Ding Sheng
New Cornerstone Science Laboratory, School of Pharmaceutical Sciences, Tsinghua University, Beijing 100084, China.
Tsinghua-Peking Joint Center for Life Sciences, Tsinghua University, Beijing 100084, China.
Chem Rev. 2025 Feb 26;125(4):1805-1822. doi: 10.1021/acs.chemrev.4c00332. Epub 2025 Feb 5.
Therapeutic reprogramming represents a transformative paradigm in regenerative medicine, developing new approaches in cell therapy, small molecule drugs, biologics, and gene therapy to address unmet medical challenges. This paradigm encompasses the precise modulation of cellular fate and function to either generate safe and functional cells for cell-based therapies or to directly reprogram endogenous cells or for tissue repair and regeneration. Building on the discovery of induced pluripotent stem cells (iPSCs), advancements in chemical modulation and CRISPR-based gene editing have propelled a new iterative medicine paradigm, focusing on developing scalable, standardized cell therapy products from universal starting materials and enabling iterative improvements for more effective therapeutic profiles. Beyond cell-based therapies, non-cell-based therapeutic strategies targeting endogenous cells may offer a less invasive, more convenient, accessible, and cost-effective alternative for treating a broad range of diseases, potentially rejuvenating tissues and extending healthspan.
治疗性重编程是再生医学中的一种变革性范式，它在细胞治疗、小分子药物、生物制品和基因治疗等方面开发新方法，以应对尚未满足的医学挑战。这种范式包括对细胞命运和功能的精确调控，从而为基于细胞的疗法生成安全且功能正常的细胞，或直接对内源性细胞进行重编程以实现组织修复和再生。基于诱导多能干细胞（iPSC）的发现，化学调控和基于CRISPR的基因编辑方面的进展推动了一种新的迭代医学范式，其重点是从通用起始材料开发可扩展、标准化的细胞治疗产品，并实现迭代改进以获得更有效的治疗效果。除了基于细胞的疗法外，针对内源性细胞的非细胞治疗策略可能为治疗多种疾病提供一种侵入性较小、更方便、可及且具成本效益的替代方案，有可能使组织恢复活力并延长健康寿命。
