Title:
GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA
Document Type and Number:
Japanese Patent JP2022160505
Kind Code:
A
Abstract:
To provide compositions and methods for restoring FANCA expression in cells with diminished or abolished FANCA gene products, particularly, methods and compositions for the gene therapy of Fanconi anemia.SOLUTION: Provided is an expression cassette comprising a polynucleotide sequence comprising in order from 5' to 3': (a) a human phosphoglycerate kinase (PGK) promoter sequence or a functional homologue or variant thereof; (b) a sequence encoding a human FANCA polypeptide or a functional fragment or variant thereof; (c) a woodchuck hepatitis virus regulatory element (WPRE) RNA transport signal sequence or a functional variant or fragment thereof, the sequence encoding a human FANCA polypeptide or a functional fragment or variant thereof being operably linked to the PGK promoter sequence.SELECTED DRAWING: Figure 1
Inventors:
Buren Huan Ei.
Rio Paula
Navarro Susana
Sevilla Julian
Segovia Jose Carlos
gonzalez africa
Cassard Jose Antonio
Genechea Guillermo
Rio Paula
Navarro Susana
Sevilla Julian
Segovia Jose Carlos
gonzalez africa
Cassard Jose Antonio
Genechea Guillermo
Application Number:
JP2022117708A
Publication Date:
October 19, 2022
Filing Date:
July 25, 2022
Export Citation:
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
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; A61K35/28; A61K38/17; A61P7/06; A61P43/00; C12N5/10; C12N15/867
Domestic Patent References:
| JP2014513727A | 2014-06-05 | |||
| JP2007054069A | 2007-03-08 |
Foreign References:
| US20120172418A1 | 2012-07-05 | |||
| WO2015168547A2 | 2015-11-05 | |||
| US5952190A | 1999-09-14 | |||
| US20150203852A1 | 2015-07-23 | |||
| US20120071859A1 | 2012-03-22 | |||
| US20090111106A1 | 2009-04-30 | |||
| WO2015056014A1 | 2015-04-23 | |||
| WO2016118780A1 | 2016-07-28 |
Other References:
HUMAN GENE THERAPY, vol. 21, JPN6021016830, 2010, pages 1 - 8, ISSN: 0005204018
BECKER PS; ET AL: "PRECLINICAL CORRECTION OF HUMAN FANCONI ANEMIA COMPLEMENTATION GROUP A BONE MARROW CELLS 以下備考", GENE THERAPY, vol. VOL:17, NR:10, JPN5019008397, 2010, pages 1244 - 1252, ISSN: 0005204019
MOLECULAR THERAPY, vol. 19, no. 7, JPN6021016929, 2011, pages 1193 - 1198, ISSN: 0005204020
BECKER PS; ET AL: "PRECLINICAL CORRECTION OF HUMAN FANCONI ANEMIA COMPLEMENTATION GROUP A BONE MARROW CELLS 以下備考", GENE THERAPY, vol. VOL:17, NR:10, JPN5019008397, 2010, pages 1244 - 1252, ISSN: 0005204019
MOLECULAR THERAPY, vol. 19, no. 7, JPN6021016929, 2011, pages 1193 - 1198, ISSN: 0005204020
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
Masao Haruna
Hirotaka Yamaguchi
Shun Jinbe
Ryuichi Inoue
Toshimitsu Sato
Koichi Niimi
Tomohiko Kobayashi
Hideki Kodera
Masato Ozeki
Kazuya Kawamoto