Department / Division Affiliations
Member,
Brain Research Institute
Professor Emerita,
Biological Chemistry
Research Interest:
The cause and treatment of the Sanfilippo syndrome
The Sanfilippo syndrome is a rare genetic disease of children, characterized by profound mental retardation and death in late adolescence. We study a mouse model of the disease to understand why the brain is so severely affected and to develop treatment.
Biography:
Born in France, Elizabeth Neufeld immigrated to the United States in 1940. She obtained a BS from Queens College, New York and a Ph.D. from the University of California Berkeley. After postdoctoral training in, she moved to the NIH in Bethesda, MD, where she began her studies of a rare group of genetic diseases. She moved back to California in 1984 as Chair of the Department of Biological Chemistry - a position that she occupied till 2004.
Publications:
Neufeld, Elizabeth F. From serendipity to therapy.
Annual Reviews of Biochemistry
2011; 80:
1-15.
Kakkis E.D., Muenzer J., Tiller G.E., Waber L., Belmont J., Passage M., Izykowski B., Phillips J., Doroshow R., Walot I., Hoft R., Neufeld E.F. Enzyme- Replacement therapy in mucopolysaccharidosis I..
N. Engl. J. Med.
2001; 344:
182-188.
Neufeld E.F., Muenzer J. The Mucopolysaccharidoses..
The Metabolic and Molecular Bases of Inherited Disease
2001; Eighth edition:
3421-3452.
Li H.H., Yu W.H., Rozengurt N., Zhao H.Z., Lyons K.M., Anagnostaras S., Fanselow M.S., Suzuki K.. Vanier M.T., Neufeld E.F. Mouse model of Sanfilippo syndrome type B producedby targeted disruption of the gene encoding alpha-N-acetylglucosaminidase..
Proc. Natl. Acad. Sci. USA
1999; 96:
14505-14510.
Chen Chi-hong B, Dellamaggiore Kenneth R, Ouellette Christopher P, Sedano Cecilia D, Lizadjohry Meikana, Chernis George A, Gonzales Michelle, Baltasar Francis E, Fan Audrey L, Myerowitz Rachel, Neufeld Elizabeth F Aptamer-based endocytosis of a lysosomal enzyme..
Proceedings of the National Academy of Sciences of the United States of America.
2008; 105(41):
15908-13.
Jordan M.C., Zheng Y., Ryazantsev S., Rozengurt N., Roos K.P., Neufeld E.F. Cardiac manifestations in the mouse model of mucopolysaccharidosis I..
Molecular genetics and metabolism.
2005; 86(1-2):
233-43.
Ryazantsev S., Yu W.H., Zhao H.Z., Neufeld E.F., Ohmi K. Lysosomal accumulation of SCMAS (subunit c of mitochondrial ATP synthase) in neurons of the mouse model of mucopolysaccharidosis III B..
Molecular genetics and metabolism.
2007; 90(4):
393-401.
Zheng Y., Ryazantsev S., Ohmi K., Zhao H.Z., Rozengurt N., Kohn D.B., Neufeld E.F. Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB..
Molecular genetics and metabolism.
2004; 82(4):
286-95.
Zheng Y., Rozengurt N., Ryazantsev S., Kohn D.B, Satake N., Neufeld E.F. Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow..
Molecular genetics and metabolism.
2003; 79(4):
233-44.
Li H.H., Zhao H.Z., Neufeld E.F., Cai Y. Gómez-Pinilla F. Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo syndrome type B..
Journal of neuroscience research
2002; 69(1):
30-8.
Yu W.H., Zhao K.W., Ryazantsev S., Rozengurt N., Neufeld E.F. Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B..
Molecular genetics and metabolism. .
2000; 71(4):
573-80.
Zhao K.W., Neufeld E.F. Purification and characterization of recombinant human alpha-N-acetylglucosaminidase secreted by Chinese hamster ovary cells..
Protein expression and purification. .
2000; 19(1):
202-11.
Schmidtchen A., Greenberg D., Zhao H.G., Li H.H., Huang Y., Tieu P., Zhao H.Z., Cheng S. Zhao Z., Whitley C.B., Di Natale P., Neufeld E.F. NAGLU mutations underlying Sanfilippo syndrome type B..
American journal of human genetics. .
1998; 62(1):
64-9.
Zhao K.W., Faull K.F., Kakkis E.D., Neufeld E.F. Carbohydrate structures of recombinant human alpha-L-iduronidase secreted by Chinese hamster ovary cells..
The Journal of biological chemistry. .
1997; 272(36):
22758-65.
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