Title:
NEW THERAPY FOR POMPE DISEASE
Document Type and Number:
WIPO Patent Application WO/2019/050406
Kind Code:
A8
Abstract:
The invention relates to methods for gene therapy in a subject suffering from Pompe disease, comprising gene-editing of a glucosidase, acid, alpha gene (GAA) in said subject. The invention further relates to a cell culture of genetically changed, differentiated myogenic progenitor cells derived from a donor subject suffering from Pompe disease, to a vector for use in a method for gene-editing a eukaryotie cell, and to a myotube prepared from myogenic progenitor cells that have been genetically changed by gene-editing.
Inventors:
PIJNAPPEL WILHELMUS WENCESLAUS MATTHIAS (NL)
VAN DER PLOEG ANTJE TJITSKE (NL)
VAN DER WAL ERIK (NL)
KRUIJT JUR (NL)
HERRERO HERNADEZ PABLO (NL)
VAN DER PLOEG ANTJE TJITSKE (NL)
VAN DER WAL ERIK (NL)
KRUIJT JUR (NL)
HERRERO HERNADEZ PABLO (NL)
Application Number:
PCT/NL2018/050581
Publication Date:
April 02, 2020
Filing Date:
September 07, 2018
Export Citation:
Assignee:
UNIV ERASMUS MED CT ROTTERDAM (NL)
International Classes:
A61K48/00; C12N9/24; C12N15/63
Attorney, Agent or Firm:
WITMANS, H.A. (NL)
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