Title:
ファブリー病の遺伝子治療
Document Type and Number:
Japanese Patent JP6688812
Kind Code:
B2
Abstract:
There is described a nucleic acid molecule comprising a nucleotide sequence encoding for a functional ±-galactosidase A protein wherein the nucleotide sequence has at least 85% identity to the sequence of SEQ ID NO. 1. Also described is a vector, host cell or transgenic animal comprising the nucleic acid molecule; and a pharmaceutical composition comprising the nucleic acid molecule or the vector. Further, the use of the nucleic acid molecule in a method of treating Fabry disease is described.
Inventors:
Eggplant, Amit
Raj, Deepak
Raj, Deepak
Application Number:
JP2017558727A
Publication Date:
April 28, 2020
Filing Date:
May 10, 2016
Export Citation:
Assignee:
UCL Business Limited
International Classes:
C12N15/56; A01K67/027; A61K35/761; A61K48/00; A61P3/06; A61P9/00; A61P9/10; A61P13/12; C12N1/15; C12N1/19; C12N1/21; C12N5/10; C12N15/63
Foreign References:
| US20040204379 |
Other References:
Molecular Therapy,2007, Vol.15, No.3,pp.492-500
PLoS Genetics,2006, Vol.2, No.12,e221, pp.2107-2115
PNAS,2004, Vol.101, No.34,pp.12588-12591
Cell,2015, Vol.160, No.6,pp.1111-1124
PLoS Genetics,2006, Vol.2, No.12,e221, pp.2107-2115
PNAS,2004, Vol.101, No.34,pp.12588-12591
Cell,2015, Vol.160, No.6,pp.1111-1124
Attorney, Agent or Firm:
Takashima Ichi
Kyoko Doi
Mitsunori Kamata
Yaeko Tamura
Junzo Koike
Hirofumi Toma
Atsuko Akai
Tomiya Tozaki
Kyoko Doi
Mitsunori Kamata
Yaeko Tamura
Junzo Koike
Hirofumi Toma
Atsuko Akai
Tomiya Tozaki