Thomas Justin, Zimmerlin Ludovic, Huo Jeffrey S, Considine Michael, Cope Leslie, Zambidis Elias T
Institute for Cell Engineering, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Sidney Kimmel Comprehensive Cancer Center, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.
NPJ Regen Med. 2021 May 17;6(1):25. doi: 10.1038/s41536-021-00135-1.
Human pluripotent stem cells (hPSCs) can generate specialized cell lineages that have great potential for regenerative therapies and disease modeling. However, the developmental stage of the lineages generated from conventional hPSC cultures in vitro are embryonic in phenotype, and may not possess the cellular maturity necessary for corrective regenerative function in vivo in adult recipients. Here, we present the scientific evidence for how adult human tissues could generate human-animal interspecific chimeras to solve this problem. First, we review the phenotypes of the embryonic lineages differentiated from conventional hPSC in vitro and through organoid technologies and compare their functional relevance to the tissues generated during normal human in utero fetal and adult development. We hypothesize that the developmental incongruence of embryo-stage hPSC-differentiated cells transplanted into a recipient adult host niche is an important mechanism ultimately limiting their utility in cell therapies and adult disease modeling. We propose that this developmental obstacle can be overcome with optimized interspecies chimeras that permit the generation of adult-staged, patient-specific whole organs within animal hosts with human-compatible gestational time-frames. We suggest that achieving this goal may ultimately have to await the derivation of alternative, primitive totipotent-like stem cells with improved embryonic chimera capacities. We review the scientific challenges of deriving alternative human stem cell states with expanded embryonic potential, outline a path forward for conducting this emerging research with appropriate ethical and regulatory oversight, and defend the case of why current federal funding restrictions on this important category of biomedical research should be liberalized.
人类多能干细胞(hPSCs)能够产生具有再生治疗和疾病建模巨大潜力的特化细胞谱系。然而,体外常规hPSC培养所产生的谱系在表型上处于胚胎发育阶段,可能不具备成年受体体内矫正性再生功能所需的细胞成熟度。在此,我们展示了成体人类组织如何产生人 - 动物种间嵌合体以解决这一问题的科学证据。首先,我们回顾了通过体外常规hPSC以及类器官技术分化出的胚胎谱系的表型,并比较它们与正常人类子宫内胎儿及成人发育过程中产生的组织的功能相关性。我们推测,移植到成年受体宿主微环境中的胚胎阶段hPSC分化细胞的发育不一致性是最终限制其在细胞治疗和成人疾病建模中效用的重要机制。我们提出,可以通过优化种间嵌合体来克服这一发育障碍,这种嵌合体能够在与人类兼容的妊娠期内,在动物宿主体内产生成年阶段、患者特异性的全器官。我们认为,实现这一目标可能最终不得不等待具有更高胚胎嵌合能力的替代性原始全能样干细胞的获得。我们回顾了获得具有扩展胚胎潜能的替代性人类干细胞状态的科学挑战,勾勒出在适当的伦理和监管监督下开展这项新兴研究的前进道路,并为放宽当前联邦政府对这一重要生物医学研究类别的资金限制的理由进行辩护。