-
1
-
-
44049094771
-
Clinical gene therapy using recombinant adeno-associated virus vectors
-
10.1038/gt.2008.68, 18418415
-
Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 2008, 15:858-863. 10.1038/gt.2008.68, 18418415.
-
(2008)
Gene Ther
, vol.15
, pp. 858-863
-
-
Mueller, C.1
Flotte, T.R.2
-
2
-
-
0029843596
-
Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector
-
190884, 8892935
-
Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996, 70:8098-8108. 190884, 8892935.
-
(1996)
J Virol
, vol.70
, pp. 8098-8108
-
-
Xiao, X.1
Li, J.2
Samulski, R.J.3
-
3
-
-
0032853699
-
Immune responses to adenovirus and adeno-associated virus in humans
-
10.1038/sj.gt.3300994, 10490767
-
Chirmule N, Propert K, Magosin S, Qian Y, Qian R, Wilson J. Immune responses to adenovirus and adeno-associated virus in humans. Gene Ther 1999, 6:1574-1583. 10.1038/sj.gt.3300994, 10490767.
-
(1999)
Gene Ther
, vol.6
, pp. 1574-1583
-
-
Chirmule, N.1
Propert, K.2
Magosin, S.3
Qian, Y.4
Qian, R.5
Wilson, J.6
-
4
-
-
77956628142
-
RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy
-
10.1002/jgm.1496, 2948027, 20821747
-
Hu C, Busuttil RW, Lipshutz GS. RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy. J Gene Med 2010, 12:766-778. 10.1002/jgm.1496, 2948027, 20821747.
-
(2010)
J Gene Med
, vol.12
, pp. 766-778
-
-
Hu, C.1
Busuttil, R.W.2
Lipshutz, G.S.3
-
5
-
-
44349170706
-
Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection
-
10.1038/mt.2008.76, 18414476
-
Zincarelli C, Soltys S, Rengo G, Rabinowitz JE. Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection. Mol Ther 2008, 16:1073-1080. 10.1038/mt.2008.76, 18414476.
-
(2008)
Mol Ther
, vol.16
, pp. 1073-1080
-
-
Zincarelli, C.1
Soltys, S.2
Rengo, G.3
Rabinowitz, J.E.4
-
6
-
-
79959201563
-
The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
-
10.1182/blood-2010-10-314518, 3123017, 21474674
-
Martino AT, Suzuki M, Markusic DM, Zolotukhin I, Ryals RC, Moghimi B, Ertl HC, Muruve DA, Lee B, Herzog RW. The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver. Blood 2011, 117:6459-6468. 10.1182/blood-2010-10-314518, 3123017, 21474674.
-
(2011)
Blood
, vol.117
, pp. 6459-6468
-
-
Martino, A.T.1
Suzuki, M.2
Markusic, D.M.3
Zolotukhin, I.4
Ryals, R.C.5
Moghimi, B.6
Ertl, H.C.7
Muruve, D.A.8
Lee, B.9
Herzog, R.W.10
-
7
-
-
44049090692
-
AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects
-
10.1038/gt.2008.71, 18432276
-
Hasbrouck NC, High KA. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther 2008, 15:870-875. 10.1038/gt.2008.71, 18432276.
-
(2008)
Gene Ther
, vol.15
, pp. 870-875
-
-
Hasbrouck, N.C.1
High, K.A.2
-
8
-
-
57749190573
-
Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells
-
10.1089/hum.2008.117, 18778196
-
White AF, Mazur M, Sorscher EJ, Zinn KR, Ponnazhagan S. Genetic modification of adeno-associated viral vector type 2 capsid enhances gene transfer efficiency in polarized human airway epithelial cells. Hum Gene Ther 2008, 19:1407-1414. 10.1089/hum.2008.117, 18778196.
-
(2008)
Hum Gene Ther
, vol.19
, pp. 1407-1414
-
-
White, A.F.1
Mazur, M.2
Sorscher, E.J.3
Zinn, K.R.4
Ponnazhagan, S.5
-
9
-
-
79957918498
-
The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice
-
10.1172/JCI57367, 3104778, 21576824
-
Bell CL, Vandenberghe LH, Bell P, Limberis MP, Gao GP, Van Vliet K, Agbandje-McKenna M, Wilson JM. The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J Clin Invest 2011, 121:2427-2435. 10.1172/JCI57367, 3104778, 21576824.
-
(2011)
J Clin Invest
, vol.121
, pp. 2427-2435
-
-
Bell, C.L.1
Vandenberghe, L.H.2
Bell, P.3
Limberis, M.P.4
Gao, G.P.5
Van Vliet, K.6
Agbandje-McKenna, M.7
Wilson, J.M.8
-
10
-
-
0031906147
-
Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions
-
124624, 9445046
-
Summerford C, Samulski RJ. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J Virol 1998, 72:1438-1445. 124624, 9445046.
-
(1998)
J Virol
, vol.72
, pp. 1438-1445
-
-
Summerford, C.1
Samulski, R.J.2
-
11
-
-
0038618725
-
Identification of amino acid residues in the capsid proteins of adeno-associated virus type 2 that contribute to heparan sulfate proteoglycan binding
-
10.1128/JVI.77.12.6995-7006.2003, 156206, 12768018
-
Opie SR, Warrington KH, 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 2003, 77:6995-7006. 10.1128/JVI.77.12.6995-7006.2003, 156206, 12768018.
-
(2003)
J Virol
, vol.77
, pp. 6995-7006
-
-
Opie, S.R.1
Warrington, K.H.2
Agbandje-McKenna, M.3
Zolotukhin, S.4
Muzyczka, N.5
-
12
-
-
0033851104
-
Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism
-
10.1128/JVI.74.18.8635-8647.2000, 102071, 10954565
-
Wu P, Xiao W, Conlon T, Hughes J, Agbandje-McKenna M, Ferkol T, Flotte T, Muzyczka N. Mutational analysis of the adeno-associated virus type 2 (AAV2) capsid gene and construction of AAV2 vectors with altered tropism. J Virol 2000, 74:8635-8647. 10.1128/JVI.74.18.8635-8647.2000, 102071, 10954565.
-
(2000)
J Virol
, vol.74
, pp. 8635-8647
-
-
Wu, P.1
Xiao, W.2
Conlon, T.3
Hughes, J.4
Agbandje-McKenna, M.5
Ferkol, T.6
Flotte, T.7
Muzyczka, N.8
-
13
-
-
69249213448
-
A muscle-targeting peptide displayed on AAV2 improves muscle tropism on systemic delivery
-
10.1038/gt.2009.59, 2726895, 19474807
-
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 2009, 16:953-962. 10.1038/gt.2009.59, 2726895, 19474807.
-
(2009)
Gene Ther
, vol.16
, pp. 953-962
-
-
Yu, C.Y.1
Yuan, Z.2
Cao, Z.3
Wang, B.4
Qiao, C.5
Li, J.6
Xiao, X.7
-
14
-
-
0034843445
-
Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells
-
10.1006/mthe.2001.0424, 11545607
-
Nicklin SA, Buening H, Dishart KL, de Alwis M, Girod A, Hacker U, Thrasher AJ, Ali RR, Hallek M, Baker AH. Efficient and selective AAV2-mediated gene transfer directed to human vascular endothelial cells. Mol Ther 2001, 4:174-181. 10.1006/mthe.2001.0424, 11545607.
-
(2001)
Mol Ther
, vol.4
, pp. 174-181
-
-
Nicklin, S.A.1
Buening, H.2
Dishart, K.L.3
de Alwis, M.4
Girod, A.5
Hacker, U.6
Thrasher, A.J.7
Ali, R.R.8
Hallek, M.9
Baker, A.H.10
-
15
-
-
0034963928
-
Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids
-
10.1006/mthe.2001.0345, 11407911
-
Grifman M, Trepel M, Speece P, Gilbert LB, Arap W, Pasqualini R, Weitzman MD. Incorporation of tumor-targeting peptides into recombinant adeno-associated virus capsids. Mol Ther 2001, 3:964-975. 10.1006/mthe.2001.0345, 11407911.
-
(2001)
Mol Ther
, vol.3
, pp. 964-975
-
-
Grifman, M.1
Trepel, M.2
Speece, P.3
Gilbert, L.B.4
Arap, W.5
Pasqualini, R.6
Weitzman, M.D.7
-
16
-
-
0034056847
-
Selective drug delivery system to bone: small peptide (Asp)6 conjugation
-
Kasugai S, Fujisawa R, Waki Y, Miyamoto K, Ohya K. Selective drug delivery system to bone: small peptide (Asp)6 conjugation. J Bone Miner Res 2000, 15:936-943.
-
(2000)
J Bone Miner Res
, vol.15
, pp. 936-943
-
-
Kasugai, S.1
Fujisawa, R.2
Waki, Y.3
Miyamoto, K.4
Ohya, K.5
-
17
-
-
33745497185
-
Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide
-
10.1016/j.ymgme.2006.02.012, 2587042, 16616566
-
Nishioka T, Tomatsu S, Gutierrez MA, Miyamoto K, Trandafirescu GG, Lopez PL, Grubb JH, Kanai R, Kobayashi H, Yamaguchi S, et al. Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide. Mol Genet Metab 2006, 88:244-255. 10.1016/j.ymgme.2006.02.012, 2587042, 16616566.
-
(2006)
Mol Genet Metab
, vol.88
, pp. 244-255
-
-
Nishioka, T.1
Tomatsu, S.2
Gutierrez, M.A.3
Miyamoto, K.4
Trandafirescu, G.G.5
Lopez, P.L.6
Grubb, J.H.7
Kanai, R.8
Kobayashi, H.9
Yamaguchi, S.10
-
18
-
-
77953140333
-
Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome
-
10.1038/mt.2010.32, 2889747, 20332769
-
Tomatsu S, Montano AM, Dung VC, Ohashi A, Oikawa H, Oguma T, Orii T, Barrera L, Sly WS. Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome. Mol Ther 2010, 18:1094-1102. 10.1038/mt.2010.32, 2889747, 20332769.
-
(2010)
Mol Ther
, vol.18
, pp. 1094-1102
-
-
Tomatsu, S.1
Montano, A.M.2
Dung, V.C.3
Ohashi, A.4
Oikawa, H.5
Oguma, T.6
Orii, T.7
Barrera, L.8
Sly, W.S.9
-
19
-
-
81855216435
-
Rescue of severe infantile hypophosphatasia mice by AAV-mediated sustained expression of soluble alkaline phosphatase
-
10.1089/hum.2010.210, 21388343
-
Matsumoto T, Miyake K, Yamamoto S, Orimo H, Miyake N, Odagaki Y, Adachi K, Iijima O, Narisawa S, Millan JL, et al. Rescue of severe infantile hypophosphatasia mice by AAV-mediated sustained expression of soluble alkaline phosphatase. Hum Gene Ther 2011, 22(11):1355-1364. 10.1089/hum.2010.210, 21388343.
-
(2011)
Hum Gene Ther
, vol.22
, Issue.11
, pp. 1355-1364
-
-
Matsumoto, T.1
Miyake, K.2
Yamamoto, S.3
Orimo, H.4
Miyake, N.5
Odagaki, Y.6
Adachi, K.7
Iijima, O.8
Narisawa, S.9
Millan, J.L.10
-
20
-
-
0036428801
-
Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
-
10.1016/S1046-2023(02)00220-7, 12413414
-
Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW, et al. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods 2002, 28:158-167. 10.1016/S1046-2023(02)00220-7, 12413414.
-
(2002)
Methods
, vol.28
, pp. 158-167
-
-
Zolotukhin, S.1
Potter, M.2
Zolotukhin, I.3
Sakai, Y.4
Loiler, S.5
Fraites, T.J.6
Chiodo, V.A.7
Phillipsberg, T.8
Muzyczka, N.9
Hauswirth, W.W.10
-
21
-
-
77958099436
-
Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material
-
10.1089/hum.2009.223, 2957240, 20486768
-
Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, et al. Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Hum Gene Ther 2010, 21:1273-1285. 10.1089/hum.2009.223, 2957240, 20486768.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1273-1285
-
-
Lock, M.1
McGorray, S.2
Auricchio, A.3
Ayuso, E.4
Beecham, E.J.5
Blouin-Tavel, V.6
Bosch, F.7
Bose, M.8
Byrne, B.J.9
Caton, T.10
-
22
-
-
16844383364
-
Evaluation of the patient with muscle weakness
-
Saguil A. Evaluation of the patient with muscle weakness. Am Fam Physician 2005, 71:1327-1336.
-
(2005)
Am Fam Physician
, vol.71
, pp. 1327-1336
-
-
Saguil, A.1
|