Engineering a permanent solution to genetic diseases

In his mind, 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 and many others are treated permanently through the science of genome engineering.

Thanks to his latest work, Hubbard is bringing that future closer to reality. Hubbard's research, published in the journal 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.

"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)."

Through his research, undertaken as a post-doctoral fellow in the lab of David R. Liu at Harvard University, Hubbard has developed a way to reduce the off-target DNA binding of a class of gene editing proteins known as transcription activator-like effector nucleases (TALENs). The new method allows researchers to rapidly evolve the proteins autonomously to make them more specific and targeted over time.