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Research
Arthrits and Rheumatism Branch
Staff Scientist
Phone: 301-496-1474
Building: 50, Room: 1345
E-mail: rabenn@mail.nih.gov
Nina Raben was born in Moscow, Russia (the former Soviet Union). She received her medical degree from the Moscow Medical Institute, and her Ph.D. degree in Biochemistry from the National Institute of Surgery, Academy of Medical Science, Moscow. She joined NIDDK in 1987, and since 1990 has been working in the Arthritis and Rheumatism Branch, NIAMS, in the laboratory of Dr. Paul Plotz. The major focus of her research in recent years is a genetic disorder caused by a deficiency of lysosomal enzyme acid alpha-glucosidase, known as Glycogen storage disease type II or Pompe disease. The studies include extensive mutational analysis of the gene, development of several knockout and transgenic mouse models of the disease, and pre-clinical studies with recombinant human enzyme likely to be used in clinical trials.
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.
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, Danon M, Gilbert AL, Dwivedi S, Collins B, Thurberg BL, Mattaliano RJ, Nagaraju K, Plotz PH. Enzyme replacement therapy in the mouse model of Pompe disease. Mol Genet Metab. 2003; 80(1-2):159-69.
Martiniuk F, Chen A, Mack A, Donnabella V, Slonim A, Bulone L, Arvanitopoulos E, Raben N, Plotz P, Rom WN. Helios gene gun particle delivery for therapy of acid maltase deficiency. DNA Cell Biol. 2002; 21(10): 717-25
Dwivedi S, Chang J, Raben N, Collins B, Nagaraju K, Plotz P. Liver and skeletal muscle cells are differentially susceptible to MHC class I over-expression. FASEB J. 17(7): C38-C38 Suppl. S.
