METHOD FOR TREATING PROGEROID LAMINOPATHY USING OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA

摘要

To provide a method for treating progeroid laminopathy using oligonucleotide analogues targeting human LMNA.SOLUTION: The invention provides a method of treatment of a subject having progeroid diseases and related conditions, based on LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin. The present invention relates generally to a method of treating progeroid laminopathy including Hutchinson-Gilford progeria syndrome, using human lamin A targeted antisense compounds and related compositions.SELECTED DRAWING: None