Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins
Amit Chunilal Nathwani, Cecilia Rosales, Jenny McIntosh, Ghasem Rastegar Lari, Devhrut Nathwani, Deepak Raj, Sushmita Nawathe, Simon N. Waddington, Roderick Terry Bronson, Scott Jackson, Robert E. Donahue, Katherine A. High, Federico Mingozzi, Catherine Y. Ng, Junfang Zhou, Yunyu Spence, M. Beth McCarville, Marc B. Valentine, James A. Allay, John Coleman, Susan Sleep, John T. Gray, Arthur W. Nienhuis, Andrew M. Davidoff
The abstract is not reproduced here because its license does not permit redistribution. Read it at the original source.
Record
- Archive ID
- doi:10.1038/mt.2010.274
- Published
- Jan 18, 2011
- Venue
- Molecular Therapy
- DOI
- 10.1038/mt.2010.274
- PubMed
- 21245849
- Categories
- Virus-based gene therapy research
- License
- cc-by-nc-nd
- Provenance
- openalex