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Cell Therapy

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

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