首页> 外国专利> MEGAKARYOCYTIC PARTICLES AND MICROPARTICLES FOR CELL THERAPY amp; FATE MODIFICATION OF STEM AND PROGENITOR CELLS

MEGAKARYOCYTIC PARTICLES AND MICROPARTICLES FOR CELL THERAPY amp; FATE MODIFICATION OF STEM AND PROGENITOR CELLS

机译:巨核细胞颗粒和微颗粒用于干细胞和祖细胞的细胞治疗和命运修饰

摘要

Applications in transfusion medicine requiring platelets, and hematopoietic stem-cell transplantations require either platelets or enhancement of in vivo platelet biogenesis. Gene therapy applications of hematopoietic stem and progenitor cells (HSPCs) require effective and specific modification of HSPCs by DNA, RNA or other biological molecules. Here we disclose methods for the generation, and modification of megakaryocytic microparticles (MkMPs) or microvesicles, that can be used in the aforementioned transfusion and transplantation medicine applications and in gene therapy applications involving hematopoietic stem cells. The biological effects of modified or unmodified MkMPs have never been previously disclosed and thus, this invention claims all biological applications of MkMPs in in vivo therapeutic applications or ex vivo applications to produce various cells and cell parts, modify various target cells or deliver molecules including drugs to HSPCs and related cells.
机译:在需要血小板的输血医学和造血干细胞移植中的应用需要血小板或体内血小板生物发生的增强。造血干细胞和祖细胞(HSPC)的基因治疗应用需要通过DNA,RNA或其他生物分子对HSPC进行有效和特异性的修饰。在这里,我们公开了用于巨核细胞微粒(MkMP)或微囊泡的产生和修饰的方法,其可用于上述输血和移植医学应用以及涉及造血干细胞的基因治疗应用中。修饰或未修饰的MkMP的生物学作用以前从未公开过,因此,本发明要求MkMP在体内治疗应用或离体应用中的所有生物学应用,以产生各种细胞和细胞部分,修饰各种靶细胞或递送包括药物的分子。 HSPC和相关细胞。

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