1
Bennett C Frank, Dean Nicholas M, Kole Ryszard, Kopczynski Casey C: Chimeric oligomeric compounds for modulation of splicing. Isis Pharmaceuticals, Bennett C Frank, Dean Nicholas M, Ercole Biotech, Kole Ryszard, Kopczynski Casey C, FULLER Michael L, March 8, 2007: WO/2007/028065 (65 worldwide citation)

Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulati ...


2
Sazani Peter L, Kole Ryszard, Orum Henrik: Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease. The University Of North Carolina At Chapel Hill, Ercole Biotech, Santaris Pharma, Sazani Peter L, Kole Ryszard, Orum Henrik, MEIKLEJOHN Anita L, May 24, 2007: WO/2007/058894 (28 worldwide citation)

Methods and compositions are disclosed for controlling expression of TNF receptors (TNFRl and TNFR2) and of other receptors in the TNFR superfamily using compounds that modulate splicing of pre-mRNA encoding these receptors. More specifically these compounds cause the removal of the transmembrane do ...


3
Kole Ryszard, Sazani Peter, Wan Jing: Soluble her2 and her3 splice variant proteins, splice-switching oligonucleotides, and their use in the treatment of disease. Avi Biopharma, Kole Ryszard, Sazani Peter, Wan Jing, DEHLINGER Peter J, December 18, 2008: WO/2008/153933 (7 worldwide citation)

Soluble epidermal growth factor receptors 2 and 3 (HER2 and HER3) splice variant proteins with HER2 and HER3 antagonist activity and anti-proliferative properties, as well as the corresponding nucleic acids, are provided for treatment of proliferative diseases, in particular cancer. Also provided ar ...


4
Sazani Peter L, Graziewicz Maria, Kole Ryszard: Soluble tnf receptors and their use in treatment of disease. Ercole Biotech, The University Of North Carolina At Chapel Hill, Sazani Peter L, Graziewicz Maria, Kole Ryszard, PASSNER Jonathan, May 2, 2008: WO/2008/051306 (7 worldwide citation)

The present invention relates to tumor necrosis factor (TNF) antagonists and corresponding nucleic acids derived from tumor necrosis factor receptors (TNFRs) and their use in the treatment of inflammatory diseases. These proteins are soluble secreted decoy receptors that bind to TNF and prevent TNF ...


5
Kole Ryszard: Methods and compositions for modification of splicing of pre-mrna. University Of North Carolina At Chapel Hill, Kole Ryszard, MYERS BIGEL SIBLEY PO Box 37428 Raleigh NC 27627, April 8, 2004: WO/2004/028464 (1 worldwide citation)

The present invention provides a method of preventing a splicing event in a pre-mRNA molecule, comprising contacting the pre-mRNA and/or elements of the splicing machinery with a small molecule compound identified according to the methods described herein to prevent the splicing event in the pre-mRN ...


6
SAZANI PETER , KOLE RYSZARD : [de] MEHRERE EXON-SKIPPING-ZUSAMMENSETZUNGEN FÜR DMD, [en] MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD, [fr] COMPOSITIONS DE SAUT DEXON MULTIPLES POUR DMD. SAREPTA THERAPEUTICS  , February 22, 2017: EP3133160-A1

[en] The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes ...


7
Kole Ryszard, Dominski Zbigniew: Antisense oligonucleotides which combat aberrant splicing and methods of using the same. Univ North Carolina, March 12, 2008: EP1897942-A1

A method of blocking a splicing process in a pre-mRNA molecule is disclosed. The splicing process is mediated by splicing factors and the pre-mRNA molecule contains a set of splice elements which bind the splicing factors and define an intron which is removed by splicing to produce a mRNA molecule e ...


8
Bennett C Frank, Dean Nicholas M, Kole Ryszard, Kopczynski Casey C: Chimeric oligomeric compounds for modulation of splicing. Isis Pharmaceuticals, Ercole Biotech, July 2, 2008: EP1937312-A2

Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulati ...


9
SAZANI PETER , KOLE RYSZARD : [fr] Compositions de saut dexon multiples pour dmd, [de] Mehrere Exon-Skipping-Zusammensetzungen für DMD, [en] Multiple exon skipping compositions for DMD. SAREPTA THERAPEUTICS  , August 6, 2014: EP2762567-A1

[en] The present invention relates to an oligonucleotide which specifically hybridizes to a target region in an exon of the human dystrophin gene inducing exon skipping. The present invention further relates to a pharmaceutical composition comprising an oligonucleotide which specifically hybridizes ...


10
Kole Ryszard, Dominski Zbigniew: Oligonucleotides antisens actifs contre lepissage aberrant; methodes dutilisation, Antisense oligonucleotides which combat aberrant splicing and methods of using the same. University Of North Carolina At Chapel Hill, SIM & MCBURNEY, November 24, 1994: CA2162361

A method of combatting aberrant splicing in a pre-mRNA molecule containing a mutation is disclosed. When present in the pre-mRNA, the mutation causes the pre-mRNA to splice incorrectly and produce an aberrant mRNA or mRNA fragment different from the mRNA ordinarily encoded by the pre-mRNA. The metho ...



Click the thumbnails below to visualize the patent trend.