Title:
MATERIALS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY
Document Type and Number:
Japanese Patent JP2023037643
Kind Code:
A
Abstract:
To provide materials and methods for treating Duchenne muscular dystrophy.SOLUTION: This application provides materials and methods for treating a patient with Duchenne Muscular Dystrophy (DMD) both ex vivo and in vivo. In addition, this application provides materials and methods for editing a dystrophin gene in a cell by genome editing. The present disclosure presents an approach to address the genetic basis of DMD. By using genome engineering tools to create permanent changes to the genome that can restore the dystrophin reading frame and restore the dystrophin protein activity with as few as a single treatment, the resulting therapy can correct the underlying genetic defect causing the disease.SELECTED DRAWING: None
Inventors:
Ami Medha Kabaddi
Chad Albert Cowan
Ante Sven Lundberg
Chad Albert Cowan
Ante Sven Lundberg
Application Number:
JP2022212927A
Publication Date:
March 15, 2023
Filing Date:
December 14, 2022
Export Citation:
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
International Classes:
C12N15/09; A61K9/14; A61K31/7105; A61K35/34; A61K35/76; A61K38/46; A61K47/44; A61K48/00; A61P21/00; C12N5/077; C12N5/10; C12N15/11; C12N15/12; C12N15/55; C12N15/864; C12N15/88
Attorney, Agent or Firm:
Shusaku Yamamoto
Morishita Natsuki
Morishita Natsuki
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