Document Type

Article

Publication Date

5-1-2025

Keywords

JMG, Animals, Anemia, Sickle Cell, Mice, Disease Models, Animal, Humans, Genetic Variation

JAX Source

Trends Mol Med. 2025;31(5):413-23.

ISSN

1471-499X

PMID

39643521

DOI

https://doi.org/10.1016/j.molmed.2024.11.004

Grant

This work is partially supported by National Institutes of Health (NIH) grant R00-DK129705 (R.A.).

Abstract

A milestone in sickle cell disease (SCD) therapeutics was achieved in December 2023 with the FDA-approved gene therapy for patients aged 12 years and older. However, these therapies may only suit a fraction of patients because of cost or health risks. A better understanding of SCD outcome heterogeneity is needed to propose patient-specific pharmacological interventions. To achieve this, humanized and genetically diverse mouse models are essential for associating candidate genotypes with specific hematological traits, organ function, and disease resilience. Here, we discuss the challenges and opportunities in developing genetically diverse sickle cell mice (GDS mice). These models are expected to complement current approaches in SCD research and enhance our understanding of SCD heterogeneity and anemia.

Creative Commons License

Creative Commons Attribution 4.0 International License
This work is licensed under a Creative Commons Attribution 4.0 International License.

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