Curriculum
Module 15 · 65 min
Stem Cells, Development & Regeneration
Pluripotency, niches, organoids, and what cell therapy can — and cannot — yet do.
CoreClinicalResearch
Topics
What this module covers
- 01Embryonic vs adult vs induced pluripotent stem cells
- 02Stem-cell niche concept; HSC, intestinal Lgr5+, neural, mesenchymal
- 03Reprogramming: OSKM and partial reprogramming
- 04Organoids and assembloids
- 05Allogeneic vs autologous cell therapy
- 06What is approved, what is experimental, what is fraud
Deep dives
Lesson sub-pages
Learning objectives
By the end of this module you will be able to
- L01Distinguish potency states (totipotent, pluripotent, multipotent, unipotent).
- L02Critique a typical 'stem cell clinic' offering and explain its risks.
- L03Outline the manufacturing chain for an autologous CAR-T therapy.
Expected takeaways
What you should walk away believing
- →Hematopoietic stem cell transplant is the established cell therapy; CAR-T is the second.
- →Most 'stem-cell clinic' offerings outside HSCT and CAR-T are unproven and often unsafe.
- →Organoids enable patient-specific drug testing but do not yet replicate full tissue physiology.
Core summary
At the Core level
Stem cells self-renew and differentiate; their behaviour depends on intrinsic transcriptional state and the niche. Pluripotency can be induced from fibroblasts (Yamanaka, 2006) but full clinical realisation has been slower than predicted — a few approved therapies exist, many trials are ongoing, and many marketed treatments are unjustified.
Myth vs reality
Common misconception
Claim
IV mesenchymal stem cells treat orthopaedic, cardiac and neurologic disease.
Reality
MSCs are largely immunomodulatory and rarely engraft. Most marketed indications lack RCT support; many countries have prosecuted clinics for fraud.
Evidence-graded claims
Claims, scored A–F
B
iPSCs and ESCs are functionally equivalent for most uses
Some residual epigenetic memory; but most assays show strong equivalence.
B
Casgevy (CRISPR-edited HSC) cures sickle cell disease
Sustained benefit in long-term follow-up; long-term safety still accruing.
F
IV MSC therapy reverses spinal cord injury
No controlled evidence; many predatory offerings.
Quiz
Check your understanding
Q1. Yamanaka factors are:
Q2. Casgevy is a treatment for:
Flashcards
Lock it in
1 / 3
Front
Stem-cell niche?
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Suggested reading
Primary literature
- Induced pluripotent stem cells from adult human fibroblasts — Takahashi et al., Cell 2007 ↗
- Exagamglogene autotemcel for severe sickle cell disease — Frangoul et al., NEJM 2024 ↗