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UHLMANN Eugen, WEBER Markus, SUBRAMANIAN Romesh, ROCKEL Thomas Dino, KRIEG Arthur M: [fr] COMPOSÉS OLIGONUCLÉOTIDIQUES MULTIMÈRES, [en] MULTIMERIC OLIGONUCLEOTIDE COMPOUNDS. UHLMANN Eugen, WEBER Markus, SUBRAMANIAN Romesh, ROCKEL Thomas Dino, KRIEG Arthur M, RANA THERAPEUTICS, YOUNG Daniel W, March 21, 2013: WO/2013/040429 (11 worldwide citation)

[en] The disclosure provides multimeric oligonucleotide compounds, comprising two or more target- specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target- specific oligo ...


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KRIEG Arthur M, SUBRAMANIAN Romesh, MCSWIGGEN James, LEE Jeannie T: [fr] COMPOSITIONS ET MÉTHODES POUR MODULER LEXPRESSION GÉNIQUE, [en] COMPOSITIONS AND METHODS FOR MODULATING GENE EXPRESSION. RANA THERAPEUTICS, THE GENERAL HOSPITAL CORPORATION D B A MASSACHUSETTS GENERAL HOSPITAL, YOUNG Daniel W, November 21, 2013: WO/2013/173652 (10 worldwide citation)

[en] Aspects of the invention provide methods for selecting a candidate oligonucleotide for activating expression of a target gene. Further aspects of the invention provide methods of selecting a set of oligonucleotides that is enriched in oligonucleotides that activate expression of a target gene. ...


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KRIEG Arthur M, SUBRAMANIAN Romesh, MCSWIGGEN James, LEE Jeannie T: [fr] COMPOSITIONS ET MÉTHODES POUR MODULER LEXPRESSION GÉNIQUE, [en] COMPOSITIONS AND METHODS FOR MODULATING GENE EXPRESSION. RANA THERAPEUTICS, THE GENERAL HOSPITAL CORPORATION D B A, YOUNG Daniel W, November 21, 2013: WO/2013/173637 (9 worldwide citation)

[en] Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of a target gene. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of a target gene. Methods for modulating ex ...


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KRIEG Arthur M, SUBRAMANIAN Romesh, MCSWIGGEN James, LEE Jeannie T: [fr] COMPOSITIONS ET PROCÉDÉS DE MODULATION DE LEXPRESSION DE LA FAMILLE GÉNIQUE SMN, [en] COMPOSITIONS AND METHODS FOR MODULATING SMN GENE FAMILY EXPRESSION. RANA THERAPEUTICS, THE GENERAL HOSPITAL CORPORATION d b a MASSACHUSETTS GENERAL HOSPITAL, YOUNG Daniel W, November 21, 2013: WO/2013/173638 (9 worldwide citation)

[en] Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMNl or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMNl or SMN2 that comprises exon 7. Metho ...


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KRIEG Arthur M, SUBRAMANIAN Romesh, MCSWIGGEN James, LEE Jeannie T: [fr] COMPOSITIONS ET MÉTHODES POUR MODULER LEXPRESSION DE LA FAMILLE MULTIGÉNIQUE DE LHÉMOGLOBINE, [en] COMPOSITIONS AND METHODS FOR MODULATING HEMOGLOBIN GENE FAMILY EXPRESSION. RANA THERAPEUTICS, THE GENERAL HOSPITAL CORPORATION d b a MASSACHUSETTS GENERAL HOSPITAL, YOUNG Daniel W, November 21, 2013: WO/2013/173599 (8 worldwide citation)

[en] Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of hemoglobin genes (HBB, HBD, HBEl, HBG1 or HBG2). Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of hemogl ...


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KRIEG Arthur M, SUBRAMANIAN Romesh, MCSWIGGEN James, LEE Jeannie T: [fr] COMPOSITIONS ET MÉTHODES POUR MODULER LEXPRESSION DE PTEN, [en] COMPOSITIONS AND METHODS FOR MODULATING PTEN EXPRESSION. RANA THERAPEUTICS, THE GENERAL HOSPITAL CORPORATION DB A MASSACHUSETTS GENERLA HOSPITAL, YOUNG Daniel W, November 21, 2013: WO/2013/173605 (8 worldwide citation)

[en] Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of PTEN. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of PTEN. Methods for modulating expression of PTEN u ...


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Eugen Uhlmann, Markus Weber, Romesh Subramanian, Thomas Dino Rockel, Arthur M Krieg: Multimeric oligonucleotides compounds. RaNA Therapeutics, Wolf Greenfield & Sacks P C, February 28, 2017: US09580708 (3 worldwide citation)

The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleot ...


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Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang: Cleavable lipids. RaNA Therapeutics, Proskauer Rose, Fangli Chen, August 1, 2017: US09717690 (2 worldwide citation)

Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent tran ...


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Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith: CFTR mRNA compositions and related methods and uses. RaNA Therapeutics, Ethris, Fangli Chen, Proskauer Rose, July 25, 2017: US09713626

Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising th ...


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