Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

Laura García-Pérez; Marja van Eggermond; Lieke van Roon; Sandra A. Vloemans; Martijn Cordes; Axel Schambach; Michael Rothe; Dagmar Berghuis; Chantal Lagresle‐Peyrou; Marina Cavazzana; Fang Zhang; Adrian J. Thrasher; Daniela Salvatori; Pauline Meij; Anna Villa; Jacques J. M. van Dongen; Jaap Jan Zwaginga; Mirjam van der Burg; H. Bobby Gaspar; Arjan C. Lankester; Frank J. T. Staal; Karin Pike‐Overzet

doi:10.1016/j.omtm.2020.03.016

Ask about this paper

Answers are grounded in this paper and cite their sources.

Abstract

cells transduced with our clinical RAG1 vector and transplanted into NSG mice led to improved human B and T cell development. Considering this efficacy outcome, together with favorable safety data, these results substantiate the need for a clinical trial for RAG1-SCID.

Discussion

No takes yet. Share an insight, caveat, or question.

Cite This Study

García-Pérez et al. (2020) studied this question.

synapsesocial.com/papers/6a17b814d990e918e6b46bcc https://doi.org/10.1016/j.omtm.2020.03.016

View Full Paper

Ask AI

Bookmark

Share

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

Ask about this paper

Abstract

Discussion

Cite This Study

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID

Ask about this paper

Abstract

Discussion

Cite This Study

Keep reading

Keep reading