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Huntington's treatment could be transformed

Huntington's treatment could be transformed
31st January 2012

Exciting advances in Huntington's disease gene therapy could help to improve treatment in the future.

A study by the University of California Davis Health System has seen scientists deliver inhibitory RNA sequences from stem cells directly into neurons successfully for the first time.

According to study author Jan Nolta, this process will significantly decrease the production of mutant huntingtin proteins.

"Our team has made a breakthrough that gives families affected by this disease hope that genetic therapy may one day become a reality," she said.

The scientists demonstrated that mesenchymal stem cells genetically engineered to carry RNAi were capable of reducing mutant huntingtin.

They claim that a number of currently incurable diseases could benefit if the same process could be replicated.

Ms Nolta added: "The potential benefits of cellular therapies and RNAi therapies combined may be sufficient to delay or even halt disease progression."

A study last month suggested that the over-expression of regulatory enzymes in people with Huntington's disease could protect against neurodegeneration.