Examining the efficacy of GDF11 antibody as a rejuvenator of aged human muscle stem cell capacity and muscle repair .

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Research Objective To examine the efficacy of blocking blood borne GDF11 activity to rejuvenate aged human muscle stem cell regenerative capacity. Impact This project will provide a proof-of-principle that GDF11…

Continue ReadingExamining the efficacy of GDF11 antibody as a rejuvenator of aged human muscle stem cell capacity and muscle repair .

Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

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Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…

Continue ReadingDevelopment of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

Development of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

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Translational Candidate An earlier, safer noninvasive prenatal screening test for β-thalassemia and sickle cell disease to identify candidates for prenatal stem cell therapy Area of Impact Our test is safer…

Continue ReadingDevelopment of a Noninvasive Prenatal Test for Beta-Hemoglobinopathies for Earlier Stem Cell Therapeutic Interventions

Toward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells

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Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells…

Continue ReadingToward a Cure for Gaucher Disease Type 1: Autologous Transplantation of Genome Edited Hematopoietic Stem Cells