Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Cancer Institute (WIA) · London Cancer · +11 more institutions
Abstract
Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We investigated the use of a new gene therapy in patients with the disorder.
We infused a single dose of a serotype-8-pseudotyped, self-complementary adenovirus-associated virus (AAV) vector expressing a codon-optimized human factor IX (FIX) transgene (scAAV2/8-LP1-hFIXco) in a peripheral vein in six patients with severe hemophilia B (FIX activity,
Citation impact
- FWCI
- 98.22
- Percentile
- 100%
- References
- 27
Authors
32- ACAmit C. NathwaniCorresponding
Cancer Institute (WIA), London Cancer, CRUK Lung Cancer Centre of Excellence, University College London, NHS Blood and Transplant
- EGEdward G. D. Tuddenham
- SRSavita Rangarajan
Basingstoke and North Hampshire Hospital
- CRCecilia Rosales
University College London, NHS Blood and Transplant, Cancer Institute (WIA), London Cancer
- JMJenny McIntosh
University College London, NHS Blood and Transplant, Cancer Institute (WIA), London Cancer
Topics & keywords
- Medicine
- Genetic enhancement
- Asymptomatic
- Factor IX
- Vector (molecular biology)
- Virus
- Viral vector
- Internal medicine
- Good health and well-being
Funding
- HHHoward Hughes Medical Institute
- AFAssisi Foundation of Memphis
- SJSt. Jude Children's Research Hospital
- NBNHS Blood and Transplant
- ALAmerican Lebanese Syrian Associated Charities
- NINational Institute for Health and Care ResearchAward: RP-PG-0310-1001
- UCUniversity College London
- MRMedical Research Council
- NHNational Heart, Lung, and Blood InstituteAward: HL094396