Gene therapy for severe combined immunodeficiency: are we there yet?

Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1. 1Severe combined immunodeficiency and microcephaly in siblings with hypomorphic mutations in DNA ligase IV2005 · 206 citations
2. 2Administration of herpes simplex-thymidine kinase-expressing donor T cells with a T-cell-depleted allogeneic marrow graft2001 · 288 citations
3. 3Retrovirus Silencing and Vector Design: Relevance to Normal and Cancer Stem Cells?2005 · 63 citations
4. 4Long-term T-cell reconstitution after hematopoietic stem-cell transplantation in primary T-cell–immunodeficient patients is associated with myeloid chimerism and possibly the primary disease phenotype2007 · 232 citations
5. 5Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy2007 · 257 citations