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Skeletal Muscle Stem Cells Derived from Teratomas

来自畸胎瘤的骨骼肌干细胞

基本信息

批准号：
9763683
负责人：
Michael Kyba
金额：
$ 42.78万
依托单位：
UNIVERSITY OF MINNESOTA
依托单位国家：
美国
项目类别：
财政年份：
2019
资助国家：
美国
起止时间：
2019-05-01 至 2024-04-30
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/9763683
关键词：
Address Advanced Development Cell Differentiation process Cell Therapy Cells Data Development Environment Fiber Generations Glean Histologic Human In Vitro Knowledge Label Literature Maps Mediating Methods Mus Muscle Muscle Fibers Muscle satellite cell Myopathy Natural regeneration Pluripotent Stem Cells Population Protocols documentation Public Health Regenerative Medicine Risk Series Signal Transduction Signaling Protein Skeletal Development Skeletal Muscle Stem cells Surface System Teratoma Time Tissues Translating Transplantation base human pluripotent stem cell improved in vivo in vivo regeneration induced pluripotent stem cell insight muscle regeneration muscle transplantation novel progenitor regenerative regenerative therapy single cell sequencing single-cell RNA sequencing stem cell differentiation success tibialis anterior muscle tool

项目摘要

Abstract Pluripotent stem cells hold tremendous promise for the eventual development of cell therapies for muscle disease; however it has proven exceptionally difficult to derive cells with meaningful in vivo regeneration potential from unmodified wild type (WT) pluripotent stem cells. Mouse transplantation studies with WT PSC- derived cells are rare, and success in the field currently equates to generation of a few hundred fibers in a transplanted tibialis anterior muscle with 3-4,000 total fibers. We have recently discovered that when mouse PSCs are differentiated into teratomas in vivo, they generate large numbers of skeletal muscle stem cells, and these cells have tremendous in vivo functional potential, regenerating the great majority of the TA muscle with force-generating muscle fibers upon transplantation. We propose a series of studies aimed at understanding how these cells arise, what makes them unique, translating these findings to the human system, and using this knowledge to improve in vitro differentiation protocols.

摘要多能干细胞为肌肉细胞疗法的最终发展带来了巨大的希望疾病；然而，事实证明，获得有意义的体内再生细胞极其困难来自未经修饰的野生型(WT)多能干细胞的潜力。WT-PSC-小鼠移植研究衍生细胞很少见，目前在这一领域的成功相当于在一年中产生数百根纤维移植的胫前肌总纤维数为3~4,000。我们最近发现，当老鼠 PSCs在体内分化为畸胎瘤，产生大量的骨骼肌干细胞，这些细胞在体内具有巨大的功能潜力，可以再生绝大多数TA肌肉移植后产生力量的肌肉纤维。我们提出了一系列研究，旨在了解这些细胞是如何产生的，是什么使它们独一无二，将这些发现转化为人类系统，并使用改进体外分化方案的知识。