Toward Permanent Engineered Solutions to Genetic Diseases; New Advance Enables Evolution of More Targeted Gene Editing Tools; Continuous Directed Evolution of DNA-Binding Proteins Improves TALEN Specificity 100-Fold

In his mind, Dr. Basil Hubbard can already picture a new world of therapeutic treatments for millions of patients just over the horizon. It's a future in which diseases like muscular dystrophy, cystic fibrosis, polycystic kidney disease (PKD), and many others may be treated permanently through the science of genome engineering. Thanks to his latest work, Dr. Hubbard is bringing that future closer to reality. His latest research, published online on August 10, 2015 in Nature Methods, demonstrates a new technology advancing the field of genome engineering. The method significantly improves the ability of scientists to target specific faulty genes, and then "edit" them, replacing the damaged genetic code with healthy DNA. "There is a trend in the scientific community to develop therapeutics in a more rational fashion, rather than just relying on traditional chemical screens," says Dr. Hubbard, now an Assistant Professor of Pharmacology in the University of Alberta's Faculty of Medicine & Dentistry. "We're moving towards a very logical type of treatment for genetic diseases, where we can actually say, 'Your disease is caused by a mutation in gene X, and we're going to correct this mutation to treat it'. In theory, genome engineering will eventually allow us to permanently cure genetic diseases by editing the specific faulty gene(s)." The Nature Methods article is titled “The article is titled “Continuous Directed Evolution of DNA-binding Proteins to Improve TALEN Specificity.” Genome engineering involves the targeted, specific modification of an organism's genetic information. Much as a computer programmer edits computer code, scientists could one day replace a person's broken or unhealthy genes with healthy ones through the use of sequence-specific DNA-binding proteins attached to DNA-editing tools.
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