with suboptimal tissue specificity necessitating high vector doses that can result in toxicity such as hepatotoxicity and immune responses. To overcome these barriers, efforts over the past two decades have focused on engineering the AAV capsid to redirect them toward specific target cells or tissues, with techniques ranging from directed evolution of mutant libraries, screening of random peptide insertions at specific locations, to rational design of point mutations or insertions to chemical modification.
Source: Molecular Therapy Oncology
Authors: Stephen L. Trisno, Mark A. Kay
Institution: Stanford University
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $3,900
OpenAlex ID: W4416985437