Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA

US9833468B2 · kind B2 · utility

Assignees

• Sarepta Therapeutics, Inc. · US
• The United States of America as Represented by the Secretary, Dept. of Health and Human Services · US
• University of Maryland · US

Inventors

• Ryszard Kole · Chapel Hill, US
• Francis S. Collins · Rockville, US
• Michael R. Erdos · Bethesda, US
• Kan Cao · Bowie, US

Key dates

• Filing date: Mar 29, 2016
• Grant date: Dec 5, 2017
• Expiry date: Mar 29, 2036

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2320/33
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

Provided are methods of treatment in subjects having progeroid diseases and related conditions which rely upon LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.