Transgene Regulation Using the Tetracycline-Inducible TetR-KRAB System after AAV-Mediated Gene Transfer in Rodents and Nonhuman Primates

Regulatory evaluation of Glybera in Europe — two committees, one mission

(2013) Daniela Melchiorri et al.   NATURE REVIEWS DRUG DISCOVERY

Lessons Learned from the Clinical Development and Market Authorization of Glybera

(2013) Laura M. Bryant et al.   Human Gene Therapy Clinical Development

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

Integration Frequency and Intermolecular Recombination of rAAV Vectors in Non-human Primate Skeletal Muscle and Liver

(2012) Ali Nowrouzi et al.   MOLECULAR THERAPY

Specific Micro RNA-Regulated TetR-KRAB Transcriptional Control of Transgene Expression in Viral Vector-Transduced Cells

(2012) Virginie Pichard et al.   PLoS One

Regulation of Episomal Gene Expression by KRAB/KAP1-Mediated Histone Modifications

(2009) I. Barde et al.   JOURNAL OF VIROLOGY

Lack of Immunotoxicity After Regional Intravenous (RI) Delivery of rAAV to Nonhuman Primate Skeletal Muscle

(2009) Alice Toromanoff et al.   MOLECULAR THERAPY

The zinc finger DNA-binding domain of K-RBP plays an important role in regulating Kaposi's sarcoma-associated herpesvirus RTA-mediated gene expression

(2009) Zhilong Yang et al.   VIROLOGY

Doxycycline-controlled splicing modulation by regulated antisense U7 snRNA expression cassettes

(2008) J Marquis et al.   GENE THERAPY

Adeno-Associated Virus Vector Genomes Persist as Episomal Chromatin in Primate Muscle

(2008) M. Penaud-Budloo et al.   JOURNAL OF VIROLOGY

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle

(2008) Alice Toromanoff et al.   MOLECULAR THERAPY

Transduction Efficiency and Immune Response Associated With the Administration of AAV8 Vector Into Dog Skeletal Muscle

(2008) Sachiko Ohshima et al.   MOLECULAR THERAPY