Sickle cell disease (SCD)results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen. It occurs with a frequency of…
Disorders affecting the blood, including Sickle Cell Disease (SCD), are the most common genetic disorders in the world. SCD causes significant suffering and early death, despite major improvements in medical…
Sickle cell disease (SCD), which results from an inherited mutation in the hemoglobin gene that causes red blood cells to "sickle" under conditions of low oxygen, occurs with a frequency…
Boston Children's Hospital is conducting a gene therapy clinical trial for sickle cell disease (SCD). This project is part of an agreement between CIRM and the National Heart, Lung, and…
Sickle Cell Disease (SCD) is an inherited blood disorder caused by a single gene mutation that results in the production of "sickle" shaped red blood cells. It affects an estimated…
Scientists at the City of Hope are conducting a Phase 1 clinical trial testing a stem cell-based therapy for adult patients with severe sickle cell disease (SCD) - a chronic, debilitating…
Sickle cell disease (SCD) is caused by a genetic mutation in the hemoglobin gene which causes red blood cells to "sickle" under conditions of low oxygen. SCD affects 1:500 African-Americans and…
Therapeutic Candidate or Device Autologous CD34+ cells transduced ex vivo with the BCH_BB694 LCR(-HS4) bGp D12shmiR lentiviral vector Indication Sickle cell disease with severe phenotype. Therapeutic Mechanism Silencing of beta-sickle…
Therapeutic Candidate or Device CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person Indication Persons with sickle…
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