A Novel, Robust and Comprehensive Predictive Tool Using Human Disease-Specific Induced Pluripotent Stem Cells for Preclinical Drug Screening

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Translational Candidate A library of induced pluripotent stem cell-derived cardiomyocytes from healthy subjects as well as patients with common hereditary cardiac disorders Area of Impact Preclinical toxicity screening and drug…

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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

Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy.

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Translational Candidate Development of a universal allogeneic human interneuron cell therapy candidate for the treatment of drug resistant focal epilepsy. Area of Impact Non-destructive treatment option for drug-resistant focal epilepsy…

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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…

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