Stem cells translational medicineSep 18, 2025

Gene-Modified Blood Stem Cell Therapy for Sickle Cell Disease

Regenerative Health Weekly Brief ↗PubMed ↗DOI ↗

Updated Jan 7, 2026

Abstract

Hematopoietic stem cells (HSCs) can be genetically corrected to potentially provide a one-time cure for sickle cell disease (SCD).

SCD is one of the most common single-gene disorders, making it a significant target for gene therapy.
Current treatments for SCD, such as hydroxyurea, require lifelong use.
Allogeneic HSC transplantation offers a curative option but is limited by the availability of compatible donors.
Autologous HSC gene therapy uses patients' own stem cells, potentially expanding treatment accessibility.
In vivo HSC gene therapy is being developed to enable a single-injection delivery method for gene correction.
The review covers various therapeutic approaches, including both established and novel gene therapies for SCD.

Simplified

Hematopoietic stem cells (HSCs) reconstitute blood cells throughout life. DNA-level correction of HSCs allows for a one-time cure of genetic diseases, including sickle cell disease (SCD). Sickle cell disease is one of the most common single-gene disorders; therefore, SCD is a prime candidate for gene therapy. Several drug therapies are available for SCD, including hydroxyurea, which is the first-line choice despite requiring lifelong administration. Allogeneic HSC transplantation is a one-time, curative treatment for SCD with limited availability of histocompatible donors. Therefore, autologous HSC gene therapy was developed using patients' own HSCs with lentiviral gene addition/silencing and clustered regularly interspaced short palindromic repeats gene editing, making gene therapy applicable to most patients. However, the established method of HSC gene therapy requires costly and complex ex vivo HSC culture. Therefore, in vivo HSC gene therapy is being developed to treat SCD, envisioning a single-injection HSC-targeted gene delivery system. This review discusses various therapeutic methods to treat SCD, the development of HSC gene therapy, and clinical gene therapy trials in SCD, ranging from FDA-approved to novel in vivo gene therapy.

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Funding

Competing interests

The authors declared no potential conflicts of interest.

PubMed

Gene-Modified Blood Stem Cell Therapy for Sickle Cell Disease

Abstract

Full Text

Funding

Competing interests

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