Adeno-associated virus vectors: potential applications for cancer gene therapy

被引:83
作者
Li, CW
Bowles, DE
van Dyke, T
Samulski, RJ
机构
[1] Univ N Carolina, Gene Therapy Ctr, Chapel Hill, NC 27599 USA
[2] Univ N Carolina, Dept Biochem & Biophys, Chapel Hill, NC 27599 USA
[3] Univ N Carolina, Lineberger Comprehens Canc Ctr, Chapel Hill, NC 27599 USA
[4] Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27599 USA
关键词
adeno-associated virus vector;
D O I
10.1038/sj.cgt.7700876
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
Augmenting cancer treatment by protein and gene delivery continues to gain momentum based on success in animal models. The primary hurdle of fully exploiting the arsenal of molecular targets and therapeutic transgenes continues to be efficient delivery. Vectors based on adeno-associated virus (AAV) are of particular interest as they are capable of inducing transgene expression in a broad range of tissues for a relatively long time without stimulation of a cell-mediated immune response. Perhaps the most important attribute of AAV vectors is their safety profile in phase I clinical trials ranging from CF to Parkinson's disease. The utility of AAV vectors as a gene delivery agent in cancer therapy is showing promise in preclinical studies. In this review, we will focus on the basic biology of AAV as well as recent progress in the use of this vector in cancer gene therapy.
引用
收藏
页码:913 / 925
页数:13
相关论文
共 177 条
[91]   An AAV-derived Apaf-1 dominant negative inhibitor prevents MPTP toxicity as antiapoptotic gene therapy for Parkinson's disease [J].
Mochizuki, H ;
Hayakawa, H ;
Migita, M ;
Shibata, M ;
Tanaka, R ;
Suzuki, A ;
Shimo-Nakanishi, Y ;
Urabe, T ;
Yamada, M ;
Tamayose, K ;
Shimada, T ;
Miura, M ;
Mizuno, Y .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2001, 98 (19) :10918-10923
[92]   AAV-encoded expression of TRAIL in experimental human colorectal cancer leads to tumor regression [J].
Mohr, A ;
Henderson, G ;
Dudus, L ;
Herr, I ;
Kuerschner, T ;
Debatin, KM ;
Weiher, H ;
Fisher, KJ ;
Zwacka, RM .
GENE THERAPY, 2004, 11 (06) :534-543
[93]   Adeno-associated virus vectors for gene therapy: more pros than cons? [J].
Monahan, PE ;
Samulski, RJ .
MOLECULAR MEDICINE TODAY, 2000, 6 (11) :433-440
[94]   Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia [J].
Monahan, PE ;
Samulski, RJ ;
Tazelaar, J ;
Xiao, X ;
Nichols, TC ;
Bellinger, DA ;
Read, MS ;
Walsh, CE .
GENE THERAPY, 1998, 5 (01) :40-49
[95]   Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: Implications for gene therapy and virus structure [J].
Moskalenko, M ;
Chen, LL ;
van Roey, M ;
Donahue, BA ;
Snyder, RO ;
McArthur, JG ;
Patel, SD .
JOURNAL OF VIROLOGY, 2000, 74 (04) :1761-1766
[96]   Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors [J].
Müller, OJ ;
Kaul, F ;
Weitzman, MD ;
Pasqualini, R ;
Arap, W ;
Kleinschmidt, JA ;
Trepel, M .
NATURE BIOTECHNOLOGY, 2003, 21 (09) :1040-1046
[97]   Nucleotide sequencing and generation of an infectious clone of adeno-associated virus 3 [J].
Muramatsu, S ;
Mizukami, H ;
Young, NS ;
Brown, KE .
VIROLOGY, 1996, 221 (01) :208-217
[98]   Behavioral recovery in a primate model of Parkinson's disease by triple transduction of striatal cells with adeno-associated viral vectors expressing dopamine-synthesizing enzymes [J].
Muramatsu, SI ;
Fujimoto, KI ;
Ikeguchi, K ;
Shizuma, N ;
Kawasaki, K ;
Ono, F ;
Shen, Y ;
Wang, LJ ;
Mizukami, H ;
Kume, A ;
Matsumura, M ;
Nagatsu, I ;
Urano, F ;
Ichinose, H ;
Nagatsu, T ;
Terao, K ;
Nakano, I ;
Ozawa, K .
HUMAN GENE THERAPY, 2002, 13 (03) :345-354
[99]  
MUZYCZKA N, 1992, CURR TOP MICROBIOL, V158, P97
[100]   Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors [J].
Nakai, H ;
Storm, TA ;
Kay, MA .
NATURE BIOTECHNOLOGY, 2000, 18 (05) :527-532