-
1
-
-
79954622209
-
Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
-
F. Mingozzi, K. A. High, Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges. Nat. Rev. Genet. 12, 341-355 (2011).
-
(2011)
Nat. Rev. Genet.
, vol.12
, pp. 341-355
-
-
Mingozzi, F.1
High, K.A.2
-
2
-
-
84855161388
-
Adenovirus-associated virus vector-mediated gene transfer in hemophilia
-
A. C. Nathwani, E. G. D. Tuddenham, S. Rangarajan, C. Rosales, J. McIntosh, D. C. Linch, P. Chowdary, A. Riddell, A. J. Pie, C. Harrington, J. O'Beirne, K. Smith, J. Pasi, B. Glader, P. Rustagi, C. Y. C. Ng, M. A. Kay, J. Zhou, Y. Spence, C. L. Morton, J. Allay, J. Coleman, S. Sleep, J. M. Cunningham, D. Srivastava, E. Basner-Tschakarjan, F. Mingozzi, K. A. High, J. T. Gray, U. M. Reiss, A. W. Nienhuis, A. M. Davidoff, Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N. Engl. J. Med. 365, 2357-2365 (2011).
-
(2011)
B. N. Engl. J. Med.
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.D.2
Rangarajan, S.3
Rosales, C.4
McIntosh, J.5
Linch, D.C.6
Chowdary, P.7
Riddell, A.8
Pie, A.J.9
Harrington, C.10
O'Beirne, J.11
Smith, K.12
Pasi, J.13
Glader, B.14
Rustagi, P.15
Ng, C.Y.C.16
Kay, M.A.17
Zhou, J.18
Spence, Y.19
Morton, C.L.20
Allay, J.21
Coleman, J.22
Sleep, S.23
Cunningham, J.M.24
Srivastava, D.25
Basner-Tschakarjan, E.26
Mingozzi, F.27
High, K.A.28
Gray, J.T.29
Reiss, U.M.30
Nienhuis, A.W.31
Davidoff, A.M.32
more..
-
3
-
-
80455174705
-
AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta)
-
L. Wang, P. Bell, J. Lin, R. Calcedo, A. F. Tarantal, J. M. Wilson, AAV8-mediated hepatic gene transfer in infant rhesus monkeys (Macaca mulatta). Mol. Ther. 19, 2012-2020 (2011).
-
(2011)
Mol.Ther
, vol.19
, pp. 2012-2020
-
-
Wang, L.1
Bell, P.2
Lin, J.3
Calcedo, R.4
Tarantal, A.F.5
Wilson, J.M.6
-
4
-
-
67651006589
-
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors
-
G. C. Pien, E. Basner-Tschakarjan, D. J. Hui, A. N. Mentlik, J. D. Finn, N. C. Hasbrouck, S. Zhou, S. L. Murphy, M. V. Maus, F. Mingozzi, J. S. Orange, K. A. High, Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J. Clin. Invest. 119, 1688-1695 (2009).
-
(2009)
J. Clin. Invest.
, vol.119
, pp. 1688-1695
-
-
Pien, G.C.1
Basner-Tschakarjan, E.2
Hui, D.J.3
Mentlik, A.N.4
Finn, J.D.5
Hasbrouck, N.C.6
Zhou, S.7
Murphy, S.L.8
Maus, M.V.9
Mingozzi, F.10
Orange, J.S.11
High, K.A.12
-
5
-
-
35349027352
-
Immune responses to AAV in clinical trials
-
F. Mingozzi, K. A. High, Immune responses to AAV in clinical trials. Curr. Gene Ther. 7, 316-324 (2007).
-
(2007)
Curr. Gene Ther.
, vol.7
, pp. 316-324
-
-
Mingozzi, F.1
High, K.A.2
-
6
-
-
33644820684
-
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
-
C. S. Manno, G. F. Pierce, V. R. Arruda, B. Glader, M. Ragni, J. J. E. Rasko, M. C. Ozelo, K. Hoots, P. Blatt, B. Konkle, M. Dake, R. Kaye, M. Razavi, A. Zajko, J. Zehnder, P. Rustagi, H. Nakai, A. Chew, D. Leonard, J. F. Wright, R. R. Lessard, J. M. Sommer, M. Tigges, D. Sabatino, A. Luk, H. Jiang, F. Mingozzi, L. Couto, H. C. Ertl, K. A. High, M. A. Kay, Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342-347 (2006).
-
(2006)
Nat. Med.
, vol.12
, pp. 342-347
-
-
Manno, C.S.1
Pierce, G.F.2
Arruda, V.R.3
Glader, B.4
Ragni, M.5
Rasko, J.J.E.6
Ozelo, M.C.7
Hoots, K.8
Blatt, P.9
Konkle, B.10
Dake, M.11
Kaye, R.12
Razavi, M.13
Zajko, A.14
Zehnder, J.15
Rustagi, P.16
Nakai, H.17
Chew, A.18
Leonard, D.19
Wright, J.F.20
Lessard, R.R.21
Sommer, J.M.22
Tigges, M.23
Sabatino, D.24
Luk, A.25
Jiang, H.26
Mingozzi, F.27
Couto, L.28
Ertl, H.C.29
High, K.A.30
Kay, M.A.31
more..
-
7
-
-
84863970005
-
Induction of hepatocellular carcinoma by in vivo gene targeting
-
P.-R. Wang, M. Xu, S. Toffanin, Y. Li, J. M. Llovet, D. W. Russell, Induction of hepatocellular carcinoma by in vivo gene targeting. Proc. Natl. Acad. Sci. U.S.A. 109, 11264-11269 (2012).
-
(2012)
Proc. Natl. Acad. Sci. U.S.A.
, vol.109
, pp. 11264-11269
-
-
Wang, P.-R.1
Xu, M.2
Toffanin, S.3
Li, Y.4
Llovet, J.M.5
Russell, D.W.6
-
8
-
-
34547610960
-
AAV vector integration sites in mouse hepatocellular carcinoma
-
A. Donsante, D. G. Miller, Y. Li, C. Vogler, E. M. Brunt, D. W. Russell, M. S. Sands, AAV vector integration sites in mouse hepatocellular carcinoma. Science 317, 477 (2007).
-
(2007)
Science
, vol.317
, pp. 477
-
-
Donsante, A.1
Miller, D.G.2
Li, Y.3
Vogler, C.4
Brunt, E.M.5
Russell, D.W.6
Sands, M.S.7
-
9
-
-
84942905508
-
Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas
-
J.-C. Nault, S. Datta, S. Imbeaud, A. Franconi, M.Mallet, G. Couchy, E. Letouzé, C. Pilati, B. Verret, J.-F. Blanc, C. Balabaud, J. Calderaro, A. Laurent, M. Letexier, P. Bioulac-Sage, F. Calvo, J. Zucman-Rossi, Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas. Nat. Genet. 47, 1187-1193 (2015).
-
(2015)
Nat. Genet.
, vol.47
, pp. 1187-1193
-
-
Nault, J.-C.1
Datta, S.2
Imbeaud, S.3
Franconi, A.4
Mallet, M.5
Couchy, G.6
Letouzé, E.7
Pilati, C.8
Verret, B.9
Blanc, J.-F.10
Balabaud, C.11
Calderaro, J.12
Laurent, A.13
Letexier, M.14
Bioulac-Sage, P.15
Calvo, F.16
Zucman-Rossi, J.17
-
10
-
-
84864360337
-
AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. Hum
-
N. K. Paulk, L. M. Loza, M. J. Finegold, M. Grompe, AAV-mediated gene targeting is significantly enhanced by transient inhibition of nonhomologous end joining or the proteasome in vivo. Hum. Gene Ther. 23, 658-665 (2012).
-
(2012)
Gene Ther.
, vol.23
, pp. 658-665
-
-
Paulk, N.K.1
Loza, L.M.2
Finegold, M.J.3
Grompe, M.4
-
11
-
-
77950610272
-
Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo
-
N. K. Paulk, K. Wursthorn, Z. Wang, M. J. Finegold, M. A. Kay, M. Grompe, Adeno-associated virus gene repair corrects a mouse model of hereditary tyrosinemia in vivo. Hepatology 51, 1200-1208 (2010).
-
(2010)
Hepatology
, vol.51
, pp. 1200-1208
-
-
Paulk, N.K.1
Wursthorn, K.2
Wang, Z.3
Finegold, M.J.4
Kay, M.A.5
Grompe, M.6
-
12
-
-
33747046320
-
Gene targeting in vivo by adeno-associated virus vectors
-
D. G. Miller, P.-R. Wang, L. M. Petek, R. K. Hirata, M. S. Sands, D. W. Russell, Gene targeting in vivo by adeno-associated virus vectors. Nat. Biotechnol. 24, 1022-1026 (2006).
-
(2006)
Nat. Biotechnol.
, vol.24
, pp. 1022-1026
-
-
Miller, D.G.1
Wang, P.-R.2
Petek, L.M.3
Hirata, R.K.4
Sands, M.S.5
Russell, D.W.6
-
13
-
-
79960424171
-
In vivo genome editing restores haemostasis in a mouse model of haemophilia
-
H. Li, V. Haurigot, Y. Doyon, T. Li, S. Y.Wong, A. S. Bhagwat, N. Malani, X. M. Anguela, R. Sharma, L. Ivanciu, S. L. Murphy, J. D. Finn, F. R. Khazi, S. Zhou, D. E. Paschon, E. J. Rebar, F. D. Bushman, P. D. Gregory, M. C. Holmes, K. A. High, In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature 475, 217-221 (2011).
-
(2011)
Nature
, vol.475
, pp. 217-221
-
-
Li, H.1
Haurigot, V.2
Doyon, Y.3
Li, T.4
Wong, Y.S.5
Bhagwat, A.S.6
Malani, N.7
Anguela, X.M.8
Sharma, R.9
Ivanciu, L.10
Murphy, S.L.11
Finn, J.D.12
Khazi, F.R.13
Zhou, S.14
Paschon, D.E.15
Rebar, E.J.16
Bushman, F.D.17
Gregory, P.D.18
Holmes, M.C.19
High, K.A.20
more..
-
14
-
-
84902095353
-
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
-
H. Yin, W. Xue, S. Chen, R. L. Bogorad, E. Benedetti, M. Grompe, V. Koteliansky, P. A. Sharp, T. Jacks, D. G. Anderson, Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat. Biotechnol. 32, 551-553 (2014).
-
(2014)
Nat. Biotechnol.
, vol.32
, pp. 551-553
-
-
Yin, H.1
Xue, W.2
Chen, S.3
Bogorad, R.L.4
Benedetti, E.5
Grompe, M.6
Koteliansky, V.7
Sharp, P.A.8
Jacks, T.9
Anderson, D.G.10
-
15
-
-
84960882884
-
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
-
H. Yin, C.-Q. Song, J. R. Dorkin, L. J. Zhu, Y. Li, Q. Wu, A. Park, J. Yang, S. Suresh, A. Bizhanova, A. Gupta, M. F. Bolukbasi, S. Walsh, R. L. Bogorad, G. Gao, Z. Weng, Y. Dong, V. Koteliansky, S. A. Wolfe, R. Langer, W. Xue, D. G. Anderson, Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo. Nat. Biotechnol. 34, 328-333 (2016).
-
(2016)
Nat. Biotechnol.
, vol.34
, pp. 328-333
-
-
Yin, H.1
Song, C.-Q.2
Dorkin, J.R.3
Zhu, L.J.4
Li, Y.5
Wu, Q.6
Park, A.7
Yang, J.8
Suresh, S.9
Bizhanova, A.10
Gupta, A.11
Bolukbasi, M.F.12
Walsh, S.13
Bogorad, R.L.14
Gao, G.15
Weng, Z.16
Dong, Y.17
Koteliansky, V.18
Wolfe, S.A.19
Langer, R.20
Xue, W.21
Anderson, D.G.22
more..
-
16
-
-
0028146998
-
Self-induced correction of the genetic defect in tyrosinemia type
-
E. A. Kvittingen, H. Rootwelt, R. Berger, P. Brandtzaeg, Self-induced correction of the genetic defect in tyrosinemia type I. J. Clin. Invest. 94, 1657-1661 (1994).
-
(1994)
I. J. Clin. Invest.
, vol.94
, pp. 1657-1661
-
-
Kvittingen, E.A.1
Rootwelt, H.2
Berger, R.3
Brandtzaeg, P.4
-
17
-
-
0029851752
-
Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in Long-Evans cinnamon rats
-
Y. Yoshida, Y. Tokusashi, G. H. Lee, K. Ogawa, Intrahepatic transplantation of normal hepatocytes prevents Wilson's disease in Long-Evans cinnamon rats. Gastroenterology 111, 1654-1660 (1996).
-
(1996)
Gastroenterology
, vol.111
, pp. 1654-1660
-
-
Yoshida, Y.1
Tokusashi, Y.2
Lee, G.H.3
Ogawa, K.4
-
18
-
-
79955495937
-
Spontaneous hepatic repopulation in transgenic mice expressing mutant human a1-antitrypsin by wild-type donor hepatocytes
-
J. Ding, G. R. Yannam, N. Roy-Chowdhury, T. Hidvegi, H. Basma, S. I. Rennard, R. J. Wong, Y. Avsar, C. Guha, D. H. Perlmutter, I. J. Fox, J. Roy-Chowdhury, Spontaneous hepatic repopulation in transgenic mice expressing mutant human a1-antitrypsin by wild-type donor hepatocytes. J. Clin. Invest. 121, 1930-1934 (2011).
-
(2011)
J. Clin. Invest.
, vol.121
, pp. 1930-1934
-
-
Ding, J.1
Yannam, G.R.2
Roy-Chowdhury, N.3
Hidvegi, T.4
Basma, H.5
Rennard, S.I.6
Wong, R.J.7
Avsar, Y.8
Guha, C.9
Perlmutter, D.H.10
Fox, I.J.11
Roy-Chowdhury, J.12
-
19
-
-
0034466173
-
Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis
-
J. M. L. De Vree, R. Ottenhoff, P. J. Bosma, A. J. Smith, J. Aten, R. P. J. Oude Elferink, Correction of liver disease by hepatocyte transplantation in a mouse model of progressive familial intrahepatic cholestasis. Gastroenterology 119, 1720-1730 (2000).
-
(2000)
Gastroenterology
, vol.119
, pp. 1720-1730
-
-
De Vree, J.M.L.1
Ottenhoff, R.2
Bosma, P.J.3
Smith, A.J.4
Aten, J.5
Oude Elferink, R.P.J.6
-
20
-
-
0029875835
-
Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type
-
K. Overturf, M. Al-Dhalimy, R. Tanguay, M. Brantly, C.-N. Ou, M. Finegold, M. Grompe, Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 12, 266-273 (1996).
-
(1996)
I. Nat. Genet.
, vol.12
, pp. 266-273
-
-
Overturf, K.1
Al-Dhalimy, M.2
Tanguay, R.3
Brantly, M.4
Ou, C.-N.5
Finegold, M.6
Grompe, M.7
-
21
-
-
0035805603
-
Mechanistic inferences from the crystal structure of fumarylacetoacetate hydrolase with a bound phosphorusbased inhibitor
-
R. L. Bateman, P. Bhanumoorthy, J. F. Witte, R.W. McClard, M. Grompe, D. E. Timm, Mechanistic inferences from the crystal structure of fumarylacetoacetate hydrolase with a bound phosphorusbased inhibitor. J. Biol. Chem. 276, 15284-15291 (2001).
-
(2001)
J. Biol. Chem.
, vol.276
, pp. 15284-15291
-
-
Bateman, R.L.1
Bhanumoorthy, P.2
Witte, J.F.3
McClard, R.W.4
Grompe, M.5
Timm, D.E.6
-
22
-
-
33847726934
-
Slow-onset inhibition of fumarylacetoacetate hydrolase by phosphinate mimics of the tetrahedral intermediate: Kinetics, crystal structure and pharmacokinetics
-
R. L. Bateman, J. Ashworth, J. F. Witte, L.-J. Baker, P. Bhanumoorthy, D. E. Timm, T. D. Hurley, M. Grompe, R. W. McClard, Slow-onset inhibition of fumarylacetoacetate hydrolase by phosphinate mimics of the tetrahedral intermediate: Kinetics, crystal structure and pharmacokinetics.Biochem. J. 402, 251-260 (2007).
-
(2007)
Biochem. J.
, Issue.402
, pp. 251-260
-
-
Bateman, R.L.1
Ashworth, J.2
Witte, J.F.3
Baker, L.-J.4
Bhanumoorthy, P.5
Timm, D.E.6
Hurley, T.D.7
Grompe, M.8
McClard, R.W.9
-
23
-
-
84867017075
-
In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice
-
N. K. Paulk, K. Wursthorn, A. Haft, C. Pelz, G. Clarke, A. H. Newell, S. B. Olson, C. O. Harding, M. J. Finegold, R. L. Bateman, J. F. Witte, R. McClard, M. Grompe, In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice. Mol. Ther. 20, 1981-1987 (2012).
-
(2012)
Mol. Ther.
, vol.20
, pp. 1981-1987
-
-
Paulk, N.K.1
Wursthorn, K.2
Haft, A.3
Pelz, C.4
Clarke, G.5
Newell, A.H.6
Olson, S.B.7
Harding, C.O.8
Finegold, M.J.9
Bateman, R.L.10
Witte, J.F.11
McClard, R.12
Grompe, M.13
-
24
-
-
9844238697
-
Complete rescue of lethal albino c14CoS mice by null mutation of 4-hydroxyphenylpyruvate dioxygenase and induction of apoptosis of hepatocytes in these mice by in vivo retrieval of the tyrosine catabolic pathway
-
F. Endo, S. Kubo, H. Awata, K. Kiwaki, H. Katoh, Y. Kanegae, I. Saito, J.-i.Miyazaki, T. Yamamoto, C. Jakobs, S. Hattori, I. Matsuda, Complete rescue of lethal albino c14CoS mice by null mutation of 4-hydroxyphenylpyruvate dioxygenase and induction of apoptosis of hepatocytes in these mice by in vivo retrieval of the tyrosine catabolic pathway. J. Biol. Chem. 272, 24426-24432 (1997).
-
(1997)
J. Biol. Chem.
, Issue.272
, pp. 24426-24432
-
-
Endo, F.1
Kubo, S.2
Awata, H.3
Kiwaki, K.4
Katoh, H.5
Kanegae, Y.6
Saito, I.7
Miyazaki, J.-I.8
Yamamoto, T.9
Jakobs, C.10
Hattori, S.11
Matsuda, I.12
-
25
-
-
0032692849
-
In vivo suppressormutations correct a murine model of hereditary tyrosinemia type i
-
K.Manning, M. Al-Dhalimy, M. Finegold,M.Grompe, In vivo suppressormutations correct a murine model of hereditary tyrosinemia type I. Proc. Natl. Acad. Sci. U.S.A. 96, 11928-11933 (1999).
-
(1999)
Proc. Natl. Acad. Sci. U.S.A.
, Issue.96
, pp. 11928-11933
-
-
Manning, K.1
Al-Dhalimy, M.2
Finegold, M.3
Grompe, M.4
-
26
-
-
0026675589
-
Treatment of hereditary tyrosinaemia type i by inhibition of 4-hydroxyphenylpyruvate dioxygenase
-
S. Lindstedt, E. Holme, E. A. Lock, O. Hjalmarson, B. Strandvik, Treatment of hereditary tyrosinaemia type I by inhibition of 4-hydroxyphenylpyruvate dioxygenase. Lancet 340, 813-817 (1992).
-
(1992)
Lancet
, vol.340
, pp. 813-817
-
-
Lindstedt, S.1
Holme, E.2
Lock, E.A.3
Hjalmarson, O.4
Strandvik, B.5
-
27
-
-
84866148040
-
Effect of nitisinone (NTBC) treatment on the clinical course of hepatorenal tyrosinemia in Québec
-
J. Larochelle, F. Alvarez, J.-F. Bussières, I. Chevalier, L. Dallaire, J. Dubois, F. Faucher, D. Fenyves, P. Goodyer, A. Grenier, E. Holme, R. Laframboise, M. Lambert, S. Lindstedt, B. Maranda, S. Melançon, A. Merouani, J. Mitchell, G. Parizeault, L. Pelletier, V. Phan, P. Rinaldo, C. R. Scott, C. Scriver, G. A. Mitchell, Effect of nitisinone (NTBC) treatment on the clinical course of hepatorenal tyrosinemia in Québec. Mol. Genet. Metab. 107, 49-54 (2012).
-
(2012)
Mol. Genet. Metab.
, vol.107
, pp. 49-54
-
-
Larochelle, J.1
Alvarez, F.2
Bussières, J.-F.3
Chevalier, I.4
Dallaire, L.5
Dubois, J.6
Faucher, F.7
Fenyves, D.8
Goodyer, P.9
Grenier, A.10
Holme, E.11
Laframboise, R.12
Lambert, M.13
Lindstedt, S.14
Maranda, B.15
Melançon, S.16
Merouani, A.17
Mitchell, J.18
Parizeault, G.19
Pelletier, L.20
Phan, V.21
Rinaldo, P.22
Scott, C.R.23
Scriver, C.24
Mitchell, G.A.25
more..
-
28
-
-
84866973619
-
Ribosomal DNA integrating rAAV-rDNA vectors allowfor stable transgene expression
-
L. Lisowski, A. Lau, Z. Wang, Y. Zhang, F. Zhang, M. Grompe, M. A. Kay, Ribosomal DNA integrating rAAV-rDNA vectors allowfor stable transgene expression.Mol. Ther. 20, 1912-1923 (2012).
-
(2012)
Mol.Ther
, vol.20
, pp. 1912-1923
-
-
Lisowski, L.1
Lau, A.2
Wang, Z.3
Zhang, Y.4
Zhang, F.5
Grompe, M.6
Kay, M.A.7
-
29
-
-
84866930809
-
AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence
-
Z. Wang, L. Lisowski, M. J. Finegold, H. Nakai, M. A. Kay, M. Grompe, AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. Mol. Ther. 20, 1902-1911 (2012).
-
(2012)
Mol. Ther.
, vol.20
, pp. 1902-1911
-
-
Wang, Z.1
Lisowski, L.2
Finegold, M.J.3
Nakai, H.4
Kay, M.A.5
Grompe, M.6
-
30
-
-
84923138903
-
Promoterless gene targeting without nucleases ameliorates haemophilia B in mice
-
A. Barzel, N. K. Paulk, Y. Shi, Y. Huang, K. Chu, F. Zhang, P. N. Valdmanis, L. P. Spector, M. H. Porteus, K. M. Gaensler, M. A. Kay, Promoterless gene targeting without nucleases ameliorates haemophilia B in mice. Nature 517, 360-364 (2015).
-
(2015)
Nature
, vol.517
, pp. 360-364
-
-
Barzel, A.1
Paulk, N.K.2
Shi, Y.3
Huang, Y.4
Chu, K.5
Zhang, F.6
Valdmanis, P.N.7
Spector, L.P.8
Porteus, M.H.9
Gaensler, K.M.10
Kay, M.A.11
-
31
-
-
84922152390
-
Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
-
R. J. Chandler, M. C. LaFave, G. K. Varshney, N. S. Trivedi, N. Carrillo-Carrasco, J. S. Senac, W. Wu, V. Hoffmann, A. G. Elkahloun, S. M. Burgess, C. P. Venditti, Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy. J. Clin. Invest. 125, 870-880 (2015).
-
(2015)
J. Clin. Invest.
, vol.125
, pp. 870-880
-
-
Chandler, R.J.1
LaFave, M.C.2
Varshney, G.K.3
Trivedi, N.S.4
Carrillo-Carrasco, N.5
Senac, J.S.6
Wu, W.7
Hoffmann, V.8
Elkahloun, A.G.9
Burgess, S.M.10
Venditti, C.P.11
-
32
-
-
33745299723
-
Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
-
D. Grimm, K. L. Streetz, C. L. Jopling, T. A. Storm, K. Pandey, C. R. Davis, P. Marion, F. Salazar, M. A. Kay, Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441, 537-541 (2006).
-
(2006)
Nature
, vol.441
, pp. 537-541
-
-
Grimm, D.1
Streetz, K.L.2
Jopling, C.L.3
Storm, T.A.4
Pandey, K.5
Davis, C.R.6
Marion, P.7
Salazar, F.8
Kay, M.A.9
-
33
-
-
79953698717
-
TBI during BM and SCT: Review of the past, discussion of the present and consideration of future directions
-
C. E. Hill-Kayser, J. P. Plastaras, Z. Tochner, E. Glatstein, TBI during BM and SCT: Review of the past, discussion of the present and consideration of future directions. Bone Marrow Transplant. 46, 475-484 (2011).
-
(2011)
Bone Marrow Transplant.
, vol.46
, pp. 475-484
-
-
Hill-Kayser, C.E.1
Plastaras, J.P.2
Tochner, Z.3
Glatstein, E.4
-
34
-
-
34249789022
-
In vivo selection and chemoprotection after drug resistance gene therapy in a nonmyeloablative allogeneic transplantation setting in dogs
-
S. Gerull, B. C. Beard, L. J. Peterson, T. Neff, H.-P. Kiem, In vivo selection and chemoprotection after drug resistance gene therapy in a nonmyeloablative allogeneic transplantation setting in dogs. Hum. Gene Ther. 18, 451-456 (2007).
-
(2007)
Hum. Gene Ther.
, vol.18
, pp. 451-456
-
-
Gerull, S.1
Beard, B.C.2
Peterson, L.J.3
Neff, T.4
Kiem, H.-P.5
-
35
-
-
84907010362
-
Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients
-
J. E. Adair, S. K. Johnston, M. M. Mrugala, B. C. Beard, L. A. Guyman, A. L. Baldock, C. A. Bridge, A. Hawkins-Daarud, J. L. Gori, D. E. Born, L. F. Gonzalez-Cuyar, D. L. Silbergeld, R. C. Rockne, B. E. Storer, J. K. Rockhill, K. R. Swanson, H.-P. Kiem, Gene therapy enhances chemotherapy tolerance and efficacy in glioblastoma patients. J. Clin. Invest. 124, 4082-4092 (2014).
-
(2014)
J. Clin. Invest.
, vol.124
, pp. 4082-4092
-
-
Adair, J.E.1
Johnston, S.K.2
Mrugala, M.M.3
Beard, B.C.4
Guyman, L.A.5
Baldock, A.L.6
Bridge, C.A.7
Hawkins-Daarud, A.8
Gori, J.L.9
Born, D.E.10
Gonzalez-Cuyar, L.F.11
Silbergeld, D.L.12
Rockne, R.C.13
Storer, B.E.14
Rockhill, J.K.15
Swanson, K.R.16
Kiem, H.-P.17
-
36
-
-
0034235801
-
Therapeutic liver repopulation in a mouse model of hypercholesterolemia.Hum
-
C. Mitchell, A. Mignon, J. E. Guidotti, S. Besnard, M. Fabre, N. Duverger, D. Parlier, A. Tedgui, A. Kahn, H. Gilgenkrantz, Therapeutic liver repopulation in a mouse model of hypercholesterolemia. Hum. Mol. Genet. 9, 1597-1602 (2000).
-
(2000)
Mol. Genet.
, vol.9
, pp. 1597-1602
-
-
Mitchell, C.1
Mignon, A.2
Guidotti, J.E.3
Besnard, S.4
Fabre, M.5
Duverger, N.6
Parlier, D.7
Tedgui, A.8
Kahn, A.9
Gilgenkrantz, H.10
-
37
-
-
0031752146
-
Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes
-
A. Mignon, J. E. Guidotti, C. Mitchell, M. Fabre, A. Wernet, A. De La Coste, O. Soubrane, H. Gilgenkrantz, A. Kahn, Selective repopulation of normal mouse liver by Fas/CD95-resistant hepatocytes. Nat. Med. 4, 1185-1188 (1998).
-
(1998)
Nat. Med.
, vol.4
, pp. 1185-1188
-
-
Mignon, A.1
Guidotti, J.E.2
Mitchell, C.3
Fabre, M.4
Wernet, A.5
De La Coste, A.6
Soubrane, O.7
Gilgenkrantz, H.8
Kahn, A.9
-
38
-
-
0032797275
-
Cyclin B-dependent kinase and caspase-1 activation precedes mitochondrial dysfunction in fumarylacetoacetate-induced apoptosis
-
R. Jorquera, R. M. Tanguay, Cyclin B-dependent kinase and caspase-1 activation precedes mitochondrial dysfunction in fumarylacetoacetate-induced apoptosis. FASEB J. 13, 2284-2298 (1999).
-
(1999)
FASEB J.
, vol.13
, pp. 2284-2298
-
-
Jorquera, R.1
Tanguay, R.M.2
-
39
-
-
84942941923
-
Adeno-associated virus finds its disease
-
D. W. Russell, M. Grompe, Adeno-associated virus finds its disease. Nat. Genet. 47, 1104-1105 (2015).
-
(2015)
Nat. Genet.
, vol.47
, pp. 1104-1105
-
-
Russell, D.W.1
Grompe, M.2
-
40
-
-
0033758431
-
Nontransplant treatment of tyrosinemia
-
E. Holme, S. Lindstedt, Nontransplant treatment of tyrosinemia. Clin. Liver Dis. 4, 805-814 (2000).
-
(2000)
Clin. Liver Dis.
, vol.4
, pp. 805-814
-
-
Holme, E.1
Lindstedt, S.2
-
41
-
-
84855873678
-
Metabolic activation in drug-induced liver injury
-
L. Leung, A. S. Kalgutkar, R. S. Obach, Metabolic activation in drug-induced liver injury. Drug Metab. Rev. 44, 18-33 (2012).
-
(2012)
Drug Metab. Rev.
, vol.44
, pp. 18-33
-
-
Leung, L.1
Kalgutkar, A.S.2
Obach, R.S.3
-
42
-
-
84901768135
-
Genetic basis of drug-induced liver injury: Present and future
-
T. J. Urban, A. K. Daly, G. P. Aithal, Genetic basis of drug-induced liver injury: Present and future. Semin. Liver Dis. 34, 123-133 (2014).
-
(2014)
Semin. Liver Dis.
, vol.34
, pp. 123-133
-
-
Urban, T.J.1
Daly, A.K.2
Aithal, G.P.3
-
43
-
-
0027137419
-
Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice
-
M. Grompe, M. Al-Dhalimy, M. Finegold, C.-N. Ou, T. Burlingame, N. G. Kennaway, P. Soriano, Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes Dev. 7, 2298-2307 (1993).
-
(1993)
Genes Dev.
, vol.7
, pp. 2298-2307
-
-
Grompe, M.1
Al-Dhalimy, M.2
Finegold, M.3
Ou, C.-N.4
Burlingame, T.5
Kennaway, N.G.6
Soriano, P.7
-
44
-
-
0029082193
-
Pharmacological correction of neonatal lethal hepatic dysfunction in a murine model of hereditary tyrosinaemia type i
-
M. Grompe, S. Lindstedt, M. Al-Dhalimy, N. G. Kennaway, J. Papaconstantinou, C. A. Torres-Ramos, C. N. Ou, M. Finegold, Pharmacological correction of neonatal lethal hepatic dysfunction in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 10, 453-460 (1995).
-
(1995)
Nat. Genet.
, vol.10
, pp. 453-460
-
-
Grompe, M.1
Lindstedt, S.2
Al-Dhalimy, M.3
Kennaway, N.G.4
Papaconstantinou, J.5
Torres-Ramos, C.A.6
Ou, C.N.7
Finegold, M.8
-
45
-
-
84890985901
-
An optimized microRNA backbone for effective singlecopy RNAi
-
C. Fellmann, T. Hoffmann, V. Sridhar, B. Hopfgartner, M. Muhar, M. Roth, D. Y. Lai, I. A. M. Barbosa, J. S. Kwon, Y. Guan, N. Sinha, J. Zuber, An optimized microRNA backbone for effective singlecopy RNAi. Cell Rep. 5, 1704-1713 (2013).
-
(2013)
Cell Rep.
, vol.5
, pp. 1704-1713
-
-
Fellmann, C.1
Hoffmann, T.2
Sridhar, V.3
Hopfgartner, B.4
Muhar, M.5
Roth, M.6
Lai, D.Y.7
Barbosa, I.A.M.8
Kwon, J.S.9
Guan, Y.10
Sinha, N.11
Zuber, J.12
-
46
-
-
0038150506
-
Helper virus-free, optically controllable, and twoplasmid- based production of adeno-associated virus vectors of serotypes 1 to 6
-
D. Grimm, M. A. Kay, J. A. Kleinschmidt, Helper virus-free, optically controllable, and twoplasmid- based production of adeno-associated virus vectors of serotypes 1 to 6. Mol. Ther. 7, 839-850 (2003).
-
(2003)
Mol. Ther.
, vol.7
, pp. 839-850
-
-
Grimm, D.1
Kay, M.A.2
Kleinschmidt, J.A.3
-
47
-
-
0036968715
-
Kinetics of liver repopulation after bone marrow transplantation
-
X. Wang, E. Montini, M. Al-Dhalimy, E. Lagasse, M. Finegold, M. Grompe, Kinetics of liver repopulation after bone marrow transplantation. Am. J. Pathol. 161, 565-574 (2002).
-
(2002)
Am. J. Pathol.
, vol.161
, pp. 565-574
-
-
Wang, X.1
Montini, E.2
Al-Dhalimy, M.3
Lagasse, E.4
Finegold, M.5
Grompe, M.6
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