Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep

The Intravenous Route of Injection Optimizes Engraftment and Survival in the Murine Model of In Utero Hematopoietic Cell Transplantation

(2016) Matthew M. Boelig et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Vector serotype screening for use in ovine perinatal lung gene therapy

(2016) Lauren E. McClain et al.   JOURNAL OF PEDIATRIC SURGERY

Stable long-term mixed chimerism achieved in a canine model of allogeneic in utero hematopoietic cell transplantation

(2014) J. D. Vrecenak et al.   BLOOD

In Utero Stem Cell and Gene Therapy: Current Status and Future Perspectives

(2014) Stavros Loukogeorgakis et al.   EUROPEAN JOURNAL OF PEDIATRIC SURGERY

Immunological Ignorance Allows Long-Term Gene Expression After Perinatal Recombinant Adeno-Associated Virus-Mediated Gene Transfer to Murine Airways

(2014) Marianne S. Carlon et al.   HUMAN GENE THERAPY

Intracerebral Administration of Adeno-Associated Viral Vector Serotype rh.10 Carrying Human SGSH and SUMF1 cDNAs in Children with Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/II Trial

(2014) Marc Tardieu et al.   HUMAN GENE THERAPY

In Utero Lung Gene Transfer Using Adeno-Associated Viral and Lentiviral Vectors in Mice

(2014) Luc Joyeux et al.   Human Gene Therapy Methods

Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9

(2014) David H Stitelman et al.   Molecular Therapy-Methods & Clinical Development

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

(2014) M Corti et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

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

(2013) Y Shi et al.   GENE THERAPY

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

(2013) Barbara K. Smith et al.   HUMAN GENE THERAPY

Immune Responses to Intramuscular Administration of Alipogene Tiparvovec (AAV1-LPLS447X) in a Phase II Clinical Trial of Lipoprotein Lipase Deficiency Gene Therapy

(2013) Valerie Ferreira et al.   HUMAN GENE THERAPY

A largely random AAV integration profile after LPLD gene therapy

(2013) Christine Kaeppel et al.   NATURE MEDICINE

Characterization of Naturally-Occurring Humoral Immunity to AAV in Sheep

(2013) Joseph Tellez et al.   PLoS One

Humoral Immune Response to AAV

(2013) Roberto Calcedo et al.   Frontiers in Immunology

Heparin-binding correlates with increased efficiency of AAV1- and AAV6-mediated transduction of striated muscle, but negatively impacts CNS transduction

(2012) A L H Arnett et al.   GENE THERAPY

Single Amino Acid Modification of Adeno-Associated Virus Capsid Changes Transduction and Humoral Immune Profiles

(2012) C. Li et al.   JOURNAL OF VIROLOGY

Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

(2011) Samuel G. Jacobson   ARCHIVES OF OPHTHALMOLOGY

Jaagsiekte sheep retrovirus pseudotyped lentiviral vector-mediated gene transfer to fetal ovine lung

(2011) M G Davey et al.   GENE THERAPY

Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model

(2011) J L Roybal et al.   GENE THERAPY

Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease

(2011) J L Roybal et al.   GENE THERAPY

Evaluation of Adeno-Associated Viral Vectors for Liver-Directed Gene Transfer in Dogs

(2011) Peter Bell et al.   HUMAN GENE THERAPY

Protein-energy malnutrition during early gestation in sheep blunts fetal renal vascular and nephron development and compromises adult renal function

(2011) Louise J. Lloyd et al.   JOURNAL OF PHYSIOLOGY-LONDON

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

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

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

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D

(2010) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

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

Dystrophin Immunity in Duchenne's Muscular Dystrophy

(2010) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Haploidentical In Utero Hematopoietic Cell Transplantation Improves Phenotype and Can Induce Tolerance for Postnatal Same-Donor Transplants in the Canine Leukocyte Adhesion Deficiency Model

(2009) William H. Peranteau et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors

(2009) M Endo et al.   GENE THERAPY

The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques

(2009) Lili Wang et al.   MOLECULAR THERAPY

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

(2009) Lili Wang et al.   MOLECULAR THERAPY

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Host Immune Responses to Chronic Adenovirus Infections in Human and Nonhuman Primates

(2008) R. Calcedo et al.   JOURNAL OF VIROLOGY