Sarepta Therapeutics, Inc.

National Institutes of Health, a Component of the US Dept. of Health & Human Services

University System of Maryland

Progeria Research Foundation, Inc.

The Scripps Research Institute

Avi Biopharma, Inc.

Murdoch University

Richard Keith Bestwick

Ryszard M Kole

Diane Elizabeth Frank

Patrick L Iversen

Frederick Joseph Schnell

Dwight D Weller

Francis S Collins

David A Stein

Leslie B Gordon

Baozhong Cai

Katie Thomas

Mitchell Martini

Ross Shimabuku

Kan Cao

Michael R Erdos

Gunnar J Hanson

Stephen Donald Wilton

Sue Fletcher

Hemant Kumar Roy

Bao Zhong Cai

Benjamin Neuman

Michael Buchmeier

Fred Joseph Schnell

Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 44 skipping are described.

Exon skipping compositions for treating muscular dystrophy

Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.

Oligonucleotide analogues targeting human LMNA

Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

Exon skipping oligomer conjugates for muscular dystrophy

Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomer synthesis while maintaining overall yield and purity of a synthesized oligomer.

Processes for preparing oligomers

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 disclosure relates to antisense oligomers and related compositions and methods for decreasing the expression of functional human myostatin and methods for treating muscular dystrophy and related disorders and more specifically relates to inducing exclusion of myostatin exon 2 and thereby reducing the levels of myostatin protein.

Antisense-induced exon exclusion in myostatin

Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomersynthesis while maintaining overall yield and purity of a synthesized oligomer.

Growing community of inventors

Corvallis, OR, United States of America

Richard Keith Bestwick