Minimal conditioning in Fanconi anemia promotes multi-lineage marrow engraftment at 10-fold lower cell doses.
Kevin G HaworthChristina IronsideMegan A RamirezSarah WeitzBrian C BeardJonathan D SchwartzJennifer E AdairHans-Peter KiemPublished in: The journal of gene medicine (2018)
Minimal conditioning prior to bone marrow transplant in Fanconi anemia promotes the multi-lineage engraftment of 10-fold fewer cells compared to nonconditioned controls. These data provide important insights into the potential of minimally toxic conditioning protocols for FA gene therapy applications.
Keyphrases
- gene therapy
- single cell
- bone marrow
- chronic kidney disease
- induced apoptosis
- iron deficiency
- cell cycle arrest
- mesenchymal stem cells
- hematopoietic stem cell
- electronic health record
- endoplasmic reticulum stress
- signaling pathway
- oxidative stress
- risk assessment
- cord blood
- deep learning
- cell proliferation
- data analysis
- pi k akt