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June 30, 2026Molecular Therapy AdvancesOpen Access

HSCT in SAVI: evidence for a curative hematopoietic approach and perspectives for gene therapy

Jade Cognard Sébastien Viel Alexandre Bélot

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Key Points

This research aims to explore the efficacy of hematopoietic stem cell transplantation (HSCT) in treating SAVI and its implications for gene therapy.
Analysis of HSCT effectiveness in patients with SAVI
Review of genetic mutations associated with SAVI
Examination of immune response post-treatment.
HSCT led to significant improvement in patients, with a marked reduction in disease symptoms.
Evidence of long-lasting immune reconstitution post-HSCT was observed.
Potential pathways for gene therapy targeting STING1 mutations were identified.

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Implication

Demonstrates a curative hematopoietic strategy in SAVI, suggesting new gene therapy perspectives.

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Cite This Study

Cognard et al. (2026) studied this question.

synapsesocial.com/papers/6a4750f45c29257aa25785c4 https://doi.org/10.1016/j.omta.2026.201784

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