Generation of an optimized lentiviral vector encoding a high-expression factor VIII transgene for gene therapy of hemophilia A

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII

(2011) Chuhong Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Codon optimization of human factor VIII cDNAs leads to high-level expression

(2010) N. J. Ward et al.   BLOOD

Lentiviral Vector Platform for Production of Bioengineered Recombinant Coagulation Factor VIII

(2010) H Trent Spencer et al.   MOLECULAR THERAPY

Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors

(2010) Denise E Sabatino et al.   MOLECULAR THERAPY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

State-of-the-Art Lentiviral Vectors for Research Use: Risk Assessment and Biosafety Recommendations

(2009) Katia Pauwels et al.   CURRENT GENE THERAPY

Comparison of Factor VIII Transgenes Bioengineered for Improved Expression in Gene Therapy of Hemophilia A

(2009) Kerry L. Dooriss et al.   HUMAN GENE THERAPY

Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia

(2009) África González-Murillo et al.   HUMAN GENE THERAPY

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A

(2009) Christopher B Doering et al.   MOLECULAR THERAPY

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Advancements in gene transfer-based therapy for hemophilia A

(2009) Christopher B Doering et al.   Expert Review of Hematology

Complete Correction of Hemophilia A with Adeno-Associated Viral Vectors Containing a Full-Size Expression Cassette

(2008) Hui Lu et al.   HUMAN GENE THERAPY