ArticleMolecular genetics and metabolism reports2014
Non-depleting anti-CD4 monoclonal antibody induces immune tolerance to ERT in a murine model of Pompe disease.
Article in Molecular genetics and metabolism reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 14 citations in OpenAlex.
- Advances in Immune Tolerance Induction in Enzyme Replacement Therapy.Paediatric drugs · 2024Review
- Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Article
- Molecular Approaches for the Treatment of Pompe Disease.Molecular neurobiology · 2020Review
- Immune reconstitution therapies: concepts for durable remission in multiple sclerosis.Nature reviews. Neurology · 2020Review
- Immunological challenges and approaches to immunomodulation in Pompe disease: a literature review.Annals of translational medicine · 2019Review
- Strategies for the Induction of Immune Tolerance to Enzyme Replacement Therapy in Mucopolysaccharidosis Type I.Molecular therapy. Methods & clinical development · 2019Article
- An immune tolerance approach using transient low-dose methotrexate in the ERT-naïve setting of patients treated with a therapeutic protein: experience in infantile-onset Pompe disease.Genetics in medicine : official journal of the American College of Medical Genetics · 2019Article
- ENPP1 enzyme replacement therapy improves blood pressure and cardiovascular function in a mouse model of generalized arterial calcification of infancy.Disease models & mechanisms · 2018Article
- A pilot study on using rapamycin-carrying synthetic vaccine particles (SVP) in conjunction with enzyme replacement therapy to induce immune tolerance in Pompe disease.Molecular genetics and metabolism reports · 2017Article
- Sustained immune tolerance induction in enzyme replacement therapy-treated CRIM-negative patients with infantile Pompe disease.JCI insight · 2017Article
- The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders.Journal of inherited metabolic disease · 2016Review
- Targeted approaches to induce immune tolerance for Pompe disease therapy.Molecular therapy. Methods & clinical development · 2016Review
- Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade.Human gene therapy · 2015Article
- Effects of immune modulation therapy in the first Croatian infant diagnosed with Pompe disease: a 3-year follow-up study.Wiener klinische Wochenschrift · 2014Article
Corrections and comments
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Authors and funding
10 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
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.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.