Clinical trials must produce exactly the expected result or they are declared a failure. A clinical trial can fail by producing unexpected benefit, and this has happened to Gensight Biologics’ work on allotopic expression of the mitochondrial NH4 gene, aimed at the treatment of inherited retinal degeneration caused by mutation in NH4. Allotopic expression is a process in which a copy of the correctly formed gene is placed into the cell nucleus, suitably altered to enable transport of the protein produced back to the mitochondria where it is need.
In a sane world, this therapy would long ago have been available to anyone willing to accept the risk, based on positive earlier results and lack of serious side-effects. In that same sane world, the therapy would now be available to anyone willing undergo the risk of gaining a greater benefit than was hoped for. Unfortunately we don’t live in that world, and Gensight will now have to run further very expensive trials before regulators will permit the treatment to reach the clinic.
We watch progress at Gensight because allotopic expression of NH4 is one-thirteenth of a rejuvenation therapy – Gensight is the result of research supported and encouraged by
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