专利检索 ap:("The General Hospital Corporation") AND inv:"Hun-goo Lee" 第 1 页

1.

发明公开
Compositions and Methods for the Treatment of Conditions Associated with Nucleotide Repeat Expansion 审中-公开

公开(公告)号：US20240173358A1

公开(公告)日：2024-05-30

申请号：US18282421

申请日：2022-03-16

申请人： The General Hospital Corporation

发明人： Jeannie T. Lee , Hun-goo Lee

IPC分类号： A61K35/545 , A61K48/00 , C12N9/22 , C12N15/11 , C12N15/90

CPC分类号： A61K35/545 , A61K48/005 , C12N9/22 , C12N15/11 , C12N15/907 , C12N2310/20 , C12N2800/80

摘要： Described herein are compositions and methods that can be used to contract nucleotide repeat expansion (e.g., CGG) in a gene (e.g., FMR1) in cells of subjects having a condition associated with nucleotide repeat expansion, e.g., subjects with Fragile X syndrome (FXS), FXTAS-Parkinsonism, myotonic dystrophy, and other repeat disorders, by treating the cells using an inactive Cas9 protein and a guide RNA that directs the Cas9 to the gene or by treating the cells using a reactivation cocktail comprising small molecules.