The University of Western Australia

Murdoch University

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

Sarepta Therapeutics, Inc.

Sue Fletcher

Stephen Donald Wilton

Graham McClorey

Abbie Adams

Penny Meloni

Batsheva Kerem

Michal Tur Sinai

Loren Price

Gunnar J Hanson

Richard Keith Bestwick

May Aung-Htut

Stephen Donald Wilson

Archa Fox

Ruohan Li

Graham Mcclorey

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

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.

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.

Oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, compositions including the oligonucleotides, kits including the compositions, and uses thereof. Compositions of oligonucleotides useful in methods for suppressing exon 10 skipping optionally in combination with additional CFTR therapeutics.

An antisense oligomer capable of binding to a selected target on the ITGA4 gene transcript to modify pre-mRNA splicing in an ITGA4 gene transcript or part thereof.

Growing community of inventors

Bayswater, Australia

Sue Fletcher