A Keravala, CL Chavez, G Hu, LE Woodard… - Gene Therapy, 2011 - nature.com Hemophilia B, a hereditary bleeding disorder caused by a deficiency of coagulation factor IX (FIX), is an excellent candidate for gene therapy. However, to date, success in hemophilia gene therapy clinical trials has been limited due to failure to achieve or sustain therapeutic ... Cited by 1 - Related articles - All 3 versions
S Bhaumik, TV Sekar, J Depuy, J Klimash… - Gene Therapy, 2011 - nature.com Gene-directed enzyme prodrug therapy (GDEPT) is a promising and emerging strategy that attempts to limit the systemic toxicity inherent to cancer chemotherapy by means of tumor-targeted delivery and expression of an exogenous gene whose product converts nontoxic prodrug(s) into ...
G Li, KC Biju, X Xu, Q Zhou, C Chen, AJ Valente… - Gene Therapy, 2011 - nature.com Liver X receptors (LXRs) are implicated in the regulation of cholesterol homeostasis, inflammatory response and atherogenesis. Administration of LXR agonists inhibits the progress of atherosclerosis, and also increases plasma triglyceride levels, representing an obstacle ... Cited by 1 - Related articles - All 3 versions
GV Velde, JR Rangarajan, J Toelen… - Gene Therapy, 2011 - nature.com The development of in vivo imaging protocols to reliably track transplanted cells or to report on gene expression is critical for treatment monitoring in (pre)clinical cell and gene therapy protocols. Therefore, we evaluated the potential of lentiviral vectors (LVs) and adeno- ... Cited by 4 - Related articles - All 6 versions
D Mu, R Huang, X Ma, S Li… - Gene Therapy, 2011 - nature.com The thyroid transcription factor Pax-8 could bind with the promoter/enhancer of thyroid-specific genes such as thyroglobulin (Tg), thyroperoxidase (TPO) and sodium iodide symporter (NIS), and regulate the expression of these proteins in thyrocyte. Promoting iodide ...