Title:
ムコリピドーシスＩＩ型を治療するためのアデノ随伴ウイルスベクター
Document Type and Number:
Japanese Patent JP7347932
Kind Code:
B2
Abstract:
Provided herein are recombinant adeno-associated virus (rAAV) vectors comprising nucleic acid encoding N-acetyl-glucosamine-1-phosphate transferase, alpha and beta subunits (GNPTAB) and at least one AAV inverted terminal repeat (ITR). In some embodiments, the rAAV vectors may be included in a rAAV particle, which may be contained in pharmaceutical compositions and kits. These vectors, particles, compositions, and kits may find use, inter alia, in methods and uses related to treating mucolipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal, or related to increasing body size, bone mineral content, and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).
Inventors:
Nelson Ew
Ching Tong-kyu
Ching Tong-kyu
Application Number:
JP2018531240A
Publication Date:
September 20, 2023
Filing Date:
December 14, 2016
Export Citation:
Assignee:
GENZYME CORPORATION
Samsung Life Public Welfare Foundation
Samsung Life Public Welfare Foundation
International Classes:
C12N15/864; A61K35/76; A61K48/00; A61P1/10; A61P1/12; A61P9/00; A61P11/00; A61P11/16; A61P19/00; A61P21/00; A61P25/28; A61P31/00; A61P43/00; C12N7/01
Foreign References:
| WO2015060722A1 |
Other References:
J Inherit.Metab.Dis.,vol.38,Suppl.1,S245(2015)
Attorney, Agent or Firm:
Junji Yuda
Noriyuki Takebayashi
Noriyuki Takebayashi