Gene repair technique could have various therapeutic uses

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Scientists may have unlocked a way to therapeutically correct genetic defects by using a new technique that targets and repairs defective genes.

Researchers from the Morgridge Institute for Research in Madison, WI, and collaborators at Northwestern University say the process, which uses human pluripotent stem cells and DNA-cutting protein from meningitis bacteria, is simpler than previously attempted methods and could help lead to advances in regenerative medicine, drug screening and biomedical research.

The team selected Neisseria meningitides bacteria for its study because it is a rich source of the Cas9 protein, which is needed for accurately slicing damaged pieces of DNA.

"We are able to guide this protein with different types of small RNA molecules, allowing us to carefully remove, replace or correct problem genes," researcher Yan Zhang of Northwestern University said in a statement.

Previous gene correction methods relied on engineered proteins to help with the cutting. But these proteins take weeks or months to engineer, compared to RNA, which can be synthesized for the new process in one to three days, according to the researchers.

Researchers said the new technique has a wide array of therapeutic applications, including repairing genetic defects that drive some forms of breast cancer, Parkinson's and other diseases. The research was published Aug. 12 in the Proceedings of the National Academy of Sciences.

Human pluripotent stem cells (hPSCs) are widely thought to hold promise for regenerative medicine because of their ability to differentiate into almost all of the cells types of the body.

- here's the press release
- read the study abstract

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