-
1
-
-
0022549920
-
A receptor-mediated pathway for cholesterol homeostasis
-
Brown MS, Goldstein JL. A receptor-mediated pathway for cholesterol homeostasis. Science 1986;232:34-47.
-
(1986)
Science
, vol.232
, pp. 34-47
-
-
Brown, M.S.1
Goldstein, J.L.2
-
2
-
-
0002230202
-
Familial hypercholesterolemia, lipoprotein and lipid metabolism disorders
-
In: Scriver CR, Beaudet AL, Sly WS, et al., eds. New York: McGraw-Hill
-
Goldstein JL, Brown JC. Familial hypercholesterolemia, lipoprotein and lipid metabolism disorders. In: Scriver CR, Beaudet AL, Sly WS, et al., eds. Metabolic Basis of Inherited Disease. Volume II. New York: McGraw-Hill, 1989:1215-1250.
-
(1989)
Metabolic Basis of Inherited Disease
, vol.2
, pp. 1215-1250
-
-
Goldstein, J.L.1
Brown, J.C.2
-
3
-
-
0041743167
-
Monogenic hypercholesterolemia: New insights in pathogenesis and treatment
-
Rader DJ, Cohen J, Hobbs HH. Monogenic hypercholesterolemia: new insights in pathogenesis and treatment. J Clin Invest 2003;111:1795-1803.
-
(2003)
J Clin Invest
, vol.111
, pp. 1795-1803
-
-
Rader, D.J.1
Cohen, J.2
Hobbs, H.H.3
-
4
-
-
0028292602
-
Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia
-
Grossman M, Raper SE, Kozarsky K, et al. Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia. Nat Genet 1994;6:335-341.
-
(1994)
Nat Genet
, vol.6
, pp. 335-341
-
-
Grossman, M.1
Raper, S.E.2
Kozarsky, K.3
-
5
-
-
84911383748
-
Long-term safety and efficacy of factor IX gene therapy in hemophilia B
-
Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. New Engl J Med 2014;371: 1994-2004.
-
(2014)
New Engl J Med
, vol.371
, pp. 1994-2004
-
-
Nathwani, A.C.1
Reiss, U.M.2
Tuddenham, E.G.3
-
6
-
-
84855161388
-
Adenovirus-associated virus vectormediated gene transfer in hemophilia B
-
Nathwani AC, Tuddenham EG, Rangarajan S, et al. Adenovirus-associated virus vectormediated gene transfer in hemophilia B. N Engl J Med 2011;365:2357-2365.
-
(2011)
N Engl J Med
, vol.365
, pp. 2357-2365
-
-
Nathwani, A.C.1
Tuddenham, E.G.2
Rangarajan, S.3
-
7
-
-
84872773410
-
Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia
-
Kassim SH, Li H, Bell P, et al. Adeno-associated virus serotype 8 gene therapy leads to significant lowering of plasma cholesterol levels in humanized mouse models of homozygous and heterozygous familial hypercholesterolemia. Hum Gene Ther 2013;24:19-26.
-
(2013)
Hum Gene Ther
, vol.24
, pp. 19-26
-
-
Kassim, S.H.1
Li, H.2
Bell, P.3
-
8
-
-
78149421771
-
Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis
-
Kassim SH, Li H, Vandenberghe LH, et al. Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis. PLoS One 2010;5:e13424.
-
(2010)
PLoS One
, vol.5
, pp. e13424
-
-
Kassim, S.H.1
Li, H.2
Vandenberghe, L.H.3
-
9
-
-
0024231872
-
Genetically determined hypercholesterolemia in a rhesus monkey family due to a deficiency of the LDL receptor
-
Scanu A, Khalil A, Neven L, et al. Genetically determined hypercholesterolemia in a rhesus monkey family due to a deficiency of the LDL receptor. J Lipid Res 1988;29:1671-1681.
-
(1988)
J Lipid Res
, vol.29
, pp. 1671-1681
-
-
Scanu, A.1
Khalil, A.2
Neven, L.3
-
10
-
-
0025253115
-
Familial hypercholesterolemia in a rhesus monkey pedigree: Molecular basis of low density lipoprotein receptor deficiency
-
Hummel M, Li Z, Pfaffinger D, et al. Familial hypercholesterolemia in a rhesus monkey pedigree: Molecular basis of low density lipoprotein receptor deficiency. Proc Natl Acad Sci U S A 1990;87:3122-3126.
-
(1990)
Proc Natl Acad Sci U S A
, vol.87
, pp. 3122-3126
-
-
Hummel, M.1
Li, Z.2
Pfaffinger, D.3
-
11
-
-
84871945617
-
Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: A single-arm, open-label, Phase 3 study
-
Cuchel M, Meagher EA, du Toit Theron H, et al. Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, Phase 3 study. Lancet 2013;381:40-46.
-
(2013)
Lancet
, vol.381
, pp. 40-46
-
-
Cuchel, M.1
Meagher, E.A.2
Du Toit Theron, H.3
-
12
-
-
77950858243
-
Pathology of nonalcoholic fatty liver disease
-
Brunt EM. Pathology of nonalcoholic fatty liver disease. Nat Rev Gastroenterol Hepatol 2010;7: 195-203.
-
(2010)
Nat Rev Gastroenterol Hepatol
, vol.7
, pp. 195-203
-
-
Brunt, E.M.1
-
13
-
-
77958104835
-
Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale
-
Lock M, Alvira M, Vandenberghe LH, et al. Rapid, simple, and versatile manufacturing of recombinant adeno-associated viral vectors at scale. Hum Gene Ther 2010;21:1259-1271.
-
(2010)
Hum Gene Ther
, vol.21
, pp. 1259-1271
-
-
Lock, M.1
Alvira, M.2
Vandenberghe, L.H.3
-
14
-
-
58849086758
-
Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses
-
Calcedo R, Vandenberghe LH, Gao G, et al. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 2009;199:381-390.
-
(2009)
J Infect Dis
, vol.199
, pp. 381-390
-
-
Calcedo, R.1
Vandenberghe, L.H.2
Gao, G.3
-
15
-
-
74149088098
-
The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques
-
Wang L, Calcedo R, Wang H, et al. The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques. Mol Ther 2010;18: 126-134.
-
(2010)
Mol Ther
, vol.18
, pp. 126-134
-
-
Wang, L.1
Calcedo, R.2
Wang, H.3
-
16
-
-
62749200441
-
Host immune responses to chronic adenovirus infections in human and nonhuman primates
-
Calcedo R, Vandenberghe LH, Roy S, et al. Host immune responses to chronic adenovirus infections in human and nonhuman primates. J Virol 2009;83:2623-2631.
-
(2009)
J Virol
, vol.83
, pp. 2623-2631
-
-
Calcedo, R.1
Vandenberghe, L.H.2
Roy, S.3
-
17
-
-
84901447384
-
Biodistribution of AAV8 vectors expressing human lowdensity lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia
-
Chen S-J, Sanmiguel J, Lock M, et al. Biodistribution of AAV8 vectors expressing human lowdensity lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia. Hum Gene Ther Clin Dev 2013;24:154-160.
-
(2013)
Hum Gene Ther Clin Dev
, vol.24
, pp. 154-160
-
-
Chen, S.-J.1
Sanmiguel, J.2
Lock, M.3
-
18
-
-
70349110562
-
Adenoassociated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses
-
Gao G, Wang Q, Calcedo R, et al. Adenoassociated virus-mediated gene transfer to nonhuman primate liver can elicit destructive transgene-specific T cell responses. Hum Gene Ther 2009;20:930-942.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 930-942
-
-
Gao, G.1
Wang, Q.2
Calcedo, R.3
-
19
-
-
85016128093
-
Nonclinical pharmacology/toxicology study of AAV8. TBG.mLDLR and AAV8.TBG.hLDLR in a mouse model of homozygous familial hypercholesterolemia. TBG.mLDLR
-
Greig JA, Limberis MP, Bell P, et al. Nonclinical pharmacology/toxicology study of AAV8.TBG.mLDLR and AAV8.TBG.hLDLR in a mouse model of homozygous familial hypercholesterolemia. Hum Gene Ther Clin Dev 2017; 28:10.1089/humc.2017.007
-
(2017)
Hum Gene Ther Clin Dev
, pp. 28
-
-
Greig, J.A.1
Limberis, M.P.2
Bell, P.3
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