Isis Pharmaceuticals, Inc.

Other

Ionis Pharmaceuticals, Inc.

Susan F Murray

Susan M Freier

Punit P Seth

Michael L McCaleb

Priyam Singh

Brett P Monia

Lex M Cowsert

Sanjay Bhanot

Jacqueline R Wyatt

Nicholas M Dean

Shuling Guo

Michael L Mccaleb

Methods, compounds, and compositions useful for inhibiting HSD17B13 expression are provided. Such compounds, compositions, and methods are useful for treating, preventing, or ameliorating a disease associated with HSD 17B 13.

Modulators of HSD17B13 expression

The present embodiments provide methods, compounds, and compositions for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP) by administering a P23H rhodopsin specific inhibitor to a subject. The present embodiments provided herein are directed to compounds and compositions useful for treating, preventing, ameliorating, or slowing progression of retinitis pigmentosa (RP), such as autosomal dominant retinitis pigmentosa (AdRP). In certain embodiments, P23H rhodopsin inhibitors provided herein are allele-specific antisense compounds targeted to a P23H mutant allele that are capable of selectively inhibiting expression of P23H rhodopsin mutant protein to a greater extent than wild-type protein. In certain embodiments, administration of the allele specific antisense compounds in a subject having AdRP results in selective inhibition of P23H rhodopsin and allows the normal protein produced from the wild-type allele to maintain rod survival and function in the subject.

Allele specific modulators of P23H rhodopsin

Provided herein are methods, compounds, and compositions for reducing expression of DYRKIB in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate a metabolic disease or disorder in an individual in need.

Modulation of DYRK1B expression

Disclosed herein are compounds, compositions and methods for modulating the expression of PTPR.alpha. in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Also provided are methods for the prevention, amelioration and/or treatment of airway hyperresponsiveness and pulmonary inflammation by administration of antisense compounds targeted to PTPR.alpha.

Compositions and their uses directed to PTPR alpha

Compositions and methods are provided for modulating the expression of TGF-&bgr;. Antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding TGF-&bgr; are preferred. Methods of using these compounds for modulation of TGF-&bgr; expression and for treatment of diseases associated with expression of TGF-&bgr; are also provided. Methods of sensitizing cells to apoptotic stimuli are also provided.

Antisense inhibition of transforming growth factor-&bgr; expression

Antisense compounds, compositions and methods are provided for modulating the expression of Macrophage migration inhibitory factor. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding Macrophage migration inhibitory factor. Methods of using these compounds for modulation of Macrophage migration inhibitory factor expression and for treatment of diseases associated with expression of Macrophage migration inhibitory factor are provided.

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Poway, CA, United States of America

Susan F Murray