World's first cell‑rejuvenating therapy given to patient
Published in Health & Fitness
A Boston company treated its first patient with a therapy intended to allow aging optic nerve cells to behave as though they were young again.
The experimental treatment by Life Biosciences will deliver three distinct genetic edits to regenerate neurons in the optic nerve, which can be damaged in people with glaucoma. These neurons do not normally regenerate in adults, leading to eventual blindness. The company announced its findings in the June issue of Nature.
Animal studies in several labs have suggested that partially reprogramming cell tissue can be done safely, though fears remain that the therapy could tip some cells into a cancerous state.
To ensure safety, Life Biosciences relies on a virus commonly used in gene therapy to deliver the three reprogrammed genes to retinal ganglion cells in the patient’s optic nerve. To better control the process, the rewritten genes are designed to switch on only when the participant takes the antibiotic doxycycline, said company chief scientific officer Sharon Rosenzweig-Lipson.
“It gives us a lot of control,” Rosenzweig-Lipson told Scientific American. “And the ability not just to turn it on, but to turn it off and not leave on expression longer than is necessary to rejuvenate the cells.”
The experiment aims to treat up to 12 people with glaucoma and, someday, include people with a severe acute condition called NAION that also damages optic nerves.
Researchers caution that success does not necessarily prove that they can program cellular immortality, but the company is also experimenting with animal models of liver disease.
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