Generation and Characterization of Adeno-Associated Virus Producer Cell Lines for Research and Preclinical Vector Production

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

(2012) Seppo Ylä-Herttuala   MOLECULAR THERAPY

HPV-16 E1, E2 and E6 each complement the Ad5 helper gene set, increasing rAAV2 and wt AAV2 production

(2011) M Cao et al.   GENE THERAPY

Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial

(2011) James A. Allay et al.   HUMAN GENE THERAPY

Large-scale recombinant adeno-associated virus production

(2011) R. M. Kotin   HUMAN MOLECULAR GENETICS

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Production, Purification and Characterization of Adeno-Associated Vectors

(2010) Eduard Ayuso et al.   CURRENT GENE THERAPY

HPV-Mediated Genome Instability: At the Roots of Cervical Carcinogenesis

(2010) E. Boccardo   CYTOGENETIC AND GENOME RESEARCH

Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors

(2010) N Zeltner et al.   GENE THERAPY

A Versatile Adeno-Associated Virus Vector Producer Cell Line Method for Scalable Vector Production of Different Serotypes

(2010) Zhenhua Yuan et al.   HUMAN GENE THERAPY

Manufacturing Recombinant Adeno-Associated Viral Vectors from Producer Cell Clones

(2009) Barbara A. Thorne et al.   HUMAN GENE THERAPY

Human RPE65 Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

(2009) Artur V. Cideciyan et al.   HUMAN GENE THERAPY

Transient Transfection Methods for Clinical Adeno-Associated Viral Vector Production

(2009) J. Fraser Wright   HUMAN GENE THERAPY

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

(2009) Albert M Maguire et al.   LANCET

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

Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

(2009) Artur V. Cideciyan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Site-specific integration of adeno-associated virus involves partial duplication of the target locus

(2009) E. Henckaerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A study of monoclonal antibody-producing CHO cell lines: What makes a stable high producer?

(2008) Janet Chusainow et al.   BIOTECHNOLOGY AND BIOENGINEERING

Improved Splicing of Adeno-Associated Viral (AAV) Capsid Protein-Supplying Pre-mRNAs Leads to Increased Recombinant AAV Vector Production

(2008) K. David Farris et al.   HUMAN GENE THERAPY

Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses

(2008) Bernd Hauck et al.   MOLECULAR THERAPY

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

(2008) Albert M. Maguire et al.   NEW ENGLAND JOURNAL OF MEDICINE

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

(2008) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

(2008) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA