메뉴 건너뛰기




Volumn 283, Issue 5398, 1999, Pages 88-91

Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; RAPAMYCIN;

EID: 0032949030     PISSN: 00368075     EISSN: None     Source Type: Journal    
DOI: 10.1126/science.283.5398.88     Document Type: Article
Times cited : (279)

References (31)
  • 1
    • 0030671462 scopus 로고    scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1997) Hum. Gene Ther. , vol.8 , pp. 1891
    • Snyder, R.O.1
  • 2
    • 0029744337 scopus 로고    scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1996) Proc. Natl. Acad. Sci. U.S.A. , vol.93 , pp. 10876
    • Tripathy, S.K.1
  • 3
    • 0029874105 scopus 로고    scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1996) Hum. Gene Ther. , vol.7 , pp. 11
    • Naffakh, N.1
  • 4
    • 0030671462 scopus 로고    scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1997) Nature Genet. , vol.8 , pp. 270
    • Snyder, R.O.1
  • 5
    • 0029943193 scopus 로고    scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1996) Blood , vol.87 , pp. 4671
    • Connelly, S.1    Gardner, J.M.2    Lyons, R.M.3    McClelland, A.4    Kaleko, M.5
  • 6
    • 0027231473 scopus 로고
    • R. O. Snyder et al., Hum. Gene Ther. 8, 1891 (1997); S. K. Tripathy et al., Proc. Natl. Acad. Sci. U.S.A. 93, 10876 (1996); N. Naffakh et al., Hum. Gene Ther. 7, 11 (1996); R. O. Snyder et al., Nature Genet. 8, 270 (1997); S. Connelly, J. M. Gardner, R. M. Lyons, A. McClelland, M. Kaleko, Blood 87, 4671 (1996); P. Moullier, D. Bohl, J. M. Heard, O. Danos, Nature Genet. 4, 154 (1993).
    • (1993) Nature Genet. , vol.4 , pp. 154
    • Moullier, P.1    Bohl, D.2    Heard, J.M.3    Danos, O.4
  • 7
    • 0031046584 scopus 로고    scopus 로고
    • D. Bohl and J. M. Heard, Hum. Gene Ther. 8, 195 (1997); D. Bohl, N. Naffakh, J. M. Heard, Nature Med. 3, 299 (1997); Y. Wang, J. Xu, T. Pierson, B. W. O'Malley, S. Y. Tsai, Gene Ther. 5, 432 (1997); Y. Wang, B. W. O'Malley Jr., S. Y. Tsai, B. W. O'Malley, Proc. Natl. Acad. Sci. U.S.A. 91, 8180 (1994); D. No, T. P. Yao, R. M. Evans, ibid. 93, 3346 (1996).
    • (1997) Hum. Gene Ther. , vol.8 , pp. 195
    • Bohl, D.1    Heard, J.M.2
  • 8
    • 0031040987 scopus 로고    scopus 로고
    • D. Bohl and J. M. Heard, Hum. Gene Ther. 8, 195 (1997); D. Bohl, N. Naffakh, J. M. Heard, Nature Med. 3, 299 (1997); Y. Wang, J. Xu, T. Pierson, B. W. O'Malley, S. Y. Tsai, Gene Ther. 5, 432 (1997); Y. Wang, B. W. O'Malley Jr., S. Y. Tsai, B. W. O'Malley, Proc. Natl. Acad. Sci. U.S.A. 91, 8180 (1994); D. No, T. P. Yao, R. M. Evans, ibid. 93, 3346 (1996).
    • (1997) Nature Med. , vol.3 , pp. 299
    • Bohl, D.1    Naffakh, N.2    Heard, J.M.3
  • 9
    • 0031001166 scopus 로고    scopus 로고
    • D. Bohl and J. M. Heard, Hum. Gene Ther. 8, 195 (1997); D. Bohl, N. Naffakh, J. M. Heard, Nature Med. 3, 299 (1997); Y. Wang, J. Xu, T. Pierson, B. W. O'Malley, S. Y. Tsai, Gene Ther. 5, 432 (1997); Y. Wang, B. W. O'Malley Jr., S. Y. Tsai, B. W. O'Malley, Proc. Natl. Acad. Sci. U.S.A. 91, 8180 (1994); D. No, T. P. Yao, R. M. Evans, ibid. 93, 3346 (1996).
    • (1997) Gene Ther. , vol.5 , pp. 432
    • Wang, Y.1    Xu, J.2    Pierson, T.3    O'Malley, B.W.4    Tsai, S.Y.5
  • 10
    • 0027965067 scopus 로고
    • D. Bohl and J. M. Heard, Hum. Gene Ther. 8, 195 (1997); D. Bohl, N. Naffakh, J. M. Heard, Nature Med. 3, 299 (1997); Y. Wang, J. Xu, T. Pierson, B. W. O'Malley, S. Y. Tsai, Gene Ther. 5, 432 (1997); Y. Wang, B. W. O'Malley Jr., S. Y. Tsai, B. W. O'Malley, Proc. Natl. Acad. Sci. U.S.A. 91, 8180 (1994); D. No, T. P. Yao, R. M. Evans, ibid. 93, 3346 (1996).
    • (1994) Proc. Natl. Acad. Sci. U.S.A. , vol.91 , pp. 8180
    • Wang, Y.1    O'Malley B.W., Jr.2    Tsai, S.Y.3    O'Malley, B.W.4
  • 11
    • 0029865151 scopus 로고    scopus 로고
    • D. Bohl and J. M. Heard, Hum. Gene Ther. 8, 195 (1997); D. Bohl, N. Naffakh, J. M. Heard, Nature Med. 3, 299 (1997); Y. Wang, J. Xu, T. Pierson, B. W. O'Malley, S. Y. Tsai, Gene Ther. 5, 432 (1997); Y. Wang, B. W. O'Malley Jr., S. Y. Tsai, B. W. O'Malley, Proc. Natl. Acad. Sci. U.S.A. 91, 8180 (1994); D. No, T. P. Yao, R. M. Evans, ibid. 93, 3346 (1996).
    • (1996) Proc. Natl. Acad. Sci. U.S.A. , vol.93 , pp. 3346
    • No, D.1    Yao, T.P.2    Evans, R.M.3
  • 12
    • 0028360374 scopus 로고
    • E. J. Brown et al., Nature 369, 756 (1994); R. F. Standaert, A. Galat, G. L. Verdine, S. L. Schrieber, ibid. 346, 671 (1990).
    • (1994) Nature , vol.369 , pp. 756
    • Brown, E.J.1
  • 14
    • 9544226448 scopus 로고    scopus 로고
    • V. M. Rivera et al., Nature Med. 2, 1028 (1996); S. R. Magari, V. M. Rivera, J. D. Iuliucci, M. Gilman, F. Cerasoli Jr., J. Clin. Invest. 100, 2865 (1997).
    • (1996) Nature Med. , vol.2 , pp. 1028
    • Rivera, V.M.1
  • 17
    • 0029843596 scopus 로고    scopus 로고
    • P. D. Kessler et al., Proc. Natl. Acad. Sci. U.S.A. 93, 14082 (1996); X. Xiao, J. Li, R. J. Samulski, J. Virol. 70, 8098 (1996).
    • (1996) J. Virol. , vol.70 , pp. 8098
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 20
    • 0345536075 scopus 로고    scopus 로고
    • note
    • 2-terminal epitope tag from influenza virus hemagglutin and nuclear localization signal from SV40 large T antigen (4, 16). Reporter construct AAV.Z12mEpo3S3 contains the 12 ZFHD1-binding sites (4), a minimal interleukin-2 promoter (4), a chimeric intron from pCl vector (Promega), a murine Epo cDNA, an SV40 late gene 3′ UTR, and a 2.7-kb stuffer containing a 1367-bp internal portion of human placental alkaline phosphatase coding sequence followed by a 626-bp portion of the human growth hormone 3′ UTR and a 720-bp portion of the 3′ rabbit β-globin intron and PAS (4). Reporter construct AAV.Z12rmEpo2S2 is similar except that the Epo cDNA is derived from a rhesus monkey Epo and the vector lacks the chimeric intron and the 720-bp rabbit β-globin portion of the stuffer. Reporter construct AAV.CM-VrmEpo3 contains a human CMV enhancer-promoter, a chimeric intron from pCl vector, a rhesus monkey Epo cDNA, and an SV40 late gene 3′ UTR. Recombinant AAV was generated by cloning CMVTF1, Z12mEpo3S3, Z12rmEpo2S2, and CMVrmEpo3 expression cassettes into Xba I-restricted pSub 201 backbone (17). The resulting plasmids were used to produce recombinant AAV by a triple transfection method (18) or by an Ad/AAV hybrid system (17).
  • 22
    • 0344242372 scopus 로고    scopus 로고
    • X. Ye et al., unpublished data
    • X. Ye et al., unpublished data.
  • 24
    • 0345536071 scopus 로고    scopus 로고
    • note
    • 13 genomes per kilogram) peak Epo levels of 560 and 120 mU/ml.
  • 25
    • 0344673816 scopus 로고    scopus 로고
    • note
    • Antibodies to the influenza virus hemagglutinin tag were detected in serum of the nonhuman primate 3 months after gene transfer, although it is unclear whether their presence predicts functional cellular immune responses.
  • 27
    • 0030042794 scopus 로고    scopus 로고
    • X. Ye et al., J. Biol. Chem. 271, 3639 (1996).
    • (1996) J. Biol. Chem. , vol.271 , pp. 3639
    • Ye, X.1
  • 31
    • 0345104667 scopus 로고    scopus 로고
    • note
    • Funding was provided by grants from the NIH [P30 DK47757-05 (J.M.W.) and P01 AR/NS43648-03 (H. L. Sweeney)], the Muscular Dystrophy Association, ARIAD Pharmaceuticals, and Genovo, a company J.M.W. founded and in which he holds equities. Technical support from N. Courage was greatly appreciated. W. Xiao provided plasmids for AAV production.


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.