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Long-term phenotypic correction in factor IX knockout mice by using phiC31 integrase-mediated gene therapy

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

Noninvasive optical imaging of nitroreductase gene-directed enzyme prodrug therapy system in living animals

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 ...

Macrophage LXRα gene therapy ameliorates atherosclerosis as well as hypertriglyceridemia in LDLR−/− mice

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

Evaluation of the specificity and sensitivity of ferritin as an MRI reporter gene in the mouse brain using lentiviral and adeno-associated viral vectors

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

Radioiodine therapy of thyroid carcinoma following Pax-8 gene transfer

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 ...

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