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Paper Details

Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.
Molecular Therapy - Methods and Clinical Development
31
2020
B and T cell, B and T lymphocytes, CD34+ cells, Mice, RAG1, RAG1 vector, RAG1-SCID, Rag1, Recombinase, Recombinase-Activating Gene-1-Deficient SCID, Recombinase-activating gene-1, Recombinase-activating gene-1 (RAG1)-deficient severe combined immunodeficiency, SCID, T cell receptor genes, codon-optimized RAG1, human, immunoglobulin, internal promoters, mice, patient, patients, wild-type stem cells
Author NameAffiliation
Jaap Jan ZwagingaLeiden University Medical Center
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Datasets

Release 1.2