Scripps Research Institute Scientists Develop Alternative to Gene Therapy

Scripps Research Institute Scientists Develop Alternative to Gene Therapy

Scientists at The Scripps Research Institute have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating its use as a safer alternative to an experimental gene therapy against HIV infection.

“We showed that we can modify the genomes of cells without the troubles that have long been linked to traditional gene therapy techniques,” said the study’s senior author Carlos F. Barbas III, who is the Janet and Keith Kellogg II Professor of Molecular Biology and Chemistry at The Scripps Research Institute.

The new technique, reported in Nature Methods on July 1, 2012, employs zinc finger nuclease (ZFN) proteins, which can bind and cut DNA at precisely defined locations in the genome. ZFNs are coming into widespread use in scientific experiments and potential disease treatments, but typically are delivered into cells using potentially risky gene therapy methods.

Researchers think this work removes a major bottleneck in the efficient use of ZFN proteins as a gene therapy tool in humans.

The research was funded by NIH government grants R01GM065059, DP1OD006990, and T32GM080209 and by the Skaggs Institute of Chemical Biology at Scripps Research.