Targeting adipose tissue via systemic gene therapy

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

(2012) D Gaudet et al.   GENE THERAPY

Adeno-Associated Virus Serotype 8 Gene Therapy Leads to Significant Lowering of Plasma Cholesterol Levels in Humanized Mouse Models of Homozygous and Heterozygous Familial Hypercholesterolemia

(2012) Sadik H. Kassim et al.   HUMAN GENE THERAPY

miR-122 regulates hepatic lipid metabolism and tumor suppression

(2012) Jessica Wen et al.   JOURNAL OF CLINICAL INVESTIGATION

Coagulation factor activity and clinical bleeding severity in rare bleeding disorders: results from the European Network of Rare Bleeding Disorders

(2012) F. PEYVANDI et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Endgame: Glybera Finally Recommended for Approval as the First Gene Therapy Drug in the European Union

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

Gene therapy: light is finally in the tunnel

(2012) Huibi Cao et al.   Protein & Cell

Self-complementary AAV mediates gene targeting and enhances endonuclease delivery for double-strand break repair

(2010) M L Hirsch et al.   GENE THERAPY

Celastrol enhances AAV1-mediated gene expression in mice adipose tissues

(2010) F-L Zhang et al.   GENE THERAPY

microRNA122-regulated transgene expression increases specificity of cardiac gene transfer upon intravenous delivery of AAV9 vectors

(2010) A Geisler et al.   GENE THERAPY

Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

(2010) C Qiao et al.   GENE THERAPY

Rapid, Simple, and Versatile Manufacturing of Recombinant Adeno-Associated Viral Vectors at Scale

(2010) Martin Lock et al.   HUMAN GENE THERAPY

Molecular Mechanisms of Hepatic Steatosis and Insulin Resistance in the AGPAT2-Deficient Mouse Model of Congenital Generalized Lipodystrophy

(2009) Víctor A. Cortés et al.   Cell Metabolism

Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

(2009) Francesca Simonelli et al.   MOLECULAR THERAPY

Identification of a novel distal enhancer in human adiponectin gene

(2008) Katsumori Segawa et al.   JOURNAL OF ENDOCRINOLOGY

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY

Widespread Translational Inhibition by Plant miRNAs and siRNAs

(2008) P. Brodersen et al.   SCIENCE

Posttranscriptional Gene Regulation by Spatial Rearrangement of the 3′ Untranslated Region

(2008) Andrea B Eberle et al.   PLOS BIOLOGY