In a world first, a patent which encompasses 'vector expressed' siRNAs targeting a disease gene has been issued by the Australian Patent Office. The patent, which broadly covers all vector expressed siRNAs targeting the Huntington's disease (HD) gene, has been issued to Sirna Therapeutics, Inc. In particular, the patent includes vector expressed siRNAs with 3' -overhangs, blunt ends and short hairpin RNA (shRNA), and methods of using siRNAs to inhibit Huntington's disease gene expression.

Huntington's Disease (HD) is a hereditary degenerative brain disorder that is progressive and always fatal and for which there is, at present, no effective treatment or cure. The disease-causing gene produces a protein that is toxic to certain brain cells, which subsequently results to movement disorders, psychiatric disturbances and cognitive decline. HD affects more than 30,000 people in the United States, with another 200,000 at risk of inheriting the deadly gene.

siRNA, also called small interfering RNA or short interfering RNA, is a short piece of nucleic acid which can be artificially introduced to a cell to interfere with the expression of certain genes, in this case, the Huntington's disease gene. It may be introduced into the cell using adeno-associated viral vector (AAV) delivery technology developed by Targeted Genetics.

Sirna Therapeutics is a clinical-stage biotechnology company developing RNAi-based therapies for serious diseases and conditions, including age-related macular degeneration (AMD), hepatitis B and C, dermatology, asthma, Huntington's Disease, diabetes and oncology.

Read the press release about the patent here.

Image Credits: Lydia Kibiuk, � 1997 Lydia Kibiuk, http://apu.sfn.org/content/Publications/BrainBriefings/huntingtons.html