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Five RNAi-Related Patent Applications Published by the US Patent Office: Nov 12, 2004

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Title: Treatment of Neurodegenerative Disease Through Intracranial Delivery of siRNA. Number: 20040220132. Filed: May 25, 2004. Inventor: William Kaemmerer, Medtronic.

According to the patent application’s abstract, the invention comprises “devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein.”

The invention also comprises “small interfering RNA vectors and methods for treating neurodegenerative disorders such as Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.”


Title: Compact Synthetic Expression Vector Comprising Double-Stranded DNA Molecules and Methods of Use Thereof. Number: 20040220130. Filed: March 24, 2004. Lead Inventor: Paul Robbins, University of Pittsburgh.

The invention “relates to a safe, simple, compact, synthetic, and potentially non-toxic vector that is comprised of two or more complementary strands of chemically synthesized deoxyribonucleic acid that, when annealed to one another, form a cassette for the efficient expression of single-stranded or double-stranded ribonucleic acid molecules,” the patent application’s abstract states. “The RNA molecules expressed from this synthetic vector may function as ribozymes, as antisense molecules, or as short-interfering RNA molecules. Alternatively, the RNA molecules may encode therapeutic or antigenic peptides, polypeptides, or proteins,” it adds. “This synthetic vector may be employed for the rapid screening of various candidate RNA molecules for their efficacy in gene silencing or other functions, or for the delivery of RNA molecules in therapeutic applications.”


Title: Compositions and Method for siRNA Inhibition of ICAM-1. Number: 20040220129. Filed: Jan. 16, 2004. Lead Inventor: Samuel Reich, University of Pennsylvania (Acuity Pharmaceuticals).

“RNA interference using small interfering RNAs which are specific for the ICAM-1 gene inhibits expression of this gene,” the patent application’s abstract states. “Diseases which involve ICAM-1-mediated cell adhesion, such as inflammatory and autoimmune diseases, diabetic retinopathy and other complications arising from type I diabetes, age related macular degeneration, and many types of cancer, can be treated by administering the small interfering RNAs.”


Title: Nucleic Acid-Based Modulation of Female Reproductive Diseases and Conditions. Number: 20040220128. Filed: Nov. 13, 2003. Lead Inventor: Pamela Pavco, Sirna Therapeutics.

The invention “relates to nucleic acid molecules, including dsRNA [and] siRNA … which modulate the expression of vascular endothelial growth factor receptor and/or vascular endothelial growth factor receptor genes for the treatment and/or diagnosis of diseases and conditions associated with angiogenesis, such as cancer [or] tumor angiogenesis; ocular indications such as diabetic retinopathy or age related macular degeneration; proliferative diabetic retinopathy; hypoxia-induced angiogenesis; rheumatoid arthritis; psoriasis; wound healing; and female reproductive disorders and conditions, including but not limited to endometriosis, endometrial carcinoma, gynecologic bleeding disorders, irregular menstrual cycles, ovulation, premenstrual syndrome, and menopausal dysfunction,” the patent application’s abstract states.


Title: RNA Interference-Mediated Treatment of Parkinson’s Disease Using Short-Interfering Nucleic Acid. Number: 20040219671. Filed: Oct. 31, 2003. Lead Inventor: James McSwiggen, Sirna Therapeutics.

According to the patent application’s abstract, the invention “concerns methods and reagents useful in modulating Parkinson genes — for example, PARK1 (SNCA), PARK2, PARK7, and/or PARK5 gene expression — in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to small nucleic acid molecules, such as short-interfering nucleic acid, short-interfering RNA, double-stranded RNA, microRNA, and short-hairpin RNA molecules capable of mediating RNA interference against SNCA gene expression and/or activity.”

The abstract adds that the small nucleic acid molecules “are useful in the diagnosis and treatment of Parkinson Disease, and any other disease or condition that responds to modulation of PARK1 (SNCA), PARK2, PARK7, and/or PARK5 expression or activity.”

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