Journal
BLOOD
Volume 122, Issue 9, Pages 1556-1564Publisher
AMER SOC HEMATOLOGY
DOI: 10.1182/blood-2013-04-453209
Keywords
-
Categories
Funding
- Assisi Foundation [94-000 R12]
- American Lebanese Syrian Associated Charities
Ask authors/readers for more resources
This review addresses the current status of gene therapy for immunodeficiencies, chronic granulomatous disease, suicide gene therapy for graft-versus-host disease, viral infections, malignant hematologic disorders, hemophilia, and the hemoglobin disorders. New developments in vector design have fostered improved expression as well as enhanced safety, particularly of integrating retroviral vectors. Several immunodeficiencies have been treated successfully by stem cell-targeted, retroviral-mediated gene transfer with reconstitution of the immune system following infusion of the transduced cells. In a trial for hemophilia B, long-term expression of human FIX has been observed following adeno-associated viral vector-mediated gene transfer into the liver. This approach should be successful in treating any disorder in which liver production of a specific protein is therapeutic.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available