Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients.

Hasan HashemGiorgia BucciolSeza OzenSule UnalIkbal Ok BozkayaNurten AkarsuMervi TaskinenMinna KoskenvuoJanna SaarelaDimana DimitrovaDennis D HicksteinAmy P HsuSteven M HollandRobert KranceGhadir SasaAshish R KumarIngo MüllerMonica Abreu de SousaSelket DelafontaineLeen MoensFlorian BaborFederica BarzaghiMaria Pia CicaleseRobbert BrediusJoris van MontfransValentina BarettaSimone CesaroPolina StepenskyNeven BenedicteDespina MoshousGuillaume Le GuennoDavid BoutboulJignesh DalalJoel P BrooksElif DokmeciJasmeen DaraCarrie L LucasSophie HambletonKeith WilsonStephen JollesYener KocTayfun GüngörCaroline SchniderFabio CandottiSandra SteinmannAnsgar SchulzChip ChambersMichael HershfieldAmanda OmbrelloJennifer A KanakryIsabelle Meyts

Published in: Journal of clinical immunology (2021)

HCT is a definitive cure for DADA2 with > 95% survival.

Keyphrases

end stage renal disease
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
signaling pathway
cell proliferation
patient reported outcomes
cell death
smoking cessation