Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID
Recombinase-activating gene-1 (RAG1)-deficient severe combined immunodeficiency (SCID) patients lack B and T lymphocytes due to the inability to rearrange immunoglobulin and T cell receptor genes. Gene therapy is an alternative for those RAG1-SCID patients who lack a suitable bone marrow donor. We d...
Main Authors: | , , , , , , , , , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2020-06-01
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Series: | Molecular Therapy: Methods & Clinical Development |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050120300462 |