Research Objective Our goal is to develop new gene editing methods for producing universal, off-the-shelf, therapeutic T cells from induced pluripotent stem cells (iPSC) that can be applied to a… ...

Objetivo de la investigación Se combinará un injerto de pasta articular que contiene células madre mesenquimales y un hidrogel adhesivo que favorece el crecimiento del cartílago para lograr un ...

Sphingosine phosphate lyase insufficiency syndrome (SPLIS) is a rare genetic condition caused by mutations in the SGPL1 gene. This gene encodes an essential enzyme needed to metabolize a group of ...

KIF1A-associated neurological disorder (KAND) is a neurodegenerative and often lethal ultrarare disease with a wide phenotypic spectrum associated with largely heterozygous de novo missense variants ...

Translational Candidate Genetically-corrected patient iPSC-derived neural progenitor cells, which have demonstrated efficacy to correct disease phenotype in a CD mouse model. Area of Impact This ...

Metabolism regulates cell fates during early mammalian cell differentiation. This protocol describes the steps for directed differentiation of primed human pluripotent stem cells (hPSCs) into the ...

We previously showed that inhibition of glycolysis in cutaneous squamous cell carcinoma (SCC)-initiating cells had no effect on tumorigenesis, despite the perceived requirement of the Warburg effect, ...

Colin M McCrimmon, Daniel Toker, Marie Pahos, Kevin Lozano, Jack J Lin, Jack Parent, Andrew Tidball, Jie Zheng, Laszlo Molnar, Istvan Mody, Bennett G Novitch, Ranmal A Samarasinghe Neurodevelopmental ...

beta-globin lentiviral vectors (beta-LV) have faced challenges in clinical translation for gene therapy of sickle cell disease (SCD) due to low titer and sub-optimal gene transfer to hematopoietic ...

X-linked chronic granulomatous disease (X-CGD) is a primary immunodeficiency caused by mutations in the CYBB gene, resulting in the inability of phagocytic cells to eliminate infections. To design a ...

Alcohol (ethanol, EtOH) consumption during pregnancy can result in fetal alcohol spectrum disorders (FASDs), which are characterized by prenatal and postnatal growth restriction and craniofacial ...

Kevyn L Hart, Boya Liu, Devin Brown, Beatriz Campo-Fernandez, Kevin Tam, Katherine Orr, Roger P Hollis, Christian Brendel, David A Williams, Donald B Kohn A major limitation of gene therapy for sickle ...