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First gene-editing disease cure success announced

Pathophysiology of metabolic disorders of tyrosine, resulting in elevated levels of tyrosine in blood.
Pathophysiology of metabolic disorders of tyrosine, resulting in elevated levels of tyrosine in blood.
LHcheM This file is licensed under the Creative Commons Attribution-Share Alike 3.0 Unported license by the copyright holder.

Daniel Anderson, the Samuel A. Goldblith Associate Professor of Chemical Engineering at Massachusetts Institute of Technology, and colleagues announced the first successful gene-editing that cured a disease on March 30, 2014, at the MIT news website.

The researchers cured mice of a rare liver disorder caused by a single genetic mutation by extracting the mutated DNA and replacing the mutated DNA with the correct sequence.

The scientists copied the genetic defense mechanism that bacteria use to create a molecule that delivers a coded DNA sequence, slices the defective DNA strand at the proper place, and replaces the defective DNA with the properly coded sequence.

The researchers claim that developing the molecule that carries the new DNA and slices the gene at the proper place is much simpler than other gene-editing methodologies.

The researchers have eliminated tyrosinemia in mice. The disease causes an inability to break down the amino acid tyrosine that can lead to liver failure. About one of 100,000 people in the world has tyrosinemia.

The expectations are that this methodology called CRISPR will be used to eliminate any disease in humans that is caused by a single gene mutation. The list of potential disease candidates includes hemophilia and Huntington's disease.