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December 8, 2025Blood

Engineered extracellular vesicles for In Vivo gene therapy in sickle cell disease

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Authors

RBR. E. BlackUniversity of Maryland, Baltimore SJSangmoo JeongJohns Hopkins University

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Implication

This approach shows enhanced delivery of ribonucleoprotein complexes to HSPCs in sickle cell disease, suggesting a more effective gene therapy method.

Key Points

Develop cost-effective engineered extracellular vesicles for in vivo gene therapy targeting sickle cell disease.
Designed extracellular vesicles to deliver CRISPR-Cas9 ribonucleoprotein complexes for BCL11A gene silencing.
Engineered HEK293T cells to overexpress CD63 and provide enhanced loading of Cas9 into vesicles.
Analyzed the uptake of SCF-expressing EVs by HSPCs with varying c-KIT expression.
Utilized confocal microscopy and flow cytometry to evaluate delivery and efficacy.
Extracellular vesicles selectively delivered ribonucleoprotein complexes to hematopoietic stem progenitor cells.
HEL cells exhibited over twofold uptake of SCF-expressing EVs compared to Jurkat cells (p=0.0153).
c-KIT knockdown in HEL cells reduced SCF-EV uptake by over 50% (p<0.0001), confirming the targeting efficacy.

Cite This Study

Black et al. (2025) studied this question.

synapsesocial.com/papers/69362f3a4fa91c937236d45e https://doi.org/10.1182/blood-2025-2534

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Implication

This approach shows enhanced delivery of ribonucleoprotein complexes to HSPCs in sickle cell disease, suggesting a more effective gene therapy method.

Engineered extracellular vesicles for In Vivo gene therapy in sickle cell disease

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Member takes

Implication

Key Points

Cite This Study

Engineered extracellular vesicles for In Vivo gene therapy in sickle cell disease

Discussion

Member takes

Implication

Key Points

Cite This Study

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