Human embryonic stem cells (hESC) hold great promise in regenerative medicine and cell replacement therapies because of their unique ability to self-renew and their developmental potential to form all cell…
The capacity of human embryonic stem cells (hESCs) to perpetuate themselves indefinitely in culture and to differentiate to all cell types of the body has lead to numerous studies that…
Hematopoietic stem cells (HSC) have been used successfully to cure various life-threatening blood diseases. Yet, matching HSCs are not available for every patient. Human embryonic stem cells (hESC) may provide…
For many therapeutic reasons it is important to have available large numbers of blood cells. However, it is difficult to generate large numbers of specialized blood cells that have the…
The discovery of induced pluripotent stem (iPS) cell technology promises to revolutionize our understanding of human disease and to allow the development of new cellular therapies for regenerative medicine applications.…
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…
Hematopoietic cells are responsible for generating all cell types present in the blood and therefore critical for the provision of oxygen and nutrients to all the tissues in the body.…
Research Objective A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells Impact We aim to develop a novel genome editing based therapy…
Research Objective This proposal will deliver a small molecule therapeutic candidate for the treatment of Myelodysplastic Syndromes and will act by inducing differentiation on mutated hematopoietic stem cells. Impact This…
Research Objective A new therapeutic option for DOCK8 deficiency using autologous human hematopoietic stem cells modified through either lentiviral gene addition or CRISPR/Cas9 based gene editing. Impact Allogeneic HSCT is…
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