Medicine and Science from The BMJ

CRISPR, stemcell banking, and a football world cup

13 snips
Aug 21, 2025
Kenneth Charles, a consultant haematologist from the University of the West Indies, discusses the revolutionary CRISPR therapy approved by NICE for sickle cell disease and the challenges it presents. Investigative journalist Emma Wilkinson exposes misleading claims by companies offering to bank children's teeth for stem cells, revealing a lack of evidence behind their promises. Lastly, Jamie Thorogood, captain of the Great Britain men's medical football team, celebrates their recent victory at the Medics World Cup, highlighting the blend of sports and healthcare.
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INSIGHT

How CRISPR Treats Sickle Cell

  • CRISPR for sickle cell raises fetal hemoglobin by editing patients' stem cells to produce hemoglobin F.
  • The procedure requires high-dose chemotherapy and intensive support during marrow ablation and engraftment.
INSIGHT

CRISPR Versus Established Therapies

  • Existing treatments like hydroxyurea and donor stem cell transplants also raise fetal hemoglobin or can be curative but have trade-offs.
  • CRISPR's uniqueness is targeted editing without a donor but shares intense chemo and engraftment risks.
ADVICE

Explain Risks And Focus On Prevention

  • Clinicians must communicate the significant risks of high-dose chemotherapy, including infertility and secondary cancers.
  • Prioritise preventing tissue damage so more patients remain eligible for gene therapies like CRISPR.
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