-
1
-
-
0027595949
-
The basic science of gene therapy
-
Mulligan, R.C. The basic science of gene therapy. Science 260 926-932(1993).
-
(1993)
Science
, vol.260
, pp. 926-932
-
-
Mulligan, R.C.1
-
2
-
-
22244459917
-
Gene therapy: Twenty-first century medicine
-
Verma, I.M. & Weitzman, M.D. Gene therapy: Twenty-first century medicine. Annu. Rev. Biochem. 74, 711-738 (2005).
-
(2005)
Annu. Rev. Biochem
, vol.74
, pp. 711-738
-
-
Verma, I.M.1
Weitzman, M.D.2
-
3
-
-
0034050904
-
Evidence for gene transfer and expression of factor IX in hemophilia B patients treated with an AAV vector
-
Kay, M.A. et al. Evidence for gene transfer and expression of factor IX in hemophilia B patients treated with an AAV vector. Nat. Genet. 24, 257-261 (2000).
-
(2000)
Nat. Genet
, vol.24
, pp. 257-261
-
-
Kay, M.A.1
-
4
-
-
0013442744
-
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
-
Manna, C.S. et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101 2963-2972 (2003).
-
(2003)
Blood
, vol.101
, pp. 2963-2972
-
-
Manna, C.S.1
-
5
-
-
0020684648
-
Nucleotide sequence and organization of the adeno-associated virus 2 genome
-
Srivastava, A., Lusby, E.W. & Berns, K.I. Nucleotide sequence and organization of the adeno-associated virus 2 genome. J. Virol. 45, 555-564 (1983).
-
(1983)
J. Virol
, vol.45
, pp. 555-564
-
-
Srivastava, A.1
Lusby, E.W.2
Berns, K.I.3
-
6
-
-
0024311525
-
Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression
-
Samulski, R.J., Chang, L.S. & Shenk, T. Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63, 3822-3828 (1989).
-
(1989)
J. Virol
, vol.63
, pp. 3822-3828
-
-
Samulski, R.J.1
Chang, L.S.2
Shenk, T.3
-
7
-
-
0032959311
-
Cloning and characterization of adeno-associated virus type 5
-
Chiorini, J.A., Kim, F., Yang, L. & Kotin, R.M. Cloning and characterization of adeno-associated virus type 5. J. Virol. 73 1309-1319 (1999).
-
(1999)
J. Virol
, vol.73
, pp. 1309-1319
-
-
Chiorini, J.A.1
Kim, F.2
Yang, L.3
Kotin, R.M.4
-
8
-
-
2642512201
-
Clades of Adeno-associated viruses are widely disseminated in human tissues
-
Gao, G. et al. Clades of Adeno-associated viruses are widely disseminated in human tissues. J. Virol. 78, 6381-6388 (2004).
-
(2004)
J. Virol
, vol.78
, pp. 6381-6388
-
-
Gao, G.1
-
9
-
-
0037015049
-
Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
-
Gao, G.P. et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. USA 99, 11854-11859 (2002).
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 11854-11859
-
-
Gao, G.P.1
-
10
-
-
0036678455
-
The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy
-
Xie, Q. et al. The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy. Proc. Natl. Acad. Sci. USA 99, 10406-10410 (2002).
-
(2002)
Proc. Natl. Acad. Sci. USA
, vol.99
, pp. 10406-10410
-
-
Xie, Q.1
-
11
-
-
0033590138
-
Insertional mutagenesis of AAV2 capsid and the production of recombinant virus
-
Rabinowitz, J.E., Xiao, W. & Samulski, R.J. Insertional mutagenesis of AAV2 capsid and the production of recombinant virus. Virology 265, 274-285 (1999).
-
(1999)
Virology
, vol.265
, pp. 274-285
-
-
Rabinowitz, J.E.1
Xiao, W.2
Samulski, R.J.3
-
12
-
-
0033851104
-
Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism
-
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).
-
(2000)
J. Virol
, vol.74
, pp. 8635-8647
-
-
Wu, P.1
-
13
-
-
30344450073
-
Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization
-
Lochrie, M.A. et al. Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J. Virol. 80, 821-834 (2006).
-
(2006)
J. Virol
, vol.80
, pp. 821-834
-
-
Lochrie, M.A.1
-
14
-
-
0033007934
-
Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody
-
Bartlett, J.S., Kleinschmidt, J., Boucher, R.C. & Samulski, R.J. Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'gamma)2 antibody. Nat. Biotechnol. 17, 181-186 (1999).
-
(1999)
Nat. Biotechnol
, vol.17
, pp. 181-186
-
-
Bartlett, J.S.1
Kleinschmidt, J.2
Boucher, R.C.3
Samulski, R.J.4
-
15
-
-
0032870158
-
Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2
-
Girod, A. et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat. Med. 5, 1052-1056 (1999).
-
(1999)
Nat. Med
, vol.5
, pp. 1052-1056
-
-
Girod, A.1
-
16
-
-
0042360384
-
Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors
-
Muller, O.J. et al. Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors. Nat. Biotechnol. 21, 1040-1046 (2003).
-
(2003)
Nat. Biotechnol
, vol.21
, pp. 1040-1046
-
-
Muller, O.J.1
-
17
-
-
0042031468
-
In vitro selection of viral vectors with modified tropism: The adeno-associated virus display
-
Perabo, L. et al. In vitro selection of viral vectors with modified tropism: The adeno-associated virus display. Mol. Ther. 8, 151-157 (2003).
-
(2003)
Mol. Ther
, vol.8
, pp. 151-157
-
-
Perabo, L.1
-
18
-
-
0038618725
-
Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding
-
Opie, S.R., Warrington, K.H., Jr., Agbandje-McKenna, M., Zolotukhin, S. & Muzyczka, N. Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding. J. Virol. 77, 6995-7006 (2003).
-
(2003)
J. Virol
, vol.77
, pp. 6995-7006
-
-
Opie, S.R.1
Warrington Jr., K.H.2
Agbandje-McKenna, M.3
Zolotukhin, S.4
Muzyczka, N.5
-
19
-
-
0033920298
-
Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: Implications for gene therapy and virus structure
-
Moskalenko, M. et al. Epitope mapping of human anti-adeno-associated virus type 2 neutralizing antibodies: Implications for gene therapy and virus structure. J. Virol. 74, 1761-1766 (2000).
-
(2000)
J. Virol
, vol.74
, pp. 1761-1766
-
-
Moskalenko, M.1
-
20
-
-
0033808531
-
Monoclonal antibodies against the adeno-associated virus type 2 (AAV-2) capsid: Epitope mapping and identification of capsid domains involved in AAV-2-cell interaction and neutralization of AAV-2 infection
-
Wobus, C.E. et al. Monoclonal antibodies against the adeno-associated virus type 2 (AAV-2) capsid: Epitope mapping and identification of capsid domains involved in AAV-2-cell interaction and neutralization of AAV-2 infection. J. Virol. 74, 9281-9293 (2000).
-
(2000)
J. Virol
, vol.74
, pp. 9281-9293
-
-
Wobus, C.E.1
-
21
-
-
32344438754
-
Directed evolution of adeno-associated virus yields enhanced gene delivery vectors
-
Maheshri, N., Koerber, J.T., Kaspar, B.K. & Schaffer, D.V. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat. Biotechnol. 24, 198-204 (2006).
-
(2006)
Nat. Biotechnol
, vol.24
, pp. 198-204
-
-
Maheshri, N.1
Koerber, J.T.2
Kaspar, B.K.3
Schaffer, D.V.4
-
22
-
-
0031909113
-
Molecular evolution by staggered extension process (StEP) in vitro recombination
-
Zhao, H., Giver, L., Shao, Z., Affholter, J.A. & Arnold, F.H. Molecular evolution by staggered extension process (StEP) in vitro recombination. Nat. Biotechnol. 16, 258-261 (1998).
-
(1998)
Nat. Biotechnol
, vol.16
, pp. 258-261
-
-
Zhao, H.1
Giver, L.2
Shao, Z.3
Affholter, J.A.4
Arnold, F.H.5
-
23
-
-
0028050350
-
Rapid evolution of a protein in vitro by DNA shuffling
-
Stemmer, W.P. Rapid evolution of a protein in vitro by DNA shuffling. Nature 370, 389-391 (1994).
-
(1994)
Nature
, vol.370
, pp. 389-391
-
-
Stemmer, W.P.1
-
24
-
-
0030994634
-
Yeast surface display for screening combinatorial polypeptide libraries
-
Boder, E.T. & Wittrup, K.D. Yeast surface display for screening combinatorial polypeptide libraries. Nat. Biotechnol. 15, 553-557 (1997).
-
(1997)
Nat. Biotechnol
, vol.15
, pp. 553-557
-
-
Boder, E.T.1
Wittrup, K.D.2
-
25
-
-
0034049316
-
Quantitative analysis of the effect of the mutation frequency on the affinity maturation of single chain Fv antibodies
-
Daugherty, P.S., Chen, G., Iverson, B.L. & Georgiou, G. Quantitative analysis of the effect of the mutation frequency on the affinity maturation of single chain Fv antibodies. Proc. Natl. Acad. Sci. USA 97, 2029-2034 (2000).
-
(2000)
Proc. Natl. Acad. Sci. USA
, vol.97
, pp. 2029-2034
-
-
Daugherty, P.S.1
Chen, G.2
Iverson, B.L.3
Georgiou, G.4
-
26
-
-
0034426015
-
Molecular breeding of viruses
-
Soong, N.W. et al. Molecular breeding of viruses. Nat. Genet. 25, 436-439 (2000).
-
(2000)
Nat. Genet
, vol.25
, pp. 436-439
-
-
Soong, N.W.1
-
27
-
-
0037218819
-
Sonic hedgehog regulates adult neural progenitor proliferation in viro and in vivo
-
Lai, K., Kaspar, B.K., Gage, F.H. & Schaffer, D.V. Sonic hedgehog regulates adult neural progenitor proliferation in viro and in vivo. Nat. Neurosci. 6, 21-27 (2003).
-
(2003)
Nat. Neurosci
, vol.6
, pp. 21-27
-
-
Lai, K.1
Kaspar, B.K.2
Gage, F.H.3
Schaffer, D.V.4
-
28
-
-
0035972806
-
Transfer of high copy number plasmid into mammalian cells by calcium phosphate transfection
-
Batard, P., Jordan, M. & Wurm, F. Transfer of high copy number plasmid into mammalian cells by calcium phosphate transfection. Gene 270, 61-68 (2001).
-
(2001)
Gene
, vol.270
, pp. 61-68
-
-
Batard, P.1
Jordan, M.2
Wurm, F.3
-
29
-
-
4444352441
-
Cross-dressing the virion: The transcapsidation of adeno-associated virus serotypes functionally defines subgroups
-
Rabinowitz, J.E. et al. Cross-dressing the virion: The transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J. Virol. 78, 4421-4432 (2004).
-
(2004)
J. Virol
, vol.78
, pp. 4421-4432
-
-
Rabinowitz, J.E.1
-
30
-
-
18444380824
-
Altering AAV tropism with mosaic viral capsids
-
Gigout, L. et al. Altering AAV tropism with mosaic viral capsids. Mol. Ther. 11, 856-865 (2005).
-
(2005)
Mol. Ther
, vol.11
, pp. 856-865
-
-
Gigout, L.1
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