Phase 1 Trial Results for Lentiviral Gene Therapy for Patients with Hemophilia A Hemophilia A is a X-linked disorder characterized by less than 1% of blood clotting Factor VIII in the blood and… January 1, 2025
Transfusion Patterns After CAR T-Cell Therapy for Large B-Cell Lymphoma Three different types of chimeric antigen receptor (CAR) T-cell therapies have been approved to treat refractory or relapsed large B-cell… April 10, 2024
The Genealogy of Blood Cells Hematopoietic stem cells (HSCs) give rise to all types of blood cells through myeloid progenitor cell types (e.g., red blood… January 31, 2024
Gene Therapy Phase 3 Study Results for Hemophilia B Hemophilia B is a rare, X-linked bleeding disorder caused by partial or complete deficiency of clotting factor IX. Currently, the… March 8, 2023
Anti-CD19 CAR-T Cells as First-Line Therapy for High-Risk Large B-Cell Lymphoma Large-B-cell lymphoma (LBCL) is one of the most common types of non-Hodgkin lymphoma, and many patients do not respond well… June 7, 2022
Genomic Profiles Associated with CAR T-Cell Therapy Efficacy Chimeric antigen receptor (CAR) T-cell therapy is a promising therapy used to treat hematological cancers by targeting a specific antigen. … May 24, 2022
LentiGlobin Therapy for Sickle Cell Disease Sickle cell disease (SCD) is caused by a single point mutation in the β-globin gene and affects around 100,000 people… January 4, 2022
CRISPR-Cas9 Gene Editing and Lentiviral Gene Therapy Hold Promise to Treat Transfusion Dependent β-Globin Hemoglobinopathies Both transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are common monogenetic diseases caused by mutations in the gene encoding… December 15, 2020
Gene Therapy for Hemophilia A Decreases Bleeding Adeno-associated virus (AAV)-mediated gene therapy is a promising new treatment for hemophilia A which decreases bleeding and thus risk of… January 8, 2020
CRISPR-Gene Editing Uses for Transfusion Medicine Clustered regularly interspaced short palindromic repeats (CRISPR) and Cas proteins evolved as part of the prokaryotes adaptive immune system. Cas… April 18, 2019
Cell Therapy