Title:
ADENO-ASSOCIATED VIRUS WITH SITE-DIRECTED MUTAGENESIS AND SITE-DIRECTED MODIFICATION, AND PREPARATION METHOD AND APPLICATION THEREFOR
Document Type and Number:
WIPO Patent Application WO/2015/062516
Kind Code:
A1
Abstract:
Provided is an adeno-associated virus with site-directed mutagenesis and site-directed modification, and a preparation method therefor. Genetic code expansion techniques are used to incorporate non-natural amino acids into an adeno-associated virus capsid protein VP1 or fragment thereof, thereby obtaining an adeno-associated virus with site-directed mutagenesis using non-natural amino acids. The present adeno-associated virus with site-directed mutagenesis is equivalent to a wild-type virus in terms of production, transduction and mobility, can couple with other functional molecules, such as a target molecule, and can carry a functional gene in a normal manner, acting as an adeno-associated virus vector.
Inventors:
ZHOU DEMIN (CN)
ZHANG CHUANLING (CN)
XIAO SULONG (CN)
YAO TIANZHUO (CN)
ZHENG YONGXIANG (CN)
YU FEI (CN)
SI LONGLONG (CN)
ZHANG LIHE (CN)
ZHANG CHUANLING (CN)
XIAO SULONG (CN)
YAO TIANZHUO (CN)
ZHENG YONGXIANG (CN)
YU FEI (CN)
SI LONGLONG (CN)
ZHANG LIHE (CN)
Application Number:
PCT/CN2014/089880
Publication Date:
May 07, 2015
Filing Date:
October 30, 2014
Export Citation:
Assignee:
UNIV BEIJING (CN)
International Classes:
C07K14/015; A61K48/00; C12N1/21; C12N15/35; C12N15/70; C12N15/85; C12N15/864
Domestic Patent References:
| WO2004027019A2 | 2004-04-01 |
Foreign References:
| CN102838663A | 2012-12-26 | |||
| CN102618605A | 2012-08-01 | |||
| CN102838663A | 2012-12-26 |
Other References:
See also references of EP 3070095A4
L. WANG ET AL., SCIENCE, vol. 292, 2001, pages 498 - 500
J. W. CHIN ET AL., JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 124, 2002, pages 9026 - 9027
J. W. CHIN; P. G. SCHULTZ, CHEMBIOCHEM, vol. 11, 2002, pages 1135 - 1137
ALTSCHUL ET AL., NUCL. ACID. RES, vol. 25, 1977, pages 3389 - 3402
ALTSCHUL ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403 - 410
DUY P. NGUYEN; HRVOJE LUSIC; HEINZ NEUMANN; PRASHANT B. KAPADNIS; ALEXANDER DEITERS; JASON W. CHIN: "Genetic Encoding and Labeling of Aliphatic Azides and Alkynes in Recombinant Proteins via a Pyrrolysyl-tRNA Synthetase/tRNA Pair and Click Chemistry", J. AM. CHEM. SOC, vol. 131, 2009, pages 8720 - 8721
GUO P; EL-GOHARY Y; PRASADAN K; SHIOTA C; XIAO X; WIERSCH J; PAREDES J; TULACHAN S; GITTES GK: "Rapid and simplified purification of recombinant adeno-associated virus", J VIROL METHODS, vol. 183, no. 2, pages 139 - 146
ATCHISON RW; CASTO BC; HAMMON WM: "Adenovirus-Associated Defective Virus Particles", SCIENCE, vol. 149, no. 3685, 1965, pages 754 - 756
GRAY SJ; SAMULSKI RJ: "Optimizing gene delivery vectors for the treatment of heart disease", EXPERT OPIN BIOL THER, vol. 8, no. 7, 2008, pages 911 - 922
GRIMM D; KAY MA: "From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy", CURR GENE THER, vol. 3, no. 4, 2003, pages 281 - 304
MUELLER C; FLOTTE TR: "Clinical gene therapy using recombinant adeno-associated virus vectors", GENE THER, vol. 15, no. 11, 2008, pages 858 - 863
CHOI KJ; KIM JH; LEE YS; KIM J; SUH BS; KIM H; CHO S; SOHN JH; KIM GE; YUN CO: "Concurrent delivery of GM-CSF and B7-1 using an oncolytic adenovirus elicits potent antitumor effect", GENE THER, vol. 13, no. 13, 2006, pages 1010 - 1020
WU Z; ASOKAN A; SAMULSKI RJ: "Adeno-associated virus serotypes: vector toolkit for human gene therapy", MOL THER, vol. 14, no. 3, 2006, pages 316 - 327
FISHER KJ; JOOSS K; ALSTON J; YANG Y; HAECKER SE; HIGH K; PATHAK R; RAPER SE; WILSON JM: "Recombinant adeno-associated virus for muscle directed gene therapy", NAT MED, vol. 3, no. 3, 1997, pages 306 - 312
MOSS RB; RODMAN D; SPENCER LT; AITKEN ML; ZEITLIN PL; WALTZ D; MILLA C; BRODY AS; CLANCY JP; RAMSEY B ET AL.: "Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial", CHEST, vol. 125, no. 2, 2004, pages 509 - 521
MINGOZZI F; HIGH KA: "Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.", NAT REV GENET, vol. 12, no. 5, pages 341 - 355
BAINBRIDGE JW; SMITH AJ; BARKER SS; ROBBIE S; HENDERSON R; BALAGGAN K; VISWANATHAN A; HOLDER GE; STOCKMAN A; TYLER N ET AL.: "Effect of gene therapy on visual function in Leber's congenital amaurosis", N ENGL J MED, vol. 358, no. 21, 2008, pages 2231 - 2239
HAUSWIRTH WW; ALEMAN TS; KAUSHAL S; CIDECIYAN AV; SCHWARTZ SB; WANG L; CONLON TJ; BOYE SL; FLOTTE TR; BYRNE BJ ET AL.: "Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.", HUM GENE THER, vol. 19, no. 10, 2008, pages 979 - 990
MANNO CS; PIERCE GF; ARRUDA VR; GLADER B; RAGNI M; RASKO JJ; OZELO MC; HOOTS K; BLATT P; KONKLE B ET AL.: "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.", NAT MED, vol. 12, no. 3, 2006, pages 342 - 347
MANNO CS; CHEW AJ; HUTCHISON S; LARSON PJ; HERZOG RW; ARRUDA VR; TAI SJ; RAGNI MV; THOMPSON A; OZELO M ET AL.: "AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.", BLOOD, vol. 101, no. 8, 2003, pages 2963 - 2972
WHITE SJ; NICKLIN SA; BUNING H; BROSNAN MJ; LEIKE K; PAPADAKIS ED; HALLEK M; BAKER AH: "Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors", CIRCULATION, vol. 109, no. 4, 2004, pages 513 - 519
YU CY; YUAN Z; CAO Z; WANG B; QIAO C; LI J; XIAO X: "A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery", GENE THER, vol. 16, no. 8, 2009, pages 953 - 962
WORK LM; BUNING H; HUNT E; NICKLIN SA; DENBY L; BRITTON N; LEIKE K; ODENTHAL M; DREBBER U; HALLEK M ET AL.: "Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses", MOL THER, vol. 13, no. 4, 2006, pages 683 - 693
DOUAR AM; POULARD K; DANOS O: "Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid", VIROLOGY, vol. 309, no. 2, 2003, pages 203 - 208
NING X; GUO J; WOLFERT MA; BOONS GJ: "Visualizing metabolically labeled glycoconjugates of living cells by copper-free and fast huisgen cycloadditions", ANGEW CHEM INT ED ENGL, vol. 47, no. 12, 2008, pages 2253 - 2255
JOO KI; FANG Y; LIU Y; XIAO L; GU Z; TAI A; LEE CL; TANG Y; WANG P: "Enhanced real-time monitoring of adeno-associated virus trafficking by virus-quantum dot conjugates", ACS NANO, vol. 5, no. 5, pages 3523 - 3535
ROHR, U. P.; WULF, M. A.; STAHN, S.; STEIDL, U.; HAAS, R.; KRONENWETT, R., J VIROL METHODS, vol. 106, 2002, pages 81 - 8
TRAVIS S.: "Young and Peter G. Schultz: Beyond the Canonical 20 Amino Acids: Expanding the Genetic Lexicon", J BIOL. CHEM., vol. 285, 2010, pages 11039 - 11044
JIANMING XIE; PETER G SCHULTZ: "Adding amino acids to the genetic repertoire", CURRENT OPINION IN CHEMICAL BIOLOGY, vol. 9, 2005, pages 548 - 554
L. WANG ET AL., SCIENCE, vol. 292, 2001, pages 498 - 500
J. W. CHIN ET AL., JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 124, 2002, pages 9026 - 9027
J. W. CHIN; P. G. SCHULTZ, CHEMBIOCHEM, vol. 11, 2002, pages 1135 - 1137
ALTSCHUL ET AL., NUCL. ACID. RES, vol. 25, 1977, pages 3389 - 3402
ALTSCHUL ET AL., J. MOL. BIOL., vol. 215, 1990, pages 403 - 410
DUY P. NGUYEN; HRVOJE LUSIC; HEINZ NEUMANN; PRASHANT B. KAPADNIS; ALEXANDER DEITERS; JASON W. CHIN: "Genetic Encoding and Labeling of Aliphatic Azides and Alkynes in Recombinant Proteins via a Pyrrolysyl-tRNA Synthetase/tRNA Pair and Click Chemistry", J. AM. CHEM. SOC, vol. 131, 2009, pages 8720 - 8721
GUO P; EL-GOHARY Y; PRASADAN K; SHIOTA C; XIAO X; WIERSCH J; PAREDES J; TULACHAN S; GITTES GK: "Rapid and simplified purification of recombinant adeno-associated virus", J VIROL METHODS, vol. 183, no. 2, pages 139 - 146
ATCHISON RW; CASTO BC; HAMMON WM: "Adenovirus-Associated Defective Virus Particles", SCIENCE, vol. 149, no. 3685, 1965, pages 754 - 756
GRAY SJ; SAMULSKI RJ: "Optimizing gene delivery vectors for the treatment of heart disease", EXPERT OPIN BIOL THER, vol. 8, no. 7, 2008, pages 911 - 922
GRIMM D; KAY MA: "From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy", CURR GENE THER, vol. 3, no. 4, 2003, pages 281 - 304
MUELLER C; FLOTTE TR: "Clinical gene therapy using recombinant adeno-associated virus vectors", GENE THER, vol. 15, no. 11, 2008, pages 858 - 863
CHOI KJ; KIM JH; LEE YS; KIM J; SUH BS; KIM H; CHO S; SOHN JH; KIM GE; YUN CO: "Concurrent delivery of GM-CSF and B7-1 using an oncolytic adenovirus elicits potent antitumor effect", GENE THER, vol. 13, no. 13, 2006, pages 1010 - 1020
WU Z; ASOKAN A; SAMULSKI RJ: "Adeno-associated virus serotypes: vector toolkit for human gene therapy", MOL THER, vol. 14, no. 3, 2006, pages 316 - 327
FISHER KJ; JOOSS K; ALSTON J; YANG Y; HAECKER SE; HIGH K; PATHAK R; RAPER SE; WILSON JM: "Recombinant adeno-associated virus for muscle directed gene therapy", NAT MED, vol. 3, no. 3, 1997, pages 306 - 312
MOSS RB; RODMAN D; SPENCER LT; AITKEN ML; ZEITLIN PL; WALTZ D; MILLA C; BRODY AS; CLANCY JP; RAMSEY B ET AL.: "Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial", CHEST, vol. 125, no. 2, 2004, pages 509 - 521
MINGOZZI F; HIGH KA: "Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.", NAT REV GENET, vol. 12, no. 5, pages 341 - 355
BAINBRIDGE JW; SMITH AJ; BARKER SS; ROBBIE S; HENDERSON R; BALAGGAN K; VISWANATHAN A; HOLDER GE; STOCKMAN A; TYLER N ET AL.: "Effect of gene therapy on visual function in Leber's congenital amaurosis", N ENGL J MED, vol. 358, no. 21, 2008, pages 2231 - 2239
HAUSWIRTH WW; ALEMAN TS; KAUSHAL S; CIDECIYAN AV; SCHWARTZ SB; WANG L; CONLON TJ; BOYE SL; FLOTTE TR; BYRNE BJ ET AL.: "Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.", HUM GENE THER, vol. 19, no. 10, 2008, pages 979 - 990
MANNO CS; PIERCE GF; ARRUDA VR; GLADER B; RAGNI M; RASKO JJ; OZELO MC; HOOTS K; BLATT P; KONKLE B ET AL.: "Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.", NAT MED, vol. 12, no. 3, 2006, pages 342 - 347
MANNO CS; CHEW AJ; HUTCHISON S; LARSON PJ; HERZOG RW; ARRUDA VR; TAI SJ; RAGNI MV; THOMPSON A; OZELO M ET AL.: "AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.", BLOOD, vol. 101, no. 8, 2003, pages 2963 - 2972
WHITE SJ; NICKLIN SA; BUNING H; BROSNAN MJ; LEIKE K; PAPADAKIS ED; HALLEK M; BAKER AH: "Targeted gene delivery to vascular tissue in vivo by tropism-modified adeno-associated virus vectors", CIRCULATION, vol. 109, no. 4, 2004, pages 513 - 519
YU CY; YUAN Z; CAO Z; WANG B; QIAO C; LI J; XIAO X: "A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery", GENE THER, vol. 16, no. 8, 2009, pages 953 - 962
WORK LM; BUNING H; HUNT E; NICKLIN SA; DENBY L; BRITTON N; LEIKE K; ODENTHAL M; DREBBER U; HALLEK M ET AL.: "Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses", MOL THER, vol. 13, no. 4, 2006, pages 683 - 693
DOUAR AM; POULARD K; DANOS O: "Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid", VIROLOGY, vol. 309, no. 2, 2003, pages 203 - 208
NING X; GUO J; WOLFERT MA; BOONS GJ: "Visualizing metabolically labeled glycoconjugates of living cells by copper-free and fast huisgen cycloadditions", ANGEW CHEM INT ED ENGL, vol. 47, no. 12, 2008, pages 2253 - 2255
JOO KI; FANG Y; LIU Y; XIAO L; GU Z; TAI A; LEE CL; TANG Y; WANG P: "Enhanced real-time monitoring of adeno-associated virus trafficking by virus-quantum dot conjugates", ACS NANO, vol. 5, no. 5, pages 3523 - 3535
ROHR, U. P.; WULF, M. A.; STAHN, S.; STEIDL, U.; HAAS, R.; KRONENWETT, R., J VIROL METHODS, vol. 106, 2002, pages 81 - 8
TRAVIS S.: "Young and Peter G. Schultz: Beyond the Canonical 20 Amino Acids: Expanding the Genetic Lexicon", J BIOL. CHEM., vol. 285, 2010, pages 11039 - 11044
JIANMING XIE; PETER G SCHULTZ: "Adding amino acids to the genetic repertoire", CURRENT OPINION IN CHEMICAL BIOLOGY, vol. 9, 2005, pages 548 - 554
Attorney, Agent or Firm:
CCPIT PATENT AND TRADEMARK LAW OFFICE (CN)
中国国际贸易促进委员会专利商标事务所 (CN)
中国国际贸易促进委员会专利商标事务所 (CN)
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