JPWO2019200122A5 - - Google Patents

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JPWO2019200122A5
JPWO2019200122A5 JP2020555222A JP2020555222A JPWO2019200122A5 JP WO2019200122 A5 JPWO2019200122 A5 JP WO2019200122A5 JP 2020555222 A JP2020555222 A JP 2020555222A JP 2020555222 A JP2020555222 A JP 2020555222A JP WO2019200122 A5 JPWO2019200122 A5 JP WO2019200122A5
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human
polynucleotide
engineered meganuclease
cleavage site
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JP7304888B2 (en
JP2021520800A (en
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Claims (17)

配列番号７のアミノ酸配列を含み、ヒトＴ細胞受容体（ＴＣＲ）アルファ定常領域遺伝子内の配列番号５からなる認識配列と結合し且つ切断する、操作されたメガヌクレアーゼ。 An engineered meganuclease comprising the amino acid sequence of SEQ ID NO:7, which binds and cleaves the recognition sequence consisting of SEQ ID NO:5 within the human T-cell receptor (TCR) alpha constant region gene. 請求項１に記載の操作された前記メガヌクレアーゼをコードしている核酸配列を含む、ポリヌクレオチド。 A polynucleotide comprising a nucleic acid sequence encoding the engineered meganuclease of claim 1. 前記ポリヌクレオチドがｍＲＮＡである、請求項２に記載のポリヌクレオチド。 3. The polynucleotide of claim 2, wherein said polynucleotide is mRNA. 請求項２に記載の前記ポリヌクレオチドを含む、組換えＤＮＡ構築物。 A recombinant DNA construct comprising the polynucleotide of claim 2. 請求項２に記載の前記ポリヌクレオチドを含む、ウイルスベクター。 A viral vector comprising the polynucleotide of claim 2 . 前記ウイルスベクターが組換えアデノ随伴ウイルス（ＡＡＶ）ベクターである、請求項５に記載のウイルスベクター。 6. The viral vector of claim 5, wherein said viral vector is a recombinant adeno-associated virus (AAV) vector. 生体外（エクスビボ）で、遺伝子改変された真核細胞の染色体のターゲット配列を破壊することによって遺伝子改変された前記真核細胞（ヒト配偶子、ヒト接合子、ヒト胚盤胞、及びヒト胚性幹細胞を除く）を産生するための方法であって、
前記方法は、（ａ）請求項１に記載の前記操作されたメガヌクレアーゼ；又は（ｂ）請求項２又は３に記載の前記ポリヌクレオチドを、真核細胞に導入することを含み、
前記操作されたメガヌクレアーゼは、前記真核細胞で発現されており、
前記操作されたメガヌクレアーゼは、配列番号５からなる認識配列にて前記染色体中に切断部位を作製し、前記ターゲット配列は、前記切断部位にて非相同末端結合することで破壊されている、方法。 Genetically modified eukaryotic cells (human gametes, human zygotes, human blastocysts, and human embryonic excluding stem cells), comprising:
The method comprises introducing (a) the engineered meganuclease of claim 1 ; or (b) the polynucleotide of claim 2 or 3 into a eukaryotic cell,
said engineered meganuclease being expressed in said eukaryotic cell;
The engineered meganuclease creates a cleavage site in the chromosome at a recognition sequence consisting of SEQ ID NO:5, and the target sequence is disrupted by non-homologous end joining at the cleavage site. . 前記ポリヌクレオチドがｍＲＮＡである、請求項７に記載の方法。8. The method of claim 7, wherein said polynucleotide is mRNA. 生体外（エクスビボ）で、真核細胞の染色体中へと挿入された外因性の対象配列を含む、遺伝子改変された前記真核細胞（ヒト配偶子、ヒト接合子、ヒト胚盤胞、及びヒト胚性幹細胞を除く）を産生するための方法であって、Genetically modified eukaryotic cells (human gametes, human zygotes, human blastocysts, and human excluding embryonic stem cells), comprising:
前記方法は、（ａ）請求項１に記載の前記操作されたメガヌクレアーゼ又は請求項２若しくは３に記載の核酸；及び（ｂ）前記対象配列を含む核酸を、真核細胞に導入することを含み、The method comprises introducing into a eukaryotic cell (a) the engineered meganuclease of claim 1 or the nucleic acid of claim 2 or 3; and (b) a nucleic acid comprising the sequence of interest. including
前記操作されたメガヌクレアーゼは、前記真核細胞にて発現されており、said engineered meganuclease being expressed in said eukaryotic cell;
前記操作されたメガヌクレアーゼは、配列番号５からなる認識配列にて前記染色体中に切断部位を作製し、前記対象配列は、前記切断部位にて前記染色体中へと挿入されている、方法。A method, wherein said engineered meganuclease creates a cleavage site in said chromosome at a recognition sequence consisting of SEQ ID NO:5, and wherein said sequence of interest is inserted into said chromosome at said cleavage site. 前記対象配列が、キメラ抗原受容体（ＣＡＲ）または外因性ＴＣＲのためのコード配列を含む、請求項９に記載の方法。10. The method of claim 9, wherein said sequence of interest comprises a coding sequence for a chimeric antigen receptor (CAR) or an exogenous TCR. 前記対象配列を含む前記核酸が、前記切断部位に隣接する配列に相同な配列を更に含み、前記対象配列が、相同組換えによって前記切断部位に挿入されている、請求項９又は１０に記載の方法。11. The nucleic acid comprising the target sequence further comprising a sequence homologous to a sequence flanking the cleavage site, wherein the target sequence is inserted into the cleavage site by homologous recombination. Method. 前記ポリヌクレオチドがｍＲＮＡである、請求項９～１１のいずれか１項に記載の方法。The method of any one of claims 9-11, wherein said polynucleotide is mRNA. 前記対象配列を含む前記核酸が、ウイルスベクターによって前記真核細胞中へと導入される、請求項９～１２のいずれか１項に記載の方法。A method according to any one of claims 9 to 12, wherein said nucleic acid comprising said sequence of interest is introduced into said eukaryotic cell by means of a viral vector. 前記ウイルスベクターが、組換えＡＡＶベクターである、請求項１３に記載の方法。14. The method of claim 13, wherein said viral vector is a recombinant AAV vector. 前記組換えＡＡＶベクターが、ＡＡＶ２またはＡＡＶ６のセロタイプを有する、請求項１４に記載の方法。15. The method of claim 14, wherein the recombinant AAV vector has the AAV2 or AAV6 serotype. 前記真核細胞が、ヒトＴ細胞である、請求項７～１５のいずれか１項に記載の方法。The method of any one of claims 7-15, wherein said eukaryotic cells are human T cells. 前記遺伝子改変された真核細胞は、細胞表面に内因性ＴＣＲを発現しない、請求項７～１６のいずれか１項に記載の方法。The method of any one of claims 7-16, wherein said genetically modified eukaryotic cell does not express an endogenous TCR on the cell surface.
JP2020555222A 2018-04-12 2019-04-11 Optimized engineered nucleases with specificity for the human T-cell receptor alpha constant region gene Active JP7304888B2 (en)

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