CS
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Publications (8)
- MATRIN3 deficiency in human cells triggers an autoinflammatory response via cGAS–STING activation Save
- The promise of in vivo HSC prime editing Save
- CRISPR/Cas9-mediated Cxcr4 Disease Allele Inactivation for Gene Therapy in a Mouse Model of WHIM Syndrome Save
- Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human X-linked severe combined immunodeficiency Save
- CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes Save
- Lentivector cryptic splicing mediates increase in CD34+ clones expressing truncated HMGA2 in human SCID-X1 Save
- Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair Save
- Homozygous IL37 mutation associated with infantile inflammatory bowel disease Save