Skip to content
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Menu
Home
About Us
Resources
Profiles Metrics
Authors Directory
Institutions Directory
Top Authors
Top Institutions
Top Sponsors
AI Digest
Contact Us
Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX
Gene Therapy, Volume 13, No. 2, Year 2006
Notification
URL copied to clipboard!
Description
Immune responses against an introduced transgenic protein are a potential risk in many gene replacement strategies to treat genetic disease. We have developed a gene delivery approach for hemophilia B based on lentiviral expression of human factor IX in purified hematopoietic stem cells. In both normal C57Bl/6J and hemophilic 129/Sv recipient mice, we observed the production of therapeutic levels of human factor IX, persisting for at least a year with tolerance to human factor IX antigen. Secondary and tertiary recipients also demonstrate long-term production of therapeutic levels of human factor IX and tolerance, even at very low levels of donor chimerism. Furthermore, in hemophilic mice, partial functional correction of treated mice and phenotypic rescue is achieved. These data show the potential of a stem cell approach to gene delivery to tolerize recipients to a secreted foreign transgenic protein and, with appropriate modification, may be of use in developing treatments for other genetic disorders. © 2006 Nature Publishing Group All rights reserved.
Authors & Co-Authors
Bigger, Brian W.
United Kingdom, London
Imperial College London
United Kingdom, Bristol
Nhs Blood and Transplant
Waddington, Simon N.
United Kingdom, London
Imperial College London
Nivsarkar, Megha S.
United Kingdom, London
Imperial College London
Holder, Maxine V.
United Kingdom, London
Imperial College London
Ali, Robin R.
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Coutelle, Charles C.
United Kingdom, London
Imperial College London
Wright, Nicholas A.W.
United Kingdom, London
Cancer Research uk
Alison, Malcolm Ronald
United Kingdom, London
Imperial College London
Thrasher, Adrian James
United Kingdom, London
Ucl Great Ormond Street Institute of Child Health
Bonnet, Dominique A.
United Kingdom, London
Cancer Research uk
Themis, Michael
United Kingdom, London
Imperial College London
Statistics
Citations: 50
Authors: 11
Affiliations: 5
Identifiers
Doi:
10.1038/sj.gt.3302638
ISSN:
14765462
Research Areas
Cancer
Genetics And Genomics