Title:
PROPHYLACTIC AGENT AND/OR THERAPEUTIC AGENT FOR NEUROLOGICAL DISORDERS INCLUDING AMYOTROPHIC LATERAL SCLEROSIS
Document Type and Number:
WIPO Patent Application WO/2020/022518
Kind Code:
A1
Abstract:
This prophylactic agent and/or therapeutic agent for neurological disorders including amyotrophic lateral sclerosis comprises necdin as an effective component. The prophylactic agent and/or therapeutic agent may be: necdin or a variant thereof; a derivative or modified form of necdin or a variant thereof; a substance containing a polynucleotide coding for necdin; or a compound that increases the abundance or the functionality/activity of necdin.
Inventors:
MOCHIZUKI HIDEKI (JP)
NAGANO SEIICHI (JP)
SASAKI TSUTOMU (JP)
CHOONG CHI-JING (JP)
OKADA TAKASHI (JP)
HIRAI YUKIHIKO (JP)
NAGANO SEIICHI (JP)
SASAKI TSUTOMU (JP)
CHOONG CHI-JING (JP)
OKADA TAKASHI (JP)
HIRAI YUKIHIKO (JP)
Application Number:
PCT/JP2019/029590
Publication Date:
January 30, 2020
Filing Date:
July 29, 2019
Export Citation:
Assignee:
UNIV OSAKA (JP)
NIPPON MEDICAL SCHOOL FOUNDATION (JP)
NIPPON MEDICAL SCHOOL FOUNDATION (JP)
International Classes:
A61K38/17; A61K35/76; A61K48/00; A61P21/02; A61P25/00; A61P43/00
Other References:
HASEGAWA, K. ET AL.: "Promotion of mitochondrial biogenesis by necdin protects neurons against mitochondrial insults.", NATURE COMMUNICATIONS, vol. 7, no. 10943, 2016, pages 1 - 15, XP055681706
SASAKI, TSUTOMU ET AL.: "Neuroprotective effects of Necdin in the Parkinson's disease", JAPANESE JOURNAL OF CLINICAL MEDICINE, vol. 75, no. 1, 2017, pages 36 - 41, ISSN: 0047-1852
YASUDA, TORU ET AL.: "New treatment for Parkinson's disease -Inhibition of neuronal cell death by necdin", JOURNAL OF CLINICAL AND EXPERIMENTAL MEDICINE, vol. 261, no. 9, 2017, pages 894 - 895, ISSN: 0039-2359
AEBISCHER, J. ET AL.: "Necdin protects embryonic motoneurons from programmed cell death.", PLOS ONE, vol. 6, no. 9, 2011, pages 1 - 13, XP055681715
ZHAO, W. ET AL.: "Peroxisome proliferator activator receptor gamma coactivator-lalpha (PGC-1 a) improves motor performance and survival in a mouse model of amyotrophic lateral sclerosis", MOLECULAR NEURODEGENERATION, vol. 6, no. 1, 2011, pages 1 - 8, XP021105965, DOI: 10.1186/1750-1326-6-51
LIANG, H. ET AL.: "PGC-1 alpha protects neurons and alters disease progression in an amyotrophic lateral sclerosis mouse model.", MUSCLE & NERVE, vol. 44, no. 6, 2011, pages 947 - 956, XP055681719
SONG, W. ET AL.: "Mutant SOD1G93A triggers mitochondrial fragmentation in spinal cord motor neurons: neuroprotection by SIRT3 and PGC-1 a.", NEUROBIOLOGY OF DISEASE, vol. 51, 2013, pages 72 - 81, XP055681739
DUQUE, S. ET AL.: "Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.", MOLECULAR THERAPY, vol. 17, no. 7, 2009, pages 1187 - 1196, XP055103985, DOI: 10.1038/mt.2009.71
BENKHELIFA-ZIYYAT, S. ET AL.: "Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.", MOLECULAR THERAPY, vol. 21, no. 2, 2013, pages 282 - 290, XP055293194, DOI: 10.1038/mt.2012.261
FERRAIUOLO, L. ET AL.: "Microarray analysis of the cellular pathways involved in the adaptation to and progression of motor neuron injury in the SOD1 G93A mouse model of familial ALS.", JOURNAL OF NEUROSCIENCE, vol. 27, no. 34, 2007, pages 9201 - 9219
SASAKI, TSUTOMU ET AL.: "Neuroprotective effects of Necdin in the Parkinson's disease", JAPANESE JOURNAL OF CLINICAL MEDICINE, vol. 75, no. 1, 2017, pages 36 - 41, ISSN: 0047-1852
YASUDA, TORU ET AL.: "New treatment for Parkinson's disease -Inhibition of neuronal cell death by necdin", JOURNAL OF CLINICAL AND EXPERIMENTAL MEDICINE, vol. 261, no. 9, 2017, pages 894 - 895, ISSN: 0039-2359
AEBISCHER, J. ET AL.: "Necdin protects embryonic motoneurons from programmed cell death.", PLOS ONE, vol. 6, no. 9, 2011, pages 1 - 13, XP055681715
ZHAO, W. ET AL.: "Peroxisome proliferator activator receptor gamma coactivator-lalpha (PGC-1 a) improves motor performance and survival in a mouse model of amyotrophic lateral sclerosis", MOLECULAR NEURODEGENERATION, vol. 6, no. 1, 2011, pages 1 - 8, XP021105965, DOI: 10.1186/1750-1326-6-51
LIANG, H. ET AL.: "PGC-1 alpha protects neurons and alters disease progression in an amyotrophic lateral sclerosis mouse model.", MUSCLE & NERVE, vol. 44, no. 6, 2011, pages 947 - 956, XP055681719
SONG, W. ET AL.: "Mutant SOD1G93A triggers mitochondrial fragmentation in spinal cord motor neurons: neuroprotection by SIRT3 and PGC-1 a.", NEUROBIOLOGY OF DISEASE, vol. 51, 2013, pages 72 - 81, XP055681739
DUQUE, S. ET AL.: "Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.", MOLECULAR THERAPY, vol. 17, no. 7, 2009, pages 1187 - 1196, XP055103985, DOI: 10.1038/mt.2009.71
BENKHELIFA-ZIYYAT, S. ET AL.: "Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.", MOLECULAR THERAPY, vol. 21, no. 2, 2013, pages 282 - 290, XP055293194, DOI: 10.1038/mt.2012.261
FERRAIUOLO, L. ET AL.: "Microarray analysis of the cellular pathways involved in the adaptation to and progression of motor neuron injury in the SOD1 G93A mouse model of familial ALS.", JOURNAL OF NEUROSCIENCE, vol. 27, no. 34, 2007, pages 9201 - 9219
Attorney, Agent or Firm:
IWATANI, Ryo (JP)
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