Patent ID: 11920150
Assignee: PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Field: Pharmaceuticals (Chemistry)
Classification: CPC A  C | IPC A  C

Claim 25:
26. The cell, pharmaceutical formulation, or viral particle of claim 24, wherein the expanded repeat disease is Huntington's disease, a Myotonic Dystrophy, or Facioscapulohumeral muscular dystrophy (FSHD), wherein the muscular dystrophy is Duchene muscular dystrophy, Becker Muscular dystrophy, a Limb-Girdle muscular dystrophy, an Emery Dreifuss muscular dystrophy, a myotonic dystrophy, or FSHD, optionally wherein the myotonic dystrophy is a Type 1 or a Type 2 myotonic dystrophy, wherein the cardiomyopathy is dilated cardiomyopathy, hypertrophic cardiomyopathy, Duchene muscular dystrophy-associated cardiomyopathy, or Dannon disease, wherein the sugar or glycogen storage disease is a MPS type III disease or Pompe disease, optionally wherein the MPS type III disease, is MPS Type IIIA, IIIB, IIIC, or IIID, wherein the neuro-muscular disease is Charcot-Marie-Tooth disease or Friedreich's Ataxia, or any combination thereof.