LENTIVIRAL VECTOR, CELL AND CELL PREPARATION

To provide a lentiviral vector that can be used in gene therapy of GM1-gangliosidosis.SOLUTION: There is provided a lentiviral vector comprising LV-SMPUR-MCU3-cGLB1 and including a base sequence described in SEQ ID NO: 1. GLB1 being a responsible gene is introduced into the lentiviral vector of the...

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Bibliographic Details
Main Authors TSUNOGAI TOSHIKI, KOBAYASHI HIROSHI
Format Patent
LanguageEnglish
Japanese
Published 25.04.2023
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
VINEGAR
WINE
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Summary:To provide a lentiviral vector that can be used in gene therapy of GM1-gangliosidosis.SOLUTION: There is provided a lentiviral vector comprising LV-SMPUR-MCU3-cGLB1 and including a base sequence described in SEQ ID NO: 1. GLB1 being a responsible gene is introduced into the lentiviral vector of the present invention. The lentiviral vector of the present invention is introduced into a hematopoietic stem cell.SELECTED DRAWING: Figure 1 【課題】GM1ガングリオシドースの遺伝子治療に使用できるレンチウイルスベクターを提供する。【解決手段】LV-SMPUR-MCU3-cGLB1からなるレンチウイルスベクターであって、配列番号１に記載の塩基配列を含むレンチウイルスベクターである。本発明のレンチウイルスベクターには責任遺伝子であるGLB1が導入される。造血幹細胞に本発明のレンチウイルスベクターが導入される。【選択図】図１
Bibliography:Application Number: JP20210167873