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H. Malech

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Open access Jul 2026

Implementing a First-in-Human Clinical Trial of Prime-Editing Gene Therapy in p47-CGD: Clinical Data and Site Perspectives

While the patient’s clinical course was simple, setting up the clinical trial required acrobatics and coordination worthy of a hyperactive octopus, implementing a first-in-human therapy for patients whose lives are not immediately at risk represents a high degree of complexity.

K. Leveillé, J. Gori, S. Turvey et al. · 0 citations
Open access Jul 2026

Base Editing Hematopoietic Stem/Progenitor Cell Gene Therapy for Treatment of X-Linked Severe Combined Immunodeficiency

It is concluded from preliminary data that gene therapy using BE HSPCs appear very promising for achieving an earlier and more robust multi-lineage immune reconstitution in adult patients using BE HSPCs.

S. S. de Ravin, Michelle Ma, Yuzhi Yin et al. · 0 citations

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