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Title:
COMPOSITIONS AND METHODS FOR DEGRADATION OF MISFOLDED PROTEINS
Document Type and Number:
Japanese Patent JP2022000459
Kind Code:
A
Abstract:
To provide a novel composition for promoting the degradation of misfolded proteins and protein aggregates in the nucleus.SOLUTION: There is provided a composition for treating or preventing a disease or disorder associated with misfolded protein or protein aggregates. The composition comprises a modulator of one or more TRIM proteins. The modulator is preferably an isolated peptide comprising a TRIM protein or an isolated nucleic acid molecule encoding the TRIM protein, further preferably comprising a cell membrane-penetrating peptide (CPP). The composition is useful in the treatment of a neurodegenerative disease or disorder selected from the group consisting of Spinocerebellar ataxia (SCA), Huntington's disease, Dentatorubral-pallidoluysian atrophy (DRPLA), Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), a transmissible spongiform encephalopathy (prion disease), a tauopathy, and Frontotemporal lobar degeneration (FTLD).SELECTED DRAWING: Figure 1A

Inventors:
YANG XIAOLU
GUO LILI
Application Number:
JP2021155978A
Publication Date:
January 04, 2022
Filing Date:
September 24, 2021
Export Citation:
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Assignee:
UNIV PENNSYLVANIA
International Classes:
A61K45/00; A61K31/7088; A61K31/7105; A61K38/02; A61K39/395; A61K47/62; A61K48/00; A61P3/10; A61P7/04; A61P9/00; A61P9/10; A61P11/00; A61P17/00; A61P21/00; A61P25/02; A61P25/14; A61P25/16; A61P25/28; A61P27/02; A61P27/12; A61P29/00; A61P35/00; A61P43/00; C07K14/16; C07K14/47; C07K16/18; C07K16/40; C07K19/00; C12N15/11; C12N15/113; C12N15/12; C12N15/63; C12P21/02
Foreign References:
US20090208455A12009-08-20
WO2014086835A12014-06-12
Other References:
EUROPEAN JOURNAL OF NEUROSCIENCE, vol. 17, JPN6020018362, 2003, pages 1150 - 1158, ISSN: 0005098951
MOLECULAR CELL, vol. 55, JPN6020018363, 2014, pages 15 - 30, ISSN: 0005098952
MOLECULAR BIOLOGY OF THE CELL, vol. 24, JPN6020018364, 2013, pages 74 - 84, ISSN: 0005098953
MAGIERA, M. M., ET AL.: "Trim17-mediated ubiquitination and degradation of Mcl-1 initiate apoptosis in neurons", CELL DEATH AND DIFFERENTIATION, vol. 20, no. 2, JPN6023027472, 2013, pages 281 - 292, ISSN: 0005098954
医学のあゆみ, vol. 243, no. 6, JPN6023027471, 2012, pages 508 - 514, ISSN: 0005098955
Attorney, Agent or Firm:
Patent business corporation Tsukuni