OHSU scientists unlock first critical step toward gene therapy treatme


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OHSU scientists unlock first critical step toward gene therapy treatment for patients with mitochondrial disease

Date: 20/07/2015

A study led by Shoukhrat Mitalipov, Ph.D., and Hong Ma, M.D., Ph.D., at the Center for Embryonic Cell and Gene Therapy at Oregon Health & Science University and the Oregon National Primate Research Center has revealed the first critical step in developing novel gene and stem cell therapy treatments for patients with mitochondrial disease.

This breakthrough, published online today in the journal Nature, sets the stage for replacing diseased tissue in patients and opens the door to a world of regenerative medicine where doctors are able to treat human diseases that are currently incurable.

The scientists successfully used mitochondrial replacement to create an embryonic stem cell with healthy mitochondria from a patient's skin cell containing mitochondrial DNA mutations. These mutations can cause a vast range of fatal or severely debilitating diseases, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia, and several other neurological diseases. In the United States, 1,000 to 4,000 children are born with mitochondrial DNA disease each year. There are no meaningful treatments or cures.

In May 2013, Mitalipov was the first in the world to demonstrate the successful use of somatic cell nuclear transfer to produce human embryonic stem cells from a research subject's skin cell. That breakthrough followed a six-year chain of discoveries that included his 2007 work demonstrating the nuclear transfer method to create embryonic stem cells from a nonhuman primate. This chain included what may be a new technique to help families prevent inherited mitochondrial disease in future generations.

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Source: PR News Wire

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