OLIGONUCLEOTIDE THERAPY FOR WOLMAN DISEASE AND CHOLESTERYL ESTER STORAGE DISEASE

摘要: The present disclosure provides antisense oligonucleotides, compositions, and methods that target a LIPA intron flanking exon 8, thereby modulating splicing of pre-mRNA to increase the level mRNA molecules having e.g., provide therapy for Wolman Disease or Cholesteryl Ester Storage Disease. an oligonucleotide including nucleobase sequence at least 70% complementary in 5'-flanking intron, 3'-flanking combination 8 intron.