HSCT cures ADA2 deficiency.
Sign Up to like & getrecommendations! Published in 2017 at "Blood"
DOI: 10.1182/blood-2017-10-811125
Abstract: In this issue of Blood , Hashem et al report a remarkable therapeutic result: all facets of the diverse spectrum of the rare disease adenosine deaminase 2 (ADA2) deficiency, which range from vasculopathy including stroke… read more here.
Keywords: ada2 deficiency; hsct cures; immune system; innate immune ... See more keywords
ALPS-Like Phenotype Caused by ADA2 Deficiency Rescued by Allogeneic Hematopoietic Stem Cell Transplantation
Sign Up to like & getrecommendations! Published in 2019 at "Frontiers in Immunology"
DOI: 10.3389/fimmu.2018.02767
Abstract: Adenosine deaminase 2 (ADA2) deficiency is an auto-inflammatory disease due to mutations in cat eye syndrome chromosome region candidate 1 (CECR1) gene, currently named ADA2. The disease has a wide clinical spectrum encompassing early-onset vasculopathy… read more here.
Keywords: ada2 deficiency; cell transplantation; stem cell; hematopoietic stem ... See more keywords