公开(公告)号：US20230203448A1

公开(公告)日：2023-06-29

申请号：US18009231

申请日：2021-06-07

申请人： Erasmus University Medical Center Rotterdam ,  Academisch Ziekenhuis Leiden h.o.d.n. LUMC ,  Wageningen Universiteit

发明人： Alessandro IULIANO ,  Wilhelmus Wenceslaus Matthias PIJNAPPEL ,  Antje Tjitske VAN DER PLOEG ,  Vittorio SAGGIOMO ,  Jessica Christine DE GREEF ,  Erik VAN DER WAL ,  Silvere Maria VAN DER MAAREL

IPC分类号： C12N5/077 ,  C12M3/00 ,  C12M3/06 ,  C12M1/12

CPC分类号： C12N5/0658 ,  C12M21/08 ,  C12M23/16 ,  C12M25/14 ,  C12N2501/115 ,  C12N2501/727 ,  C12N2513/00 ,  B33Y10/00

摘要： A method of manufacturing a microstructure comprises printing a positive mold structure, filling the positive mold structure with a second material to form an elastically deformable negative mold structure, filling the negative mold structure with a third material to form the microstructure, and releasing the microstructure from the negative mold structure. Advantageously, the negative mold structure can be stretched to facilitate the release of the microstructure. For example, the microstructure comprises a chamber with capped micropillars for the generation and/or analysis of muscle tissue.

公开(公告)号：US20200276334A1

公开(公告)日：2020-09-03

申请号：US16645561

申请日：2018-09-07

申请人： Erasmus University Medical Center Rotterdam

发明人： Wilhelmus Wenceslaus Matthias PIJNAPPEL ,  Antje Tjitske VAN DER PLOEG ,  Erik VAN DER WAL ,  Jur KRUIJT ,  Pablo HERRERO HERNANDEZ

IPC分类号： A61K48/00 ,  C12N15/85 ,  C12N5/074 ,  A61K35/12 ,  A61K9/00

摘要： 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 eukaryotic cell, and to a myotube prepared from myogenic progenitor cells that have been genetically changed by gene-editing.