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Gene therapy may be future retinoblastoma treatment
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MEMPHIS, Tenn. — Three genes that express at different times during retinal development in mice may be the key to developing a treatment for retinoblastoma, researchers at St. Jude Children’s Research Hospital said in a press release.
The researchers said when one of those genes — Rb — was inactivated during the development of the mouse retina, two p107 gene copies were up-regulated, thereby compensating for the initial Rb activity loss.
The researchers said “the ability of Rb and p107 to compensate for the absence of each other in mice prevents the developing retinal cells from multiplying uncontrollably and causing retinoblastoma.”
In humans, however, the key gene is RB1 and little p107 is expressed in while the retina is developing. Also, p107 is not up-regulated to compensate for a loss of RB1 activity.
In short, the difference between mice and humans might explain why people are more susceptible to retinoblastoma after a gene mutation.
Scientists might be able to prevent retinoblastoma in humans by “turning on” the p107 gene, the release said.