Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX

A TLR and Non-TLR Mediated Innate Response to Lentiviruses Restricts Hepatocyte Entry and Can be Ameliorated by Pharmacological Blockade

(2012) Judith Agudo et al.   MOLECULAR THERAPY

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy

(2012) Brandon K. Sack et al.   PLoS One

Hepatocyte-targeted expression by integrase-defective lentiviral vectors induces antigen-specific tolerance in mice with low genotoxic risk

(2011) Janka Mátrai et al.   HEPATOLOGY

Gene therapy for haemophilia: a long and winding road

(2011) K. A. HIGH   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

(2011) Amit C Nathwani et al.   MOLECULAR THERAPY

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Manipulating Immune Tolerance with Micro-RNA Regulated Gene Therapy

(2011) Kevin Scott Goudy et al.   Frontiers in Microbiology

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

(2010) V. R. Arruda et al.   BLOOD

Recombinant Adeno-Associated Virus-Mediated In Utero Gene Transfer Gives Therapeutic Transgene Expression in the Sheep

(2010) Anna L. David et al.   HUMAN GENE THERAPY

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs

(2010) Virginia Haurigot et al.   MOLECULAR THERAPY

Progress and prospects: immune responses to viral vectors

(2009) S Nayak et al.   GENE THERAPY

Induction of Immune Tolerance to FIX Following Muscular AAV Gene Transfer Is AAV-dose/FIX-level Dependent

(2009) Meagan E Kelly et al.   MOLECULAR THERAPY

Regulatory T Cells and Immune Tolerance to Coagulation Factor IX in the Context of Intramuscular AAV1 Gene Transfer

(2009) Meagan Kelly et al.   MOLECULAR THERAPY

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

(2009) Jonathan D Finn et al.   MOLECULAR THERAPY

Suppression of HLA expression by lentivirus-mediated gene transfer of siRNA cassettes and in vivo chemoselection to enhance hematopoietic stem cell transplantation

(2008) Katrin Hacke et al.   IMMUNOLOGIC RESEARCH

Erythroid-specific Human Factor IX Delivery From In Vivo Selected Hematopoietic Stem Cells Following Nonmyeloablative Conditioning in Hemophilia B Mice

(2008) Alex H Chang et al.   MOLECULAR THERAPY