肝臓ゲノム上の凝固関連因子遺伝子を破壊するためのＡＡＶベクター

Japanese Patent JP7215716

B2

The present invention provides a recombinant adeno-associated virus (rAAV) vector for treating a blood coagulation-related disease to provide a novel gene therapy means for hemophilia. The virus vector comprises a virus genome comprising a liver-specific promoter sequence and a polynucleotide sequence encoding a genome editing means operably linked to the promoter sequence, wherein the genome editing means is (a) a means comprising CRISPR/Cas9 composed of a Cas9 protein and a guide RNA (gRNA) and a repair gene, or (b) a means comprising CRISPR/Cas9 composed of a Cas9 protein and a gRNA, and the gRNA comprises a nucleotide region complementary to a part of a region related to expression of a disease-related protein on the genome of a patient and a region that interacts with the Cas9 protein.

Omori Tsukasa
Yasumitsu Nagao
Hiroaki Mizukami
Asuka Sakata
Takaya Ozawa
Shinichi Muramatsu
Tominaga Shinichi
Yutaka Hanazono
Satoshi Nishimura
Yoichi Sakata
Nobuhiko Kamoshita

JP2018561352A

January 31, 2023

January 09, 2018

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Jichi Medical School

C12N15/864; A61K31/7088; A61K35/76; A61K48/00; A61P1/16; A61P7/04; A61P43/00; C12N15/09

Nature,2011年,Vol.475,P.217-223
Blood,2013年,Vol.122,P.3283-3287
Frontiers in Haemophilia,2016年,Vol.3,P.19-23
日本血栓止血学会誌,2015年,Vol.26,P.534-540
SEHGAL,A., et al.,NATURE MEDICINE,2015年,Vol.21,p.492-497
nature biotechnology,2014年,Vol.32,P.551-553

Hiroshi Kobayashi
Yasuhito Suzuki

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