An article in Science Daily states that gene therapy will soon be used to treat a condition called X-linked retinitis pigmentosa. This genetic defect affects young children who are born with full vision, and by the time they are about 20 years old, they are completely blind. The researchers in the University of Florida have found a way to replace the light-sensitive cells in the eye with genetically altered light receptor cells. “The UF researchers and colleagues at the University of Pennsylvania performed the technically challenging task of cloning a working copy of the affected gene into a virus that served as a delivery vehicle to transport it to the appropriate part of the eye. They also cloned a genetic "switch" that would turn on the gene once it was in place, so it could start producing a protein needed for the damaged eye cells to function.” Researchers are nearing the end of trials in animals and soon hope to begin human testing, with the hopes of curing blindness in the near future.
Tuesday, April 17, 2012
Possible treatment for X-linked pigmentosa
An article in Science Daily states that gene therapy will soon be used to treat a condition called X-linked retinitis pigmentosa. This genetic defect affects young children who are born with full vision, and by the time they are about 20 years old, they are completely blind. The researchers in the University of Florida have found a way to replace the light-sensitive cells in the eye with genetically altered light receptor cells. “The UF researchers and colleagues at the University of Pennsylvania performed the technically challenging task of cloning a working copy of the affected gene into a virus that served as a delivery vehicle to transport it to the appropriate part of the eye. They also cloned a genetic "switch" that would turn on the gene once it was in place, so it could start producing a protein needed for the damaged eye cells to function.” Researchers are nearing the end of trials in animals and soon hope to begin human testing, with the hopes of curing blindness in the near future.
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Gene therapy articles are always good read since they seem to promise potent curative properties that go beyond what modern medicine can offer at this time. However, as the article pointed out, "Several complex and costly steps remain before the gene therapy technique can be used in humans, but once at that stage, it has great potential to change lives." It would be interesting to research just how far off gene therapy is and if it would indeed be the standard protocol of care for our generation. It would certainly be a historic shift in medicine to transition from pharmacokinetics to gene therapy, the same way medicine transitioned from bodily humors to pharmacokinetics in the past.
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