Long-term, high-level hepatic secretion of acid α-glucosidase for Pompe disease achieved in non-human primates using helper-dependent adenovirus

CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy

(2015) Kathryn L. Berrier et al.   GENETICS IN MEDICINE

Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade

(2015) Sang-oh Han et al.   HUMAN GENE THERAPY

ERAP1 functions override the intrinsic selection of specific antigens as immunodominant peptides, thereby altering the potency of antigen-specific cytolytic and effector memory T-cell responses

(2014) D. P. W. Rastall et al.   INTERNATIONAL IMMUNOLOGY

Adenovirus Vector-Induced Innate Inflammatory Mediators, MAPK Signaling, As Well As Adaptive Immune Responses Are Dependent upon Both TLR2 and TLR9 In Vivo

(2014) D. M. Appledorn et al.   JOURNAL OF IMMUNOLOGY

Transgene Expression up to 7 Years in Nonhuman Primates Following Hepatic Transduction with Helper-Dependent Adenoviral Vectors

(2013) Nicola Brunetti-Pierri et al.   HUMAN GENE THERAPY

Endoplasmic Reticulum Aminopeptidase-1 Functions Regulate Key Aspects of the Innate Immune Response

(2013) Yasser A. Aldhamen et al.   PLoS One

Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease

(2012) Ping Zhang et al.   HUMAN GENE THERAPY

Persistence of high sustained antibodies to enzyme replacement therapy despite extensive immunomodulatory therapy in an infant with Pompe disease: Need for agents to target antibody-secreting plasma cells

(2012) Suhrad G. Banugaria et al.   MOLECULAR GENETICS AND METABOLISM

Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques

(2012) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression

(2011) Jennifer B Nietupski et al.   MOLECULAR THERAPY

Adenovirus-based vaccination against Clostridium difficile toxin A allows for rapid humoral immunity and complete protection from toxin A lethal challenge in mice

(2011) Sergey S. Seregin et al.   VACCINE

Effects of enzyme replacement therapy in Fabry disease—A comprehensive review of the medical literature

(2010) Olivier Lidove et al.   GENETICS IN MEDICINE

Overcoming pre-existing adenovirus immunity by genetic engineering of adenovirus-based vectors

(2009) Sergey S Seregin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

(2009) Priya S. Kishnani et al.   MOLECULAR GENETICS AND METABOLISM

Efficient, Long-term Hepatic Gene Transfer Using Clinically Relevant HDAd Doses by Balloon Occlusion Catheter Delivery in Nonhuman Primates

(2008) Nicola Brunetti-Pierri et al.   MOLECULAR THERAPY