鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン

Title:

鎌状赤血球病の遺伝子的是正のための改善された胎児ヘモグロビン

Document Type and Number:

Japanese Patent JP6509884

Kind Code:

B2

Abstract:

Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increasing a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention also relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia or β-thalassemia.

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Inventors:

Malik Punnam

Application Number:

JP2016549495A

Publication Date:

May 08, 2019

Filing Date:

January 30, 2015

Export Citation:

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Assignee:

Children's Hospital Medical Center

International Classes:

C12N15/12; A61K35/28; A61K35/76; A61K48/00; A61P7/06

Domestic Patent References:

JP7504645A
JP2004537539A
JP2014530002A
JP2003517312A

Foreign References:

US20110294114

Attorney, Agent or Firm:

Hatsushi Shimizu
Masao Haruna
Hirotaka Yamaguchi
Toshi Gobe
Ryuichi Inoue
Toshimitsu Sato
Koichi Niimi
Tomohiko Kobayashi
Hideki Kodera
Masato Ozeki
Yoshihiro Igarashi
Kazuya Kawamoto

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