Gene therapy continues to crawl into our lives with promises and, this time, some surprises. Patrick Yu-Wai-Man, an ophthalmologist at Cambridge University, treated a patient with a mitochondrial gene defect leading to retinal cell death, causing the blinding disease called Leber hereditary optic neuropathy.
Image of human retina
This is due to a mutation in a single gene in mitochondria, which are energy-producing structures in cells that carry their own set of genes. Complicated? Indeed.
These researchers asked whether injecting a normal copy of the mutated mitochondrial gene might be a treatment for the debilitating disease? Surprise! Not only did it work to some extent on one eye treated with the corrected gene, it even restored the other eye to some extent as well.
Certainly promising. Apparently, the non-treated eye received the normal, injected mitochondrial gene via the optic nerve. Good news: gene therapy may work.
Yet, many questions arise. How did the transfer from one eye to the other actually take place? Why did the injected gene not go to any other tissue except the other eye? Did the injected gene find its way into mitochondria or the nuclear genes? If not, where did it go? What exactly happened? What cautions arose that need to be addressed for gene therapy? Once again, a usual, myriad new questions, and even new areas of research, accompany every step forward, not only for gene therapy, but for all aspects of basic research.
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