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Title:
ファンコニ貧血患者に対する遺伝子療法
Document Type and Number:
Japanese Patent JP7197466
Kind Code:
B2
Abstract:
The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed.

Inventors:
Buren Huan Ei.
Rio Paula
Navarro Susana
Sevilla Julian
Segovia Jose Carlos
gonzalez africa
Cassard Jose Antonio
Genechea Guillermo
Application Number:
JP2019513876A
Publication Date:
December 27, 2022
Filing Date:
September 08, 2017
Export Citation:
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Assignee:
Centro de Investigacyons Enerjetikas Medio Ambientus Ye Technology Cass O.A.M.P.
FUNDACION INSTITUTO DE INVESTIGACION SANITARIA FUNDACION JIMENEZ DIAZ
Consorcio Centro de Investigación Biomedica en Red
Fundacion Para La Investigacion Biomedica del Hospital Infantil Universitario Niño Jesús
International Classes:
C12N15/12; A61K35/15; A61K35/28; A61K38/02; A61K48/00; A61P7/06; A61P43/00; C12N5/0789; C12N5/10; C12N15/867
Domestic Patent References:
JP2007054069A
JP2014513727A
Foreign References:
US5952190
US20150203852
US20120071859
US20090111106
WO2015056014A1
US20120172418
WO2015168547A2
WO2016118780A1
Other References:
HUMAN GENE THERAPY,2010年,Vol.21,p.1-8,DOI:10.1089/hum.2009.141
Gene Ther.,2010年,17(10),p.1244-1252
Molecular Therapy,2011年,Vol.19, No.7,p.1193-1198,doi:10.1038/mt.2011.78
Attorney, Agent or Firm:
Shimizu Hatsushi
Masao Haruna
Hirotaka Yamaguchi
Shun Jinbe
Ryuichi Inoue
Toshimitsu Sato
Koichi Niimi
Tomohiko Kobayashi
Hideki Kodera
Masato Ozeki
Kazuya Kawamoto