JP7007337B2 - 標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー - Google Patents
標的遺伝子治療のための合成コンビナトリアルaavカプシドライブラリー Download PDFInfo
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Description
[0001] 本出願は、2013年9月26日に出願された米国特許仮出願第61/883,063号(係属中、代理人整理番号36689.341)の優先権を主張する。当該仮出願の内容は、その内容の明確な参照によりその全体が本明細書に具体的に組み込まれる。
[0002] 本発明は、米国国立衛生研究所によって与えられた助成番号HL-097088及びGM-082946での政府支援によってなされた。米国政府は、本発明に一定の権利を有する。
[0003] 該当なし。
[0080] 本発明は、哺乳動物の疾患、傷害、障害、外傷又は機能不全の1つ又は複数の症状を診断、予防、処置又は改善するためのキットの中に含まれる、開示されたrAAVベクターの1つ又は複数を含む組成物を提供する。そのようなキットは、ヒト疾患の診断、発病予防及び/又は治療に有用でありえ、特に、ヒトの癌、糖尿病、自己免疫疾患、腎臓疾患、心血管疾患、膵臓疾患、腸疾患、肝臓疾患、神経疾患、神経筋障害、運動神経欠損症、神経骨格障害、神経学的能力障害、神経感覚機能障害、卒中、虚血、α1アンチトリプシン(AAT)欠乏症、バッテン病、アルツハイマー病、鎌状赤血球症、βサラセミア(thalassamia)、ハンチントン病、パーキンソン病、骨格疾患、外傷、肺疾患の1つ又は複数の症状の処置、予防及び/又は改善に有用でありうる。
[0084] 本発明の遺伝子構築物は、様々な組成で調製することができ、ヒト又は動物対象への投与のために適切な医薬ビヒクルに配合することもできる。
[0086] 別段の定義がない限り、本明細書において用いるすべての専門用語は、本発明が属する技術分野の当業者によって一般に理解されているのと同じ意味を有する。分子生物学用語について一般に理解されている定義は、Riegerら(1991)、Lewin(1994)で見出すことができる。ウイルス学用語の一般に理解されている定義は、Granoff及びWebster(1999)ならびにTidona及びDarai(2002)で見出すことができる。微生物学の一般に理解されている定義は、Singleton及びSainsbury(2002)で見出すことができる。
ted modifications in adeno-associated virus serotype 8 capsid improves its hepatic gene transfer efficiency in vivo," Hum. Gene Ther. Methods, 24: 104-116 (2013). Shannon, CE "The mathematical theory of communication," Bell Syst. Tech. J., 27:379-423, 623- 656 (1948). Sharland, A et al., "Liver-directed gene expression using recombinant AAV 2/8 vectors-a tolerogenic strategy for gene delivery?" Discov. Med., 9:519-27 (2010). Sharma et al., "2A peptides provide distinct solutions to driving stop-carry on translational recoding," Nucleic Acids Res., 40:3143-3151 (2012). Shen, X et al., "Characterization of the relationship of AAV capsid domain swapping to liver transduction efficiency," Mol. Ther., 15: 1955-1962 (2007). Singleton and Sainsbury, Dictionary of Microbiology and Molecular Biology, 3rd edition, John Wiley & Sons: New York, NY (2002). Sonntag, F et al., "A viral assembly factor promotes AAV2 capsid formation in the nucleolus," Proc. Nat'l. Acad. Sci. USA, 107: 10220-10225 (2010). Stachler, MD and Bartlett, JS, "Mosaic vectors comprised of modified AAV1 capsid proteins for efficient vector purification and targeting to vascular endothelial cells," Gene Ther., 13:926-931 (2006). Summerford, C, and Samulski, RJ, "Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions," J. Virol, 72: 1438-1445 (1998). Tidona and Darai, The Springer Index of Viruses, 1st edition, Springer- Verlag: New York, NY (2002). Walters, RW et al., "Structure of adeno-associated virus serotype 5," J. Virol., 78:3361-3371 (2004). Wu, P et al., "Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism," J. Virol. 74:8635-8647 (2000). Wu, P et al., Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism," J. Virol., 74:8635-8647 (2000). Wu, Z et al., "Adeno-associated virus serotypes: vector toolkit for human gene therapy," Mol. Ther. J. Am. Soc. Gene Ther., 14:316-327 (2006). Xie, Q et al., "The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy," Proc. Nat'l. Acad. Sci. USA, 99: 10405-10410 (2002). Yang, L and Xiao, X, "Creation of a cardiotropic adeno-associated virus: the story of viral directed evolution," Virol. J., 10:50 (2013). Yang, L et al., "A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection," Proc. Nat'l. Acad. Sci. USA, 106:3946-3951 (2009). Yang, L et al., "Directed evolution of adeno-associated virus (AAV) as vector for muscle gene therapy," Methods Mol. Biol. (Clifton, NJ), 709: 127-139 (2011). Zhong, L et al., "Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses," Proc. Nat'l. Acad. Sci. USA, 105:7827-7832 (2008). Zolotukhin, S et al., "Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield," Gene Ther., 6:973-985 (1999).Gigout, L et al., "Altering AAV tropism with mosaic viral capsids," Mol. Ther., 11:856-865 (2005).
Claims (11)
- 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸262-268に対応する位置に配列番号176の配列を備える、
使用。 - 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸449-463に対応する位置に配列番号177の配列を備える、
使用。 - 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸491-501に対応する位置に配列番号178の配列を備える、
使用。 - 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸498-506に対応する位置に配列番号179の配列を備える、
使用。 - 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸491-503に対応する位置に配列番号180の配列を備える、
使用。 - 哺乳動物の1つ又は複数の障害の処置のための組成物の製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が、野生型AAV2カプシドタンパク質のVP1配列中のアミノ酸残基K507におけるトレオニン置換を備える、
使用。 - 前記ビリオンが核酸セグメントをさらに備え、前記核酸セグメントが、前記ビリオンを備える適切な宿主細胞において、前記核酸セグメントを発現することが可能なプロモーターに作動可能に連結されている診断用又は治療用分子をコードする、
請求項1から6のいずれか1項に記載の使用。 - 前記核酸セグメントが、当該核酸セグメントに作動可能に連結されている、エンハンサー、転写後調節配列、ポリアデニル化シグナル、又はそれらの任意の組み合わせをさらに備える、請求項7に記載の使用。
- 前記治療用分子が、ポリペプチド、ペプチド、リボザイム、ペプチド核酸、siRNA、RNAi、アンチセンスオリゴヌクレオチド、アンチセンスポリヌクレオチド、抗体、抗原結合断片、又はそれらの任意の組み合わせである、請求項7又は8に記載の使用。
- 前記治療用分子が、アゴニスト、アンタゴニスト、抗アポトーシス因子、阻害剤、受容体、サイトカイン、細胞毒、赤血球生成剤(erythropoietic agent)、糖タンパク質、成長因子、成長因子受容体、ホルモン、ホルモン受容体、インターフェロン、インターロイキン、インターロイキン受容体、神経成長因子、神経活性ペプチド、神経活性ペプチド受容体、プロテアーゼ、プロテアーゼ阻害剤、タンパク質デカルボキシラーゼ、プロテインキナーゼ、プロテインキナーゼ阻害剤、酵素、受容体結合タンパク質、輸送タンパク質又はその阻害剤、セロトニン受容体又はその取り込み阻害剤、セルピン、セルピン受容体、腫瘍抑制因子、あるいはそれらの任意の組み合わせである、請求項7から9のいずれか1項に記載の使用。
- 哺乳動物の1つ又は複数の障害の診断又は処置のためのキットの製造における修飾rAAV2ビリオンの使用であって、
前記ビリオンが修飾AAV2カプシドタンパク質を備え、前記修飾AAV2カプシドタンパク質が1つ又は複数のアミノ酸置換を備え、AAV2 VP1カプシドタンパク質のアミノ酸262-268に対応する位置に配列番号176、AAV2 VP1カプシドタンパク質のアミノ酸449-463に対応する位置に配列番号177、AAV2 VP1カプシドタンパク質のアミノ酸491-501に対応する位置に配列番号178、AAV2 VP1カプシドタンパク質のアミノ酸498-506に対応する位置に配列番号179、及びAAV2 VP1カプシドタンパク質のアミノ酸491-503に対応する位置に配列番号180のいずれかの配列を備えるか、野生型AAV2カプシドタンパク質のVP1配列中のアミノ酸残基K507におけるトレオニン置換を備える、
使用。
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