The University of Western Australia

Murdoch University

Université De Mons

Yissum Research Development Company of the Hebrew University of Jerusalem Ltd.

Beijing University of Technology

Sarepta Therapeutics, Inc.

Research Institute at Nationwide Children's Hospital

Stephen Donald Wilton

Sue Fletcher

Graham McClorey

Abbie Adams

Penny Meloni

Celine Vanderplanck

Eugenie Ansseau

Alexandra Belayew

Frederique Coppee

Carlos Rosado

Raelene Jane Pickering

Merlin Christopher Thomas

Batsheva Kerem

Michal Tur Sinai

Loren Price

Gunnar J Hanson

Richard Keith Bestwick

Nicolas Wein

Kevin Flanigan

May Aung-Htut

Archa Fox

Ruohan Li

Céline Vanderplanck

Eugénie Ansseau

Frédérique COPPÉE

Graham Mcclorey

Antisense agents and RNA interference agents useful for treating diseases and conditions the treatment of which can benefit from reducing the expression of double homeobox 4 and/or double homeobox 4c, more particularly facioscapulohumeral muscular dystrophy. Methods, uses and further products employing such agents are also described.

Agents useful in treating facioscapulohumeral muscular dystrophy

An isolated or purified AON for modifying pre-mRNA splicing in the Receptor for Advanced Glycation End-products (RAGE) to modulate splicing of the RAGE gene transcript or part thereof is provided.

Modulators and modulation of the receptor for advanced glycation end-products RNA

An isolated or purified antisense oligomer for modifying RNA cleavage and processing in the NEAT1 gene transcript or part thereof.

Treatment for NEAT1 associated disease

An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 59.

Antisense molecules and methods for treating pathologies

An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 202.

Antisense oligonucleotides for inducing exon skipping and methods of use thereof

The present disclosure relates to antisense oligomers and related compositions and methods for inducing exon inclusion as a treatment for glycogen storage disease type II (GSD-II) (also known as Pompe disease, glycogenosis II, acid maltase deficiency (AMD), acid alpha-glucosidase deficiency, and lysosomal alpha-glucosidase deficiency), and more specifically relates to inducing inclusion of exon 2 and thereby restoring levels of enzymatically active acid alpha-glucosidase (GAA) protein encoded by the GAA gene.

Antisense-induced exon2 inclusion in acid alpha-glucosidase

The present invention relates to the delivery of oligomers for treating patients with a 5' mutation in their DMD gene other than a DMD exon 2 duplication. The invention provides methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene resulting in translation of a functional truncated isoform of dystrophin. The methods and materials can be used for the treatment of muscular dystrophies arising from 5&#x2032; mutations in the DMD gene such as Duchenne Muscular Dystrophy or Becker Muscular Dystrophy.

Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene

An antisense molecule capable of binding to a selected target site to induce exon skipping in the dystrophin gene, as set forth in SEQ ID NO: 1 to 214.

Antisense agents and RNA interference agents useful for treating diseases and conditions the treatment of which can benefit from reducing the expression of double homeobox 4 and/or double homeobox 4c, more particularly facioscapulohumeral muscular dystrophy, are described. Methods, uses and further products employing such agents are also described.

Provided are oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, compositions including said oligonucleotides, kits including the compositions, and uses thereof. In particular, the subject matter provides compositions of oligonucleotides useful in methods for suppressing exon skipping optionally in combination with additional CFTR therapeutics.

Restoration of the CFTR function by splicing modulation

Antisense molecules capable of binding to a selected target site in the dystrophin gene to induce exon skipping are described.

Growing community of inventors

Applecross, Australia

Stephen Donald Wilton