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Review

. 2007 Jul;26(1):49-52.

Progress and problems when considering gene therapy for GSD-II

A Kiang¹, A Amalfitano

Affiliations

PMID: 17915570
PMCID: PMC2949318

Review

Progress and problems when considering gene therapy for GSD-II

A Kiang et al. Acta Myol. 2007 Jul.

. 2007 Jul;26(1):49-52.

Authors

A Kiang¹, A Amalfitano

Affiliation

¹ Department of Microbiology and Molecular Genetics, Michigan State University, East Lansing, MI 48824, USA.

PMID: 17915570
PMCID: PMC2949318

No abstract available

PubMed Disclaimer

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References

1. Kikuchi T, Yang HW, Pennybacker M, et al. Clinical and metabolic correcton of Pompe disease by enzyme therapy in acid maltase-deficient quail. J Clin Invest 1998;101:827-33. - PMC - PubMed
1. Van den Hout H, Reuser AJ, Vulto AG, et al. Recombinant human alpha-glucosidase from rabbit milk in Pompe patients. Lancet 2000;356:397-8. - PubMed
1. Vanhove JLK, Yang HW, Wu JY, Brady RO, Chen YT. High-level production of recombinant human lysosomal acid alpha-glucosidase in chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease. Proc Natl Acad Sci USA 1996;93:65-70. - PMC - PubMed
1. Amalfitano A, Bengur AR, Morse RP, et al. Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I/II clinical trial. Gen Med 2001;3:132-8. - PubMed
1. Amalfitano A. Utilization of adenovirus vectors for multiple gene transfer applications. Methods 2004;33:173-8. - PubMed

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