Molecular therapy. Nucleic acids.
Publisher:
Nature Pub. Group. Cambridge, MA : Cell Press (2017)
Frequency: Monthly
Country: United States
Language: English
Author(s):
American Society of Gene & Cell Therapy.
Start Year:2012 -
ISSN:
2162-2531 (Electronic)
2162-2531 (Linking)
2162-2531 (Linking)
Impact Factor
8.8
2022
| NLM ID: | 101581621 |
| (OCoLC): | 739098571 |
| LCCN: | 2011204562 |
Optimization of scAAVIL-1ra In Vitro and In Vivo to Deliver High Levels of Therapeutic Protein for Treatment of Osteoarthritis. Osteoarthritis (OA) affects over 40 million people annually. We evaluated interleukin-1 receptor antagonist (IL-1ra) gene transfer in an equine model based on IL-1ra protein therapy which inhibits inflammation through blocking IL-1. Using the self-complementary adeno-associated virus (scAAV)IL-1ra equine gene as a starting construct, we optimized the transgene cassette by analyzing promoters (cytomegalovirus (CMV) versus chicken β-actin hybrid (CBh)), coding sequences (optimized versus unoptimized), vector capsid (serotype 2 versus chimeric capsid), and biological activity in vitro. AAV serot...