Research

Arthritis and Rheumatism Branch

Paul H. Plotz, M.D.
Chief, Arthritis and Rheumatism Branch
Phone: (301) 496-1474
Fax: (301) 402-0012
E-mail: plotzp@mail.nih.gov

The Arthritis and Rheumatism Branch works on basic and clinical aspects of genetic and inflammatory muscle diseases and on basic questions of autoimmunity.


Selected Publications

Raben N, Fukuda T, Gilbert AL, de Jong D, Thurberg BL, Mattaliano RJ, Meikle P, Hopwood JJ, Nagashima K, Nagaraju K, Plotz PH. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers. Mol Ther. 2005; 11(1): 48-56. PubMed Icon

Raben N, Nagaraju K, Lee A, Lu N, Rivera Y, Jatkar T, Hopwood JJ, Plotz PH. Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice. Transgenic Res. 2003; 12(2): 171-8.

Raben N, Jatkar T, Lee A, Plotz P. Lessons learned from an attempt to rescue the phenotype in acid alpha-glucosiclase knockout mice. Am J Hum Genet. 2002; 71(4): 1446 Suppl. S.

Raben N, Lu N, Nagaraju K, Rivera Y, Lee A, Yan B, Byrne B, Meikle PJ, Umapathysivam K, Hopwood JJ, Plotz PH. Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy. Hum Mol Genet. 2001; 10: 2039-47. PubMed Icon

Pauly DF, Fraites TJ, Toma C, Bayes HS, Huie ML, Hirschhorn R, Plotz PH, Raben N, Kessler PD, Byrne BJ. Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease. Hum Gene Ther. 2001; 12(5):527-38. PubMed Icon

See complete list of publications

 

Updated September 17, 2007