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文献和实验Ligand- and Structure-Based Pregnane X Receptor Models
The human pregnane X receptor (PXR) is a ligand dependent transcription factor that can be activated by structurally diverse agonists including steroid hormones, bile acids, herbal drugs, and prescription medications. PXR regulates
Ligand Characterization Using Microphysiometry
of cells to the agonist and the pA2 value for the antagonist. Support protocols describe optimization of two aspects of this procedure: the duration of ligand exposure at a given concentration and the length of recovery time between the administration
Gene Therapy of X-Linked Severe Combined Immunodeficiency
This review describes the main steps which led to carrying out the gene ther-apy clinical trial for Xlinked severe combined immune deficiency (SCID-X1) patients, from the preparation of the retroviral vector up to the design of GMP conditions
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