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Published on: November 5, 2019
Sickle Cell Disease: Historical Overview and Current Therapies
Oluwaseun O Babatunde1, Madeleine G Bibby1, Anthony Atala1
1Wake Forest Institute for Regenerative Medicine, Winston-Salem, North Carolina, USA.
Prenatal Diagnosis
|June 11, 2026
Summary
Sickle cell disease (SCD) affects millions globally. In utero transplantation (IUTx) offers a potential cure before birth, avoiding current treatment limitations and risks.
Area of Science:
- Hematology
- Genetics
- Pediatric Medicine
Background:
- Sickle cell disease (SCD) is a prevalent genetic disorder with limited therapeutic options.
- Current treatments, including hematopoietic stem cell (HSC) transplantation, have significant risks and side effects.
- SCD manifestations begin in infancy, posing a risk of irreversible organ damage.
Purpose of the Study:
- To review the history and current limitations of SCD treatments.
- To introduce in utero transplantation (IUTx) as a potential curative therapy for SCD.
- To highlight the benefits of treating SCD before birth.
Main Methods:
- Literature review of SCD history and treatments.
- Conceptual overview of in utero transplantation (IUTx) for SCD.
- Analysis of current therapeutic challenges and unmet needs.
Main Results:
- Existing SCD treatments are insufficient and carry substantial risks.
- Hematopoietic stem cell (HSC) transplantation is curative but challenging.
- In utero transplantation (IUTx) presents a novel approach to correct SCD prenatally.
Conclusions:
- There is an urgent need for novel, curative SCD therapies.
- In utero transplantation (IUTx) could prevent disease onset and lifelong complications.
- Prenatal treatment of SCD offers a promising alternative to postnatal interventions.
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