News ID: 124301
Published: 0329 GMT August 11, 2015

Engineering permanent solution to genetic diseases

Engineering permanent solution to genetic diseases

A research ― conducted by Dr. Basil Hubbard, an assistant professor of pharmacology in the University of Alberta's Faculty of Medicine & Dentistry ― shows 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," said Hubbard. "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 genes."

Genome engineering involves the targeted, specific modification of an organism's genetic information. Much like how 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. The field has made large strides over the last two decades and may one day revolutionize medical care.

One of the obstacles still to be addressed in the field before it can see widespread use in humans is how to ensure the proteins only affect the specific target genes in need of repair. With current technologies, the proteins bind to and edit the correct genes the vast majority of the time, but more improvements are needed to ensure off-target genes aren't modified — a result that could potentially cause serious health problems itself.

Hubbard has developed a way to reduce the off-target DNA binding of a class of gene editing proteins known as transcription activator-like effect or nucleases (TALENs). The new method allows researchers to rapidly evolve the proteins autonomously to make them more specific and targeted over time.

This technology allows you to systematically say, “I want to target this DNA sequence, and I don't want to target these others,” and it basically evolves a protein to do just that," said Hubbard. "Using this system, we can produce gene editing tools that are 100 times more specific for their target sequence."

 

   
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