Novel Gene Therapy Method Replaces Mitochondrial DNA
Scientists have demonstrated a new type of gene therapy that would – in principle – allow mothers to avoid saddling their children with rare diseases that could result in heart problems, dementia, diabetes, deafness and other significant health issues.
The disorders in question are all due to mutations in one of the 37 genes in our mitochondrial DNA. Mitochondria have their own DNA, often designated as mtDNA. This is separate from the nuclear DNA in our chromosomes, where most of our genetic information is stored. An estimated 1 out of every 5,000 to 10,000 babies is born with some type of disorder related to faulty mtDNA, which works out to 1,000 to 4,000 kids in the U.S. each year, according to a report published online by the journal Nature. A team of researchers led by reproductive biologist Shoukhrat Mitalipov at Oregon Health & Science University wondered whether they could use gene therapy to prevent these births.