Gene Transfer into Primates and Prospects for Gene Therapy in Humans

作者: Kenneth Cornetta , Robert Wieder , W. French Anderson

DOI: 10.1016/S0079-6603(08)60179-8

关键词: Vector (molecular biology)Gene transferAdenosine deaminaseGeneticsGeneGenetic enhancementBone marrowBiologyIn vitroCell biologyIn vivo

摘要: Publisher Summary The ability to transfer and express genetic material in mammalian cells presents a new approach the treatment of many diseases. Retroviral-mediated gene has proven be an efficient system vitro. While vivo expression been obtained mouse, vectors expressing murine systems do not necessarily primates. Retroviral infect primate bone marrow transferred genes . SAX vector appears human adenosine deaminase (ADA) at normal levels, but infection efficiency is low (

参考文章(23)
G S Adrian, D A Wiginton, J J Hutton, Structure of adenosine deaminase mRNAs from normal and adenosine deaminase-deficient human cell lines. Molecular and Cellular Biology. ,vol. 4, pp. 1712- 1717 ,(1984) , 10.1128/MCB.4.9.1712
D Armentano, S F Yu, P W Kantoff, T von Ruden, W F Anderson, E Gilboa, Effect of internal viral sequences on the utility of retroviral vectors. Journal of Virology. ,vol. 61, pp. 1647- 1650 ,(1987) , 10.1128/JVI.61.5.1647-1650.1987
D Markowitz, S Goff, A Bank, A safe packaging line for gene transfer: separating viral genes on two different plasmids. Journal of Virology. ,vol. 62, pp. 1120- 1124 ,(1988) , 10.1128/JVI.62.4.1120-1124.1988
A F Voronova, J E Buss, T Patschinsky, T Hunter, B M Sefton, Characterization of the protein apparently responsible for the elevated tyrosine protein kinase activity in LSTRA cells. Molecular and Cellular Biology. ,vol. 4, pp. 2705- 2713 ,(1984) , 10.1128/MCB.4.12.2705
David A. Williams, Ihor R. Lemischka, David G. Nathan, Richard C. Mulligan, Introduction of new genetic material into pluripotent haematopoietic stem cells of the mouse Nature. ,vol. 310, pp. 476- 480 ,(1984) , 10.1038/310476A0
A. Miller, R. Eckner, D. Jolly, T Friedmann, I. Verma, Expression of a retrovirus encoding human HPRT in mice Science. ,vol. 225, pp. 630- 632 ,(1984) , 10.1126/SCIENCE.6377498
Jeanne R. McLachlin, Sheri C. Bernstein, W.French Anderson, Separation of human from mouse and monkey adenosine deaminase by lon-exchange chromatography following retroviral-mediated gene transfer Analytical Biochemistry. ,vol. 163, pp. 143- 150 ,(1987) , 10.1016/0003-2697(87)90104-7
Alexandra Joyner, Gordon Keller, Robert A. Phillips, Alan Bernstein, Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells Nature. ,vol. 305, pp. 556- 558 ,(1983) , 10.1038/305556A0
A. Dusty Miller, David R. Trauber, Carol Buttimore, Factors involved in production of helper virus-free retrovirus vectors Somatic Cell and Molecular Genetics. ,vol. 12, pp. 175- 183 ,(1986) , 10.1007/BF01560664