You are currently viewing the editor's summary.
View Full TextLog in to view the full text
AAAS login provides access to Science for AAAS members, and access to other journals in the Science family to users who have purchased individual subscriptions.
More options
Download and print this article for your personal scholarly, research, and educational use.
Buy a single issue of Science for just $15 USD.
Improving gene therapy efficacy
Clinical trial results of a gene therapy for hemophilia B reported unexpectedly low success. Cabanes-Creus et al. may have found a reason for this result. They report that the viral vector commonly used for liver-directed gene therapy, adeno-associated virus 2 (AAV2), mutates during culture, resulting in the virus binding more tightly to heparan sulfate proteoglycans (HSPGs). In contrast, naturally occurring AAVs isolated from human liver biopsies did not bind HSPGs tightly and were more successful at infecting human liver cells. However, these naturally occurring AAVs cultured over time also mutated, resulting in greater HSPG binding and decreased liver infection. Thus, naturally occurring AAVs may be more effective as vectors for liver-targeting gene therapies.
- Copyright © 2020 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works
This is an article distributed under the terms of the Science Journals Default License.
