Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade-Reference-Cited by-同舟云学术

Enhanced Efficacy from Gene Therapy in Pompe Disease Using Coreceptor Blockade

Published:2015-01 Issue:1 Volume:26 Page:26-35
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Han Sang-oh¹,Li Songtao¹,Brooks Elizabeth D.¹,Masat Elisa²,Leborgne Christian³,Banugaria Suhrad⁴,Bird Andrew¹,Mingozzi Federico²³,Waldmann Herman⁵,Koeberl Dwight¹

Affiliation:

1. Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710.

2. University Pierre and Marie Curie–Paris 6, 75005 Paris, France.

3. Genethon, 910020 Evry, France.

4. Department of Pediatrics, New Jersey Medical School, Rutgers University, Newark, NJ 07103.

5. Sir William Dunn School of Pathology, University of Oxford, Oxford OX1 3RE, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2014.115

Reference61 articles.

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

2. Pompe Disease in Infants: Improving the Prognosis by Newborn Screening and Early Treatment

3. High antibody titer in an adult with Pompe disease affects treatment with alglucosidase alfa

4. 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

5. Recombinant human acid ??-glucosidase enzyme therapy for infantile glycogen storage disease type II: Results of a phase I/II clinical trial

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