SOUTH DRIVE, MSC 0812 50 South Dr Suite 50, Bethesda, MD 20892 3015945283 (Phone)
Certifications:
Internal Medicine, 1970
Awards:
Healthgrades Honor Roll
Languages:
English
Education:
Medical School Harvard Medical School Graduated: 1963 Medical School Beth Israel Deaconess Medical Center Graduated: 1963 Medical School Clin Ctr-Nih Graduated: 1963
Nina N. Raben - N. Bethesda MD, US Cynthia Schreiner - Gowanda NY, US Rebecca Baum - Highland UT, US Shoichi Takikita - Rockville MD, US Tao Xie - Rockville MD, US Paul H. Plotz - Washington DC, US
Assignee:
The United States of America, as Represented by the Secretary, Department of Health and Human Services - Washington DC
International Classification:
A61K 48/00
US Classification:
514 44, 536 245, 536 2431, 536 241
Abstract:
Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT).
Synthesis And Secretion Of Native Recombinant Lysosomal Enzymes By Liver
The invention provides recombinant native lysosomal enzymes produced by liver cells, preferably in vitro, and methods of using the native recombinant lysosomal enzymes to treat enzyme deficiencies in vivo. Lysosomal enzymes, including acid alpha-glucosidase (GAA), produced by liver cells apparently undergo the post-translational modifications necessary to achieve good enzymatic activity. The resulting enzymes can be taken up by various other cells and can correct phenotypic abnormalities of distant organs with enzyme deficiencies. In certain preferred embodiments, the enzyme is GAA and the methods are especially adapted for treatment of type II glycogen storage disease in mammals, including humans.
Disabling Autophagy As A Treatment For Lysosomal Storage Diseases
- , unknown Tao Xie - Rockville MD, US Cynthia Schreiner - Gowanda NY, US Rebecca Baum - Highland UT, US Shoichi Takikita - Rockville MD, US Paul Plotz - Washington DC, US
International Classification:
C12N 15/113
US Classification:
514 11, 514 44 A
Abstract:
Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT).
Nina Raben - Rockville MD Ralph Nichols - Columbia MD Paul Plotz - Washington DC Richard Leff - Charleston SC
Assignee:
The United States of America as represented by the Department of Health and Human Services - Washington DC National Institutes of Health - Bethesda MD
International Classification:
C12N 900 C12N 500 C12P 2106 C07K 100
US Classification:
435183
Abstract:
Recombinant Histidyl tRNA synthetase produced by non-mammalian host cells is used in a sensitive assay to determine the presence of autoimmune diseases in mammals. Methods for isolating, cloning and expressing rHRS are described. In addition, a kit for determining the presence of an autoimmune disease is provided.
Nina Raben - Rockville MD Ralph Nichols - Columbia MD Paul Plotz - Washington DC Richard Leff - Charleston SC
Assignee:
United States of America - Washington DC
International Classification:
G01N 33564
US Classification:
435 74
Abstract:
Recombinant Histidyl tRNA synthetase produced by non-mammalian host cells is used in a sensitive assay to determine the presence of autoimmune diseases in mammals. Methods for isolating, cloning and expressing rHRS are described. In addition, a kit for determining the presence of an autoimmune disease is provided.
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