JP2019080588A - 導入遺伝子発現を制御するための方法および組成物 - Google Patents
導入遺伝子発現を制御するための方法および組成物 Download PDFInfo
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Abstract
Description
本出願は、2011年9月21日出願の米国仮出願第61/537,349号、2011年11月16日出願の米国仮出願第61/560,506号、および2012年7月11日出願の米国仮出願第61/670,490号の利益を主張するものであり、これらの開示は、参照によりその全体が本明細書に組み込まれる。
したがって、任意の関連毒性を回避しながら所望の導入遺伝子を治療関連レベルで発現させるために使用され得、かつ導入遺伝子の発現を所望の組織型に制限して、例えば、血友病、糖尿病、リソソーム蓄積症、およびA1AT欠損症等の遺伝的疾患を治療し得るさらなる方法および組成物の必要性が未だ存在する。さらに、癌等の他の疾患を治療するために所望の導入遺伝子を治療関連レベルで発現させるさらなる方法および組成物の必要性が未だ存在する。
前記1つ以上の融合タンパク質が発現し、前記アルブミン遺伝子が切断される条件下で、前記細胞に、実施形態6に記載の少なくとも1つのポリヌクレオチドを含む1つ以上の発現ベクターを導入することを含む、方法。
前記導入遺伝子が内因性アルブミン遺伝子に組み込まれるように、前記導入遺伝子を含む外因性ポリヌクレオチドの存在下で、実施形態15〜17のいずれかに記載の方法に従って内因性アルブミン遺伝子を切断することを含む、方法。
導入遺伝子が単離細胞において発現されるように、実施形態14〜19に記載の方法に従って前記ポリペプチドをコードする前記導入遺伝子を前記単離細胞に導入することと、前記単離細胞を前記対象に導入し、それによって前記疾患を治療することと、を含む、方法。
例えば、本発明は、以下の項目を提供する。
(項目1)
内因性アルブミン遺伝子に結合するジンクフィンガータンパク質および切断ドメインを含む非天然融合タンパク質であって、前記内因性アルブミン遺伝子を修飾する、融合タンパク質。
(項目2)
前記ジンクフィンガータンパク質が、認識ヘリックス領域を含む4、5、または6個のジンクフィンガードメインを含み、前記ジンクフィンガータンパク質が、表1、表3、表5、または表8の単一列に示される前記認識ヘリックス領域を含む、項目2に記載の融合タンパク質。
(項目3)
項目1に記載の1つ以上の融合タンパク質をコードするポリヌクレオチド。
(項目4)
項目1または項目2に記載の1つ以上の融合タンパク質または項目3に記載の1つ以上のポリヌクレオチドを含む単離細胞。
(項目5)
前記細胞が幹細胞である、項目4に記載の細胞。
(項目6)
前記幹細胞が、胚幹細胞(ESC)、人工多能性幹細胞(iPSC)、肝幹細胞、および肝臓幹細胞からなる群から選択される、項目5に記載の細胞。
(項目7)
項目1または項目2に記載の融合タンパク質または項目3に記載のポリヌクレオチドを含むキット。
(項目8)
細胞中の内因性アルブミン遺伝子を切断する方法であって、
前記1つ以上の融合タンパク質が発現され、かつ前記アルブミン遺伝子が切断される条件下で、前記細胞に、項目1または項目2に記載の少なくとも1つの融合タンパク質または項目3に記載の少なくとも1つのポリヌクレオチドを含む1つ以上の発現ベクターを導入することを含む、方法。
(項目9)
前記ポリヌクレオチドがAAVベクターを含む、項目8に記載の方法。
(項目10)
前記細胞が肝臓細胞である、項目8または項目9に記載の方法。
概要
periodic updates;the series METHODS IN ENZYMOLOGY,Academic Press,San Diego;Wolffe,CHROMATIN STRUCTURE AND FUNCTION,Third edition,Academic Press,San Diego,1998;METHODS IN ENZYMOLOGY,Vol.304,“Chromatin”(P.M.Wassarman and A.P.Wolffe,eds.),Academic Press,San Diego,1999;およびMETHODS IN
MOLECULAR BIOLOGY,Vol.119,“Chromatin Protocols”(P.B.Becker,ed.)Humana Press,Totowa,1999を参照されたい。
定義
ゲノムに挿入されるヌクレオチド配列を指す。ドナー配列は、任意の長さであってもよく、例えば、2〜10,000ヌクレオチド長(またはそれらの間もしくはそれらを超える任意の整数値)、好ましくは、約100〜1,000ヌクレオチド長(またはそれらの間の任意の整数)、より好ましくは、約200〜500ヌクレオチド長であってもよい。
ヌクレアーゼ
A.DNA結合ドメイン
ujon et al.(1989)Gene 82:115-118、Perler
et al.(1994)Nucleic Acids Res.22,1125-11
27、Jasin(1996)Trends Genet.12:224-228、Gi
mble et al.(1996)J.Mol.Biol.263:163-180、
Argast et al.(1998)J.Mol.Biol.280:345-35
3、およびthe New England Biolabsカタログも参照されたい。
5-118、Perler et al.(1994)Nucleic Acids R
es.22,1125-1127、Jasin(1996)Trends Genet.
12:224-228、Gimble et al.(1996)J.Mol.Biol
.263:163-180、Argast et al.(1998)J.Mol.Bi
ol.280:345-353、およびthe New England Biolab
sカタログも参照されたい。加えて、ホーミングエンドヌクレアーゼおよびメガヌクレアーゼのDNA結合特異性は、非天然標的部位に結合するように操作され得る。例えば、Chevalier et al.(2002)Molec.Cell 10:895−905、Epinat et al.(2003)Nucleic Acids Res.31:2952−2962、Ashworth et al.(2006)Nature
441:656−659、Paques et al.(2007)Current Gene Therapy 7:49−66、米国特許公開第20070117128号を参照されたい。ホーミングエンドヌクレアーゼおよびメガヌクレアーゼのDNA結合ドメインは、全体としてのヌクレアーゼという文脈において改変され得るか(すなわち、ヌクレアーゼが同族の切断ドメインを含むように)、または異種の切断ドメインに融合され得る。
B.切断ドメイン
et al.(1994b)J.Biol.Chem.269:31,978−31,982を参照されたい。したがって、一実施形態において、融合タンパク質は、少なくとも1つのIIS型制限酵素由来の切断ドメイン(または切断ハーフドメイン)および1つ以上のジンクフィンガー結合ドメイン(操作され得るか否かに関わらず)を含む。
al.(1998)Proc.Natl.Acad.Sci.USA 95:10,570−10,575。したがって、本開示の目的のために、開示される融合タンパク質において使用されるFok I酵素の一部は、切断ハーフドメインと見なされる。したがって、ジンクフィンガー−Fok I融合物を用いた細胞配列の標的二本鎖切断および/または標的置換のために、それぞれFok I切断ハーフドメインを含む2つの融合タンパク質を用いて、触媒的に活性な切断ドメインを再構築することができる。あるいは、DNA結合ドメインおよび2つのFok I切断ハーフドメインを含む単一のポリペプチド分子を用いることもできる。
Iの446、447、479、483、484、486、487、490、491、496、498、499、500、531、534、537、および538位のアミノ酸残基は、すべてFok I切断ハーフドメインの二量体化に影響を与える標的である。
標的部位
ドナー
送達
よびYu et al.,Gene Therapy 1:13−26(1994)を参照されたい。
Res.52:4817−4820(1992)、米国特許第4,186,183号、
同第4,217,344号、同第4,235,871号、同第4,261,975号、同第
4,485,054号、同第4,501,728号、同第4,774,085号、同第4,837,028号、および同第4,946,787号を参照のこと)。
et al.,Hum.Gene Ther.7:1083−9(1998))。臨床試験における遺伝子導入のためのアデノウイルスベクターの使用のさらなる例として、Rosenecker et al.,Infection 24:1 5−10(1996)、Sterman et al.,Hum.Gene Ther.9:7 1083−1089(1998)、Welsh et al.,Hum.Gene Ther.2:205−18(1995)、Alvarez et al.,Hum.Gene Ther.5:597−613(1997)、Topf et al.,Gene Ther.5:507−513(1998)、Sterman et al.,Hum.Gene
Ther.7:1083−1089(1998)が挙げられる。
et al.(1998)J.Virol.72:9873−9880、Follenzi et al.(2000)Nature Genetics 25:217−222、米国特許公開第2009/054985号を参照されたい。
適用
ジンクフィンガータンパク質を、マウスアルブミン遺伝子のイントロン1、12、および13内の切断部位を標的化するように設計した。対応する発現構築物を、本質的にUrnov et al.(2005)Nature 435(7042):646−651、Perez et al(2008)Nature Biotechnology 26(7):808−816に記載され、かつ米国特許第6,534,261号に記載されるように組み立て、プラスミドベクター、AAVベクター、またはアデノウイルスベクターに組み込んだ。表1は、例示のマウスアルブミン特異的ZFPのDNA結合ドメイン内の認識ヘリックスを示し、表2は、これらのZFPの標的部位を示す。ZFP認識ヘリックスに接触する標的部位におけるヌクレオチドは大文字で示され、接触していないヌクレオチドは、小文字で示される。
実施例3:イヌアルブミン特異的ZFN
実施例4:非ヒト霊長類アルブミン特異的ZFN
実施例5:マウスにおけるZFNによるインビボ切断
Cel1 F1:5’CCTGCTCGACCATGCTATACT 3’(配列番号69)
Cel1R1:5’CAGGCCTTTGAAATGTTGTTC 3’(配列番号70)
mAlb set4F4:5’AAGTGCAAAGCCTTTCAGGA 3’(配列番号71)
mAlb set4R4:5’GTGTCCTTGTCAGCAGCCTT 3’(配列番号72)
実施例6:ドナー核酸およびアルブミンZFNのインビボにおける共送達
実施例7:ヒトアルブミン特異的ZFNの設計
実施例8:アルブミン特異的TALENの設計
Claims (10)
- アルブミン遺伝子を切断するジンクフィンガーヌクレアーゼであって、前記ジンクフィンガーヌクレアーゼは、1つ以上の操作されたジンクフィンガー結合ドメインおよびヌクレアーゼ切断ドメインを含み、前記1つ以上の操作されたジンクフィンガー結合ドメインは、アルブミン遺伝子内の標的配列に結合し、前記標的部位は、配列番号127または配列番号128中にある、ジンクフィンガーヌクレアーゼ。
- 前記ジンクフィンガーヌクレアーゼが、第1および第2のジンクフィンガーヌクレアーゼを含み、前記第1のジンクフィンガーヌクレアーゼは、配列番号127中の標的部位に結合する1つ以上の操作されたジンクフィンガー結合ドメインを含み、前記第2のジンクフィンガーヌクレアーゼは、配列番号128中の標的部位に結合する1つ以上の操作されたジンクフィンガー結合ドメインを含む、請求項1に記載のジンクフィンガーヌクレアーゼ。
- 請求項1または2に記載の1つ以上のジンクフィンガーヌクレアーゼをコードする1つ以上のポリヌクレオチド。
- 請求項1または2に記載の1つ以上のジンクフィンガーヌクレアーゼおよび/または請求項3に記載の1つ以上のポリヌクレオチドを含む、単離細胞。
- 前記細胞が幹細胞であり、必要に応じて、前記幹細胞は、胚幹細胞(ESC)、人工多能性幹細胞(iPSC)、肝幹細胞、および肝臓幹細胞からなる群から選択される、請求項4に記載の細胞。
- 請求項1または2に記載のジンクフィンガーヌクレアーゼ、請求項3に記載の1つ以上のポリヌクレオチド、および/または請求項4または5に記載の細胞を含むキット。
- 哺乳動物対象の細胞における内因性アルブミン遺伝子を改変するための組成物であって、前記組成物は、請求項1または2に記載のジンクフィンガーヌクレアーゼ、または請求項3に記載の1つ以上のポリヌクレオチドを含む、組成物。
- 治療用タンパク質をコードする導入遺伝子を含むドナーポリヌクレオチドをさらに含む、請求項7に記載の組成物。
- 前記ジンクフィンガーヌクレアーゼをコードするポリヌクレオチドおよび/またはドナーポリヌクレオチドがAAVベクターである、請求項7または請求項8に記載の組成物。
- 前記対象が、凝固障害、COPDもしくは肝臓損傷等のA1AT欠損障害、リソソーム貯蔵疾患、代謝性疾患、糖尿病、表皮水疱症、肝硬変、リポタンパク質リパーゼ欠損症、癌、および自己免疫疾患を含む群から選択される疾患または障害に罹患している、請求項7〜9のいずれかに記載の組成物。
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