How We Manage Adenosine Deaminase-Deficient Severe Combined Immune Deficiency (ADA SCID)
Sign Up to like & getrecommendations! Published in 2017 at "Journal of Clinical Immunology"
DOI: 10.1007/s10875-017-0373-y
Abstract: Adenosine deaminase-deficient severe combined immune deficiency (ADA SCID) accounts for 10–15% of cases of human SCID. From what was once a uniformly fatal disease, the prognosis for infants with ADA SCID has improved greatly based… read more here.
Keywords: adenosine deaminase; ada scid; deficient severe; deaminase deficient ... See more keywords
Twenty-Five Years of Gene Therapy for ADA-SCID: From Bubble Babies to an Approved Drug.
Sign Up to like & getrecommendations! Published in 2017 at "Human gene therapy"
DOI: 10.1089/hum.2017.175
Abstract: Twenty-five years have passed since first attempts of gene therapy (GT) in children affected by severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) defect, also known by the general public as bubble babies. ADA-SCID… read more here.
Keywords: gene; ada scid; gene therapy;
Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants
Sign Up to like & getrecommendations! Published in 2022 at "Blood Advances"
DOI: 10.1182/bloodadvances.2022007803
Abstract: Abstract Autologous stem cell transplant with gene therapy (ASCT-GT) provides curative therapy while reducing pretransplant immune-suppressive conditioning and eliminating posttransplant immune suppression. Clonal hematopoiesis of indeterminate potential (CHIP)–associated mutations increase and telomere lengths (TLs) shorten… read more here.
Keywords: chip; ada scid; therapy; gene therapy ... See more keywords