Medtronic, Inc.

Warsaw Orthopedic, Inc.

Eric Burright

Deepak Ramesh Thakker

William Frederick Kaemmerer

Jennifer Heisel

John Myers Zanella

Carl A Schu

Paul W Wacnik

Lisa Lynn Shafer

Lothar Krinke

Paul Van Bilsen

Bill McKay

The present invention is directed to compositions methods and kits for regulation of gene therapies, including, without limitation, reversible gene therapies and allele-specific therapies.

Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity

Methods and kits are provided for determining which single nucleotide polymorphism ('SNP') variant of an allele of a heterozygous patient is on the same allele as a disease-causing mutation that is at a remote region of the gene's mRNA comprising a) an allele specific reverse transcription reaction using an allele specific primer which recognizes one SNP variant, wherein further the 3' end of the primer is positioned at the SNP nucleotide position, and b) analysis of the resulting cDNA product from the reverse transcription reaction at the region of the mutation to determine the presence or absence of the mutation on this allele specific cDNA product, wherein the allele specific primer is shorter than about 20 nucleotides.

Methods and kits for linking polymorphic sequences to expanded repeat mutations

Disclosed herein are systems and methods for contributing to the local treatment of pain. More specifically, the disclosed systems and methods contribute to the local treatment pain by inhibiting the NF&#x3ba;B family of transcription factors.

Systems and methods to treat pain locally

The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies.

The instant invention provides a method for improving efficiency of RNA delivery to cells. The method comprises applying a low strength electric field to the cells and then after a certain time period, administering the ribonucleic acid sequence to the cells. Devices, kits, and RNA molecules suitable for delivery and devices suitable for practicing the disclosed methods are also provided.

Methods and devices for improved efficiency of RNA delivery to cells

Disclosed herein are methods, sequences and nucleic acid molecules used to treat pain. Specifically, the methods and sequences include locally administering molecules that suppress the expression of amino acid sequences that encode for Na1.7 channels or that otherwise inhibit the function of Na1.7 channels. Local suppression of Na1.7 channel levels and/or function will occur in the peripheral sensory neurons of the dorsal root ganglia.

Suppression of SCN9A gene expression and/or function for the treatment of pain

The invention provides novel combination therapies for treating neurodegenerative disease which comprise a) neurotrophic factors or suitable fragments thereof and b) agents capable of causing inhibition of a gene responsible for the neurodegenerative disease. The invention provides novel nucleic acid sequences, methods, and systems suitable for applications of these combination therapies.

Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof

The invention provides novel combination therapies for treating Huntington's disease which comprise a) BDNF or suitable fragments thereof and b) agents capable of causing inhibition of a gene responsible for the neurodegenerative disease. The invention provides nucleic acid sequences, methods, and systems suitable for applications of these combination therapies.

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Eagan, MN, United States of America

Eric Burright