Severe combined immunodeficiency caused by mutations in the IL2RG gene on the x-chromosome (SCID-X1 or "bubble boy disease") is a devastating genetic disease that results in boys not being able…
Our goal is to develop tools that address major bottlenecks that have prevented the generation of blood forming stem cells in culture for therapeutic use. To help overcome these bottlenecks,…
Tens of thousands of patients need bone marrow transplants (BMT) every year, some for bone marrow (BM) cancers and some for inherited diseases such as sickle cell anemia and thalassemia,…
A goal of stem-cell therapy is to transplant into a patient “tissue-specific” stem cells, which can regenerate a particular type of healthy tissue (e.g., heart or blood cells). A major…
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…
The research performed through this project is very important for the fields of solid organ and bone marrow transplantation because it focuses on a potential new target to increase engraftment…
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