Title:
THERAPY FOR α-GALACTOSIDASE A DEFICIENCY
Document Type and Number:
Japanese Patent JP2015044830
Kind Code:
A
Abstract:
PROBLEM TO BE SOLVED: To provide human cells for implantation genetically modified to overexpress and secrete human α-gal A, and purified human α-gal A, for treating an individual suspected of having an α-galactosidase A deficiency, such as Fabry disease.SOLUTION: Compositions for therapy are provided for treating with either (1) human cells genetically modified to overexpress and secrete human α-gal A, or (2) purified human α-gal A obtained from cultured, genetically modified human cells.
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Inventors:
SELDEN RICHARD F
BOROWSKI MARIANNE
GILLESPIE FRANCES P
KINOSHITA CAROL M
TRECO DOUGLAS A
WILLIAMS MELANIE D
BOROWSKI MARIANNE
GILLESPIE FRANCES P
KINOSHITA CAROL M
TRECO DOUGLAS A
WILLIAMS MELANIE D
Application Number:
JP2014210566A
Publication Date:
March 12, 2015
Filing Date:
October 15, 2014
Export Citation:
Assignee:
SHIRE HUMAN GENETIC THERAPIES
International Classes:
A61K35/12; C12N15/09; A61K38/46; A61K38/47; A61K47/24; A61K47/42; A61K48/00; A61P1/14; A61P9/00; A61P13/12; A61P35/00; A61P43/00; C07K14/61; C07K19/00; C12N5/10; C12N9/20; C12N9/40; C12N15/56; C12N15/62; A61K38/00; C12R1/91
Domestic Patent References:
| JPH01165373A | 1989-06-29 | |||
| JPH08503615A | 1996-04-23 | |||
| JPH06503479A | 1994-04-21 | |||
| JPH03219891A | 1991-09-27 | |||
| JPH0216984A | 1990-01-19 | |||
| JPH08196293A | 1996-08-06 | |||
| JPH0870861A | 1996-03-19 |
Foreign References:
| WO1996008570A1 | 1996-03-21 | |||
| WO1995001428A1 | 1995-01-12 | |||
| WO1996025498A2 | 1996-08-22 |
Other References:
JPN6011036281; Ｐｒｏｃ Ｎａｔｌ Ａｃａｄ Ｓｃｉ Ｕ Ｓ Ａ Ｖｏｌ．９３， Ｎｏ．１５, 1996, ｐ．７９１７-２２
JPN6011036283; Ｅｕｒ Ｊ Ｂｉｏｃｈｅｍ Ｖｏｌ．１６５， Ｎｏ．２, 1987, ｐ．２７５-８０
JPN6011036283; Ｅｕｒ Ｊ Ｂｉｏｃｈｅｍ Ｖｏｌ．１６５， Ｎｏ．２, 1987, ｐ．２７５-８０
Attorney, Agent or Firm:
Spring water Original aim