Engineering adeno-associated viruses for clinical gene therapy

By M. Kotterman, David V. Schaffer. Nature reviews genetics, 2014

735 citations recorded by the source corpus.

Clinical gene therapy has been increasingly successful owing both to an enhanced molecular understanding of human disease and to progressively improving gene delivery technologies. Among these technologies, delivery vectors based on adeno-associated viruses (AAVs) have emerged as safe and effective and, in one recent case, have led to regulatory approval. Although shortcomings in viral vector properties will render extension of such successes to many other human diseases challenging, new approaches to engineer and improve AAV vectors and their genetic cargo are increasingly helping to overcome these barriers.

Subfields: Genetics, Molecular Biology

View Paper: https://www.semanticscholar.org/paper/d53b0df5a74910f913005f97d36be6b418cf3d26

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In Genetics

1. Charles R. Gerfen
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In Molecular Biology

1. Min Wu
2. Mengyuan Huang
3. Hongyi Li
4. Xia Zhao
5. Yang Yang
6. Charles R. Gerfen
7. L. Looger
8. F. Mingozzi

Citations in the corpus

• FGF/FGFR signaling in health and disease (2020, methodology): “AAV has unique advantages of gene transfer for therapeutic treatment of a number of diseases, including congenital blindness, hemophilia, and spinal muscular atrophy.(741,742) Our group screened a siRNA specifically targeting the FGFR2-P253R allele, when this siRNA was delivered to the skulls in AS ”
• Emerging Issues in AAV-Mediated In Vivo Gene Therapy (2017, background): “The increasing knowledge of AAV capsid structure-function(22) has allowed the modification of specific amino acid residues by rational design, while the development of AAV capsid libraries and highthroughput screening methods enabled the identification of the most efficient capsid variant for the de”
• A designer AAV variant permits efficient retrograde access to projection neurons (2016, background): “Here we describe the in vivo directed evolution (Kotterman and Schaffer, 2014) of a new rAAV variant (rAAV2retro) that, in addition to its regular ability to infect neuronal cell bodies at the site of exposure, is robustly internalized by axons and mediates retrograde access to projection neurons…”
• Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects (2020, background): “In contrast, viral vectors (such as retroviruses, lentivirus, adenovirus (AdV) and AAV) have high delivery efficiency, and some of them have been approved for clinical uses.(319,320) To date, viral delivery systems have been the most effective system for delivering plasmid-based nucleic acids to mam”