Today, I review, link to, and excerpt from The Cribsiders‘ #163: A Hemoglobin Makeover: Curative and Transformative Therapies for SCD.*

*Chisholm JC, Rahim M, Chiu C, Masur S. “#163 A Hemoglobin Makeover: Curative and Transformative Therapies for SCD”. The Cribsiders Pediatric Podcast. https:/www.thecribsiders.com/ December 24, 2025.

All that follows is from the above resource.

Join us in this episode with expert guest Dr. Mahvish Rahim (the Children’s Hospital at Montefiore) as we dive into the rapidly evolving world of curative and transformative therapies for sickle cell disease! From breaking down the science behind stem cell transplant and gene therapy to understanding how to choose the right candidates and counsel families, Dr. Rahim brings clarity to this exciting new era in SCD care.

Gene therapy and BMT for SCD: Pearls

1. There are two types of potentially curative/transformative treatments for children with sickle cell disease: bone marrow transplant (BMT) which is also referred to as stem cell transplant or gene therapy (GT).
2. Gene therapy and BMT both involve first going through chemotherapy to destroy the existing stem cells in the bone marrow, this is called conditioning chemotherapy.
3. Key differences between BMT and GT for sickle cell disease:

• • BMT requires donor stem cells from a HLA matched donor, a full sibling has a 25% chance of being a ‘perfect’ HLA match.
  • GT is an option even if a donor isn’t available as it uses a patient’s own cells.
  • BMT has been an option to cure SCD for decades.  GT is a new option.
  • GT currently is only available to children who are  ≥12 years old.
  • One risk of BMT is graft-versus-host-disease, GT does not carry this risk.
  • Patients who have GT will still have higher than normal levels of hemoglobin S, patients who successfully undergo BMT will not.

BMT and Gene Therapy for SCD Notes