Title: siRNA Targeting Connective Tissue Growth Factor
Number: 20080015114
Filed: March 29, 2007
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent application’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to CTGF.”
Title: Inhibition of Expression of Huntingtin Gene
Number: 20080015158
Filed: April 30, 2004 PCT Filed: April 30, 2004
Lead Inventor: Kanazawa Ichiro, University of Tokyo
The patent application, its abstract states, claims “methods for suppressing the huntingtin gene expression by using a double-stranded RNA, huntingtin gene expression inhibitors to suppress the huntingtin gene expression, and preventives and/or remedies of Huntington's disease. Targeting against a specific sequence of mRNA immediately upstream of CAG repeats in HD genes of Huntington's disease, the huntingtin gene expression is suppressed by using a dsRNA homologous to the sequence. In this invention, a short siRNA having [base pairs] as short as around 21-23 … can be effectively used as the dsRNA [that is] homologous to a specific RNA sequence in a region at immediately upstream of CAG repeats,” the abstract adds. “The dsRNA of this present invention can be used as a huntingtin gene expression inhibitor, or a preventive and/or a remedy of Huntington's disease by administering or introducing into a living body or a living cell in mammals for the prevention and/or treatment of Huntington's disease.”
Title: RNAi Modulation of TGF-Beta and Therapeutic Uses Thereof
Number: 20080015161
Filed: March 15, 2007
Lead Inventor: Hans-Peter Vornlocher, Alnylam
The invention, the patent application’s abstract states, “concerns methods of treatment using transforming growth factor beta modulators. More specifically, the invention concerns methods of treating disorders associated with undesirable TGF-beta signaling, by administering short interfering RNA which down-regulate the expression of TGF-beta, and agents useful therein.”