Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.
Abstract
Approximately 35-40% of patients with classic infantile Pompe disease treated with
enzyme replacement therapy (ERT) develop high, sustained antibody titers against the
therapeutic enzyme alglucosidase alfa, which abrogates the treatment efficacy. Induction
of antigen-specific immune tolerance would greatly enhance ERT for these patients.
Here we show that a short-course treatment with non-depleting anti-CD4 monoclonal
antibody successfully induced long-term ERT-specific immune tolerance in Pompe disease
mice. Our data suggest an effective adjuvant therapy to ERT.
Type
Journal articleSubject
Anti-CD4 antibodyAntigen-specific immune tolerance
CRIM, cross-reacting immunologic material
ERT, enzyme replacement therapy
Enzyme replacement therapy
GAA, acid-α-glucosidase
IPD, infantile Pompe disease
ITI, immune tolerance induction
MTX, methotrexate
Pompe disease
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https://hdl.handle.net/10161/15084Published Version (Please cite this version)
10.1016/j.ymgmr.2014.08.005Publication Info
(2014). Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine
model of Pompe disease. Mol Genet Metab Rep, 1. pp. 446-450. 10.1016/j.ymgmr.2014.08.005. Retrieved from https://hdl.handle.net/10161/15084.This is constructed from limited available data and may be imprecise. To cite this
article, please review & use the official citation provided by the journal.
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Show full item recordScholars@Duke
Priya Sunil Kishnani
Chen Family Distinguished Professor of Pediatrics
RESEARCH INTERESTS A multidisciplinary approach to care of individuals with genetic
disorders in conjunction with clinical and bench research that contributes to: 1)
An understanding of the natural history and delineation of long term complications
of genetic disorders with a special focus on liver Glycogen storage disorders, lysosomal
disorders with a special focus on Pompe disease, Down syndrome and hypophosphatasia2) )
The development of new therapies such
Baodong Sun
Associate Professor in Pediatrics
My overall research interests are finding effective treatment for human glycogen storage
diseases (GSDs) and other inherited metabolic disorders. My current research focuses
on identification of novel therapeutic targets and development of effective therapies
for GSD II (Pompe disease), GSD III (Cori disease), and GSD IV (Andersen disease)
using cellular and animal disease models. The main therapeutic approaches we are using
in our pre-clinical studie
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