Gene therapy has been ineffective and controversial at best, unethical at worst. But it remains one of the most promising ways of curing genetic diseases. Recently, a method of RNA interference has been used to reverse sickle cell disease in the lab.
- Sloan-Kettering researchers cultured bone marrow-derived stem cells in the lab.
- A small portion of RNA that interferes with the production of beta S-globin — most people have only hemoglobin A while those with sickle cell disease have only S — is inserted it in a gene that promotes the production of healthy hemoglobin.
- The transgene containing the synthetic RNA is placed in a virus which introduces the gene into the stem cells of the sickle cell patients.
- The transfected cells did not produce beta S-globin but could perform all the functions and produce all the products of healthy red blood cells.
Currently, the only treatment for sickle cell disease is a stem cell transplant from an outside donor
Sickle cell disease — which affects about 80,000 people in the United States — is a genetic condition that makes red blood cells produce abnormal hemoglobin and turn from their normal round disk shape to narrow sickle forms. The sickle-shaped cells don’t flow smoothly through small blood vessels the way disk-shaped red blood cells do, but pile up in clumps and block the vessels, causing pain, infections, organ damage and strokes.
United Press International, December 27, 2005
Technorati Tags: gene+therapy, sickle+cell+disease, genes, dna, health, genetics, stem+cells
This is amazing
i don’t get it
[...] Disappointing news on the gene therapy front. RNA interference (RNAi) was once thought to have great potential for directly silencing genes and stopping them from producing functional protein products. Stanford scientists report that in a study of hepatitis B in which they attemped to silence the virus’s genes, RNAi cured some mice, caused liver poisoning in others, and death in a few. [...]