Another Big Gene Therapy Breakthrough to Prevent Serious Childhood Diseases
OHSU's Dr. Shoukhrat Mitalipov
Today, OHSU is announcing a major step in making gene therapy a reality for families facing gene-linked diseases passed from mother to child via cell mitochondria.
The research is summarized in this press release:
An excerpt:
The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov’s previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women’s Health, demonstrated efficacy of this therapy in human gametes and embryos.
“Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant,” explained Mitalipov. “When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations.”
Dr. Mitalipov explains the work further in this interview:
Here are a few of the initial news stories on the breakthrough
ASSOCIATED PRESS – Oregon scientists make embryos with 2 women, 1 man
NBC – New technique replaces diseased DNA, but would give kids two mothers
WALL STREET JOURNAL – DNA Technique Shows Promise Against Mitochondrial Disease
THE TELEGRAPH (UK) – ‘Three parent embryos’ created from human eggs
YAHOO NEWS – IN LAB, PROGRESS IN FIXING A SOURCE OF GENETIC DISEASE
