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Stephanie Cherqui

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Open access Oct 2026

Small populations, big impact: leveraging rare disease gene therapies to benefit millions

GLP-1) endogenously (17). Our own work illustrates how discoveries in rare disease research can reshape therapeutic paradigms for other diseases. Our initial research program on cystinosis, a rare devastating multisystem disorder, demonstrated the therapeutic potential of autologous HSC gene therapy for diseases caused...

Stephanie Cherqui · 0 citations

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