Mitochondrial disease therapy using stem cells appears feasible, according to a study published Wednesday in Nature. However, years of preclinical testing will likely be needed before human clinical trials can be considered.
Mitochondrial diseases have no cure, and no therapy addresses the underlying defects in mitochondria, cellular organelles that produce most of the body’s energy and perform other vital functions. Mitochondria contain their own DNA, a tiny fraction of all DNA in the body.
The new study corrected the defects with two approaches. In one, researchers generated induced pluripotent stem cells from patients, selectively growing them to isolate replacement cells that contain sufficient healthy mitochondria. In therapeutic use, if approved, the cells could then be matured into the needed tissue type, and transplanted back into the patient.
The other is somatic cell nuclear transfer or SCNT. Used to create Dolly the cloned sheep, this approach was demonstrated in humans for the first time in 2013 by OHSU’s Shoukhrat Mitalipov, a lead author of the new study. It would be used in cases where no healthy mitochondria can be isolated from the patient.
While the use of IPS cells is widely accepted, SCNT is controversial because the process creates what many consider to be the equivalent of a fertilized egg cell, representing the beginning of human life. SCNT supporters say the cloning process is only carried out for the purpose of therapy, so they distinguish between therapeutic cloning and reproductive cloning for the purpose of creating a new person.
The United Kingdom recently approved a method that resembles reproductive cloning to prevent mitochondrial disease inheritance. This process results in what has been called “three-parent babies.” This method differs from reproductive cloning such as that used for Dolly in that it doesn’t create a genetic copy of an organism.
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