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Loss of nuclear receptor SHP impairs but does not eliminate negative feedback regulation of bile acid synthesis.
The in vivo role of the nuclear receptor SHP in feedback regulation of bile acid synthesis was examined. Loss of SHP in mice caused abnormal accumulation and increased synthesis of bile acids due toExpand
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Systemic delivery of genes to striated muscles using adeno-associated viral vectors
A major obstacle limiting gene therapy for diseases of the heart and skeletal muscles is an inability to deliver genes systemically to muscles of an adult organism. Systemic gene transfer to striatedExpand
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Improved foamy virus vectors with minimal viral sequences.
Foamy virus (FV) vectors show promise for gene therapy applications. However, existing FV vectors either retain a significant portion of the wild-type virus genome or are produced at low titers. WeExpand
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Extraction and analysis of sterols in biological matrices by high performance liquid chromatography electrospray ionization mass spectrometry.
We describe the development of a high performance liquid chromatography mass spectrometry (HPLC-MS) method that allows the identification and quantitation of sterols in mammalian cells and tissues.Expand
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Neuronal expression and subcellular localization of cholesterol 24‐hydroxylase in the mouse brain
Cholesterol 24‐hydroxylase is a cytochrome P450 (CYP46A1) that is selectively expressed in the brain and is responsible for the majority of cholesterol turnover in the central nervous system. MiceExpand
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AAV-mediated gene targeting methods for human cells
Gene targeting with adeno-associated virus (AAV) vectors has been demonstrated in multiple human cell types, with targeting frequencies ranging from 10−5 to 10−2 per infected cell. These targetingExpand
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HLA-E-expressing pluripotent stem cells escape allogeneic responses and lysis by NK cells
Polymorphisms in the human leukocyte antigen (HLA) class I genes can cause the rejection of pluripotent stem cell (PSC)-derived products in allogeneic recipients. Disruption of the Beta-2Expand
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Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors
Recent successes in treating genetic immunodeficiencies have demonstrated the therapeutic potential of stem cell gene therapy. However, the use of gammaretroviral vectors in these trials led toExpand
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A suppressor screen in Mecp 2 mutant mice implicates cholesterol metabolism in Rett syndrome
Rett syndrome (RTT; MIM 312750) is an X-linked neurological disorder presenting with autistic features that afflicts approximately 1 in 10,000 females. After a few months or years of apparentlyExpand
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Foamy virus vectors.
Human foamy virus (HFV) is a retrovirus of the spumavirus family. We have constructed vectors based on HFV that encode neomycin phosphotransferase and alkaline phosphatase. These vectors are able toExpand
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