Title:
CRISPR−CAS9改変CD34+ヒト造血幹・前駆細胞及びその使用
Document Type and Number:
Japanese Patent JP2021505587
Kind Code:
A
Abstract:
本明細書では、一部の実施形態において、自家CRISPR−Cas9改変CD34+ヒト造血幹・前駆細胞を用いて、β−サラセミアを有する対象及び鎌状赤血球症を有する対象を処置するための方法及び組成物が提供される。【選択図】図2
Inventors:
Eberina Moraba
Tisa Chakra Beauty
Antes Ben Lund Bali
Tony Ho
Laura Sandler
Brenda Eustas
Jerome Rossart
Robert Kaufman
Tisa Chakra Beauty
Antes Ben Lund Bali
Tony Ho
Laura Sandler
Brenda Eustas
Jerome Rossart
Robert Kaufman
Application Number:
JP2020530625A
Publication Date:
February 18, 2021
Filing Date:
December 05, 2018
Export Citation:
Assignee:
VERTEX PHARMACEUTICALS INCORPORATED
International Classes:
A61K35/12; A61K38/46; A61P7/00; A61P7/06; C12N5/10
Domestic Patent References:
JP2000201672A | 2000-07-25 |
Foreign References:
WO2017182881A2 | 2017-10-26 | |||
WO2016182917A1 | 2016-11-17 | |||
WO2017115268A1 | 2017-07-06 | |||
WO2016183298A2 | 2016-11-17 |
Other References:
GIBCO, SYNTH-A-FREEZE, 製品マニュアル, 2010, PP. 1-2, JPN6022039551, ISSN: 0004877222
Attorney, Agent or Firm:
Atsushi Aoki
Shinji Mitsuhashi
Yoichi Watanabe
Masaru Nakajima
Ryotaro Takei
Junji Shibata
Shinji Mitsuhashi
Yoichi Watanabe
Masaru Nakajima
Ryotaro Takei
Junji Shibata