What's New

Last Posted: Aug 05, 2019

• Gene therapy for primary immunodeficiency.
  Booth Claire et al. Human molecular genetics 2019 Jul
• Severe Combined Immunodeficiency: A Review for Neonatal Clinicians.
  Michniacki Thomas F et al. NeoReviews 2019 Jun 20(6) e326-e335
• Cost-effectiveness of newborn screening for severe combined immunodeficiency.
  Van der Ploeg Catharina P B et al. European journal of pediatrics 2019 May 178(5) 721-729
• Experimental gene therapy frees ‘bubble-boy’ babies from a life of isolation- Treatment restores immune-system function in young children with severe disorder.
  H Ledford, Nature, April 17, 2019
• Gene therapy restores immunity in infants with rare immunodeficiency disease
  NIH News, April 17, 2019
• Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
  Mamcarz Ewelina et al. The New England journal of medicine 2019 Apr 380(16) 1525-1534
• Clinical and economic aspects of newborn screening for severe combined immunodeficiency: DEPISTREC study results.
  Thomas Caroline et al. Clinical immunology (Orlando, Fla.) 2019 Apr 20233-39
• Rethinking newborn screening for severe combined immunodeficiency: Lessons from an international partnership for patients with primary immunodeficiencies in Pakistan.
  Wallace Jacqueline G et al. Clinical immunology (Orlando, Fla.) 2019 Mar
• Clinical, Immunological, and Molecular Findings in 57 Patients With Severe Combined Immunodeficiency (SCID) From India.
  Aluri Jahnavi et al. Frontiers in immunology 2019 1023
• Newborn Screening for Severe Combined Immunodeficiency and T-cell Lymphopenia in California, 2010-2017.
  Amatuni George S et al. Pediatrics 2019 Jan
• Lessons Learned from Newborn Screening in Pilot Studies.
  Taylor Jennifer L et al. North Carolina medical journal 80(1) 54-58
• Newborn screening for severe combined immunodeficiency and T-cell lymphopenia.
  Puck Jennifer M et al. Immunological reviews 2019 Jan 287(1) 241-252
• Newborn Screening for Severe Combined Immunodeficiency in the United States: Lessons Learned.
  Dorsey Morna J et al. Immunology and allergy clinics of North America 2019 Feb 39(1) 1-11
• Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries.
  Fernandes Juliana Folloni et al. Journal of clinical immunology 2018 Nov
• Detecting 22q11.2 Deletion Syndrome in Newborns with Low T Cell Receptor Excision Circles from Severe Combined Immunodeficiency Screening.
  Liao Hsuan-Chieh et al. The Journal of pediatrics 2018 Sep