Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use

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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,…

Continue ReadingSmall molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use

Gene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders

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Liver transplantation (LT) has been used to treat a variety of liver diseases. Within hours after birth, neonates can present with disorders of the urea cycle (UCDs), the critical metabolic…

Continue ReadingGene therapy-corrected autologous hepatocyte-like cells from induced pluripotent stem cells for the treatment of pediatric single enzyme disorders