Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

Phase 1 and phase 2 gene therapy trials using intramuscular (IM) administration of a recombinant adeno-associated virus serotype 1 (rAAV1) for replacement of serum alpha-1 antitrypsin (AAT) deficiency have shown long-term (5-year) stable transgene expression at approximately 2% to 3% of therapeutic...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Gruntman, Alisha M., Gernoux, Gwladys, Tang, Qiushi, Ye, Guo-Jie, Knop, Dave R., Wang, Gensheng, Benson, Janet, Coleman, Kristen E., Keeler, Allison M., Mueller, Christian, Chicoine, Louis G., Chulay, Jeffrey D., Flotte, Terence R.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6383191/
https://ncbi.nlm.nih.gov/pubmed/30828586
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.01.013
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