Title:
ヘモグロビン異常症を処置するためのグロビン遺伝子療法
Document Type and Number:
Japanese Patent JP7158427
Kind Code:
B2
Abstract:
The presently disclosed subject matter provides for expression cassettes that allow for expression of a globin gene or a functional portion thereof, vectors comprising thereof, and cells transduced with such expression cassettes and vectors. The presently disclosed subject matter further provides methods for treating a hemoglobinopathy in a subject comprising administering an effective amount of such transduced cells to the subject.
Inventors:
Mitchell Saderain
Isabel Riviere
Giorgi Mansilla-Soto
Su Yung Wan
George Stamatyanopoulos
John Stamatoyanopoulos
Mindon Riu
Isabel Riviere
Giorgi Mansilla-Soto
Su Yung Wan
George Stamatyanopoulos
John Stamatoyanopoulos
Mindon Riu
Application Number:
JP2020035649A
Publication Date:
October 21, 2022
Filing Date:
March 03, 2020
Export Citation:
Assignee:
Memorial Sloan Kettering Cancer Center
University of Washington
University of Washington
International Classes:
A61K48/00; A61K9/127; A61K31/7088; A61K38/17; C12N15/11
Domestic Patent References:
| JP2002536965A |
Foreign References:
| WO2014026110A2 |
Other References:
Cell, 2007, Vol.128, pp.1231-1245
Semin. Hematol., 2004, Vol.41, pp.279-286
Stem cell Rev. and Rep., 2013, Vol.9, pp.397-407
Semin. Hematol., 2004, Vol.41, pp.279-286
Stem cell Rev. and Rep., 2013, Vol.9, pp.397-407
Attorney, Agent or Firm:
Shusaku Yamamoto
Morishita Natsuki
Morishita Natsuki