A5041469671- Pluripotent Stem Cells Research
- Cancer, Hypoxia, and Metabolism
- Nitric Oxide and Endothelin Effects
- Virus-based gene therapy research
- Biomedical Ethics and Regulation
- Amino Acid Enzymes and Metabolism
- Barrier Structure and Function Studies
- RNA Interference and Gene Delivery
- Metabolism and Genetic Disorders
- Cancer Research and Treatments
- Musicology and Musical Analysis
- Connective tissue disorders research
- Ion Transport and Channel Regulation
- Intracerebral and Subarachnoid Hemorrhage Research
- Travel Writing and Literature
- CRISPR and Genetic Engineering
- Diverse Musicological Studies
- Neurological Disease Mechanisms and Treatments
- Caveolin-1 and cellular processes
- Global Maritime and Colonial Histories
Baylor College of Medicine
2015-2020
German Jordanian University
2015
University of Washington
2010-2011
Seattle University
2010
Precise genetic manipulation of human pluripotent stem cells will be required to realize their scientific and therapeutic potential. Here, we show that adeno-associated virus (AAV) gene targeting vectors can used genetically engineer embryonic (ESCs) induced (iPSCs). Different types sequence-specific changes, including the creation correction mutations, were introduced into HPRT1 HMGA1 genes (HPRT1 mutations being responsible for Lesch–Nyhan syndrome). Gene occurred at high frequencies in...
Osteogenesis imperfecta (OI) is caused by dominant mutations in the type I collagen genes. In principle, skeletal abnormalities of OI could be treated transplantation patient-specific, bone-forming cells that no longer express mutant gene. Here, we develop this approach isolating mesenchymal from patients, inactivating their genes adeno-associated virus (AAV)-mediated gene targeting, and deriving induced pluripotent stem (iPSCs) were expanded differentiated into (iMSCs). Gene-targeted iMSCs...
Previous studies have shown that nitric oxide (NO) supplements may prevent bone loss and fractures in preclinical models of estrogen deficiency. However, the mechanisms by which NO modulates anabolism remain largely unclear. Argininosuccinate lyase (ASL) is only mammalian enzyme capable synthesizing arginine, sole precursor for synthase-dependent (NOS-dependent) synthesis. Moreover, ASL also required channeling extracellular arginine to NOS production. deficiency (ASLD) thus a model study...
Nitric oxide (NO) is a critical signaling molecule that has been implicated in the pathogenesis of neurocognitive diseases. Both excessive and insufficient NO production have linked to pathology. Previously, we shown argininosuccinate lyase deficiency (ASLD) novel model system investigate cell-autonomous, nitric synthase-dependent deficiency. Humans with ASLD are at increased risk for developing hyperammonemia due block ureagenesis. However, natural history studies individuals multisystem...
Nitric oxide (NO) is an important mediator of vascular homeostasis and its deficiency in murine models results hypertension. However, there are few monogenic causes NO humans the effects such genetic forms on vasculature not well-studied. We have recently shown that argininosuccinate lyase (ASL), a urea cycle enzyme, necessary for synthesis NO. ASL decreased production hypertension mice. To investigate whether loss Asl-mediated endothelium alone can cause hypertension, we generated mouse...