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C. Banuelos

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Open access Jul 2026

Autologous Ex Vivo Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency-I Achieves Durable Immune Reconstitution and Reduction of Infection-Related Morbidity: Updated 3.5-5.5-Year Results from a Phase I/II Study

Autologous HSC gene therapy is supported as an effective alternative to alloHSCT with a favorable risk-benefit profile for severe LAD-I, and markedly lower annualized incidences of prespecified serious infections, infection-related hospitalizations, and prolonged infection-related hospitalizations after RP-L201 treatment relative to pre-treatment incidences.

C. Booth, J. Sevilla, E. Almarza et al. · 0 citations