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The ultimate goal of this approach is to develop a one-time, life-long treatment for SCD and beta-thalassemia.
72 newborns with beta-thalassemia were diagnosed as a result of the marriage of the carrier couples in 6 years.
17) Furthermore, the nutrient may be reduced in beta-thalassemia, (18) an hereditary anemia associated with reduced production of normal adult hemoglobin.
Diagnosis of sickle cell anemia and beta-thalassemia with enzymatically amplified DNA and nonradioactive allele-specific oligonucleotide probes.
At Johns Hopkins University, David Weatherall and associates labeled reticulocytes of thalassemic patients with radioactive amino acids in vitro and were able to demonstrate that in patients with alpha- and beta-thalassemia, alpha- or beta-chain production was defective because of unbalanced globin chain synthesis (21).
About 100,000 children are born with beta-thalassemia each year, and many suffer from serious, sometimes fatal, anemia.
Cline's attempt, no one would be ready to try human gene therapy for years, but many still thought beta-thalassemia was the best target.
Gene Therapy for Patients with Severe Beta-Thalassemia May Reduce or Eliminate Life-Long Need for Blood Transfusions Update of Results from the Northstar Study (HGB-204): A Phase I/II Study of Gene Therapy for Beta-Thalassemia Major via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product) [201]