'Bubble Bassets' Cured of Genetic Disorder
Posted on: Tuesday, 16 May 2006, 15:03 CDT
U.S. scientists report using in-vivo gene therapy to restore the immune system in basset pups with X-linked severe combined immunodeficiency.
The dogs suffered from XSCID, a life-threatening genetic disorder that effectively disables the immune system.
Researchers from the University of Pennsylvania School of Veterinary Medicine and the National Institute of Allergy and Infectious Disease injected a retrovirus containing a corrective version of the gene responsible for XSCID, an important proof of principle for the technique of in-vivo gene therapy.
In humans, XSCID affects one in 100,000 boys, resulting from the inheritance of a faulty gene on one of the mother's X-chromosomes. It often proves fatal before the child's first birthday.
The disease came to public attention in the late 1970s with the Bubble Boy, David Vetter, who lived his entire life in a sterilized environment in order to protect him from outside germs. Vetter died in 1984.
The only current treatment for XSCID is through a bone-marrow transplant from a normal donor designed to replenish the hematopoietic stem cells that are capable of constantly renewing new functional immune cell.
The latest research appeared in the April 15 issue of the journal Blood.
Source: United Press International
Related Articles
- Progress in Gene Therapy for Immune Deficiency Diseases, Presented at the ASGT 9th Annual Meeting
- HLA-C and Killer Cell Immunoglobulin-Like Receptor Genes in Idiopathic Bronchiectasis
- French Immunologist Addresses Physicians and Scientists at Childrens Hospital Los Angeles
- Chair of the California Institute of Regenerative Medicine Delivers Keynote Address at the 2005 Saban Research Institute Symposium on Cell and Tissue Engineering
- Promoter Polymorphism of the CD14 Endotoxin Receptor Gene Is Associated With Biliary Atresia and Idiopathic Neonatal Cholestasis
- Positional Identification of an Asthma Susceptibility Gene on Human Chromosome 5q33
- Renal Manifestations of Sexually Transmitted Diseases: Sexually Transmitted Diseases and the Kidney
- Genetics of Asthma and COPD*: Similar Results for Different Phenotypes
- Genetic Approaches to Assessing Evidence for a T Helper Type 1 Cytokine Defect in Adult Asthma
- DNA vaccination with CD25 protects rats from adjuvant arthritis and induces an antiergotypic response
 |
 |
User Comments (0)
RSS Feeds