-
1
-
-
85036786762
-
-
Online Mendelian Inheritance in Animals (OMIA). Reprogen, University of Sydney Faculty of Veterinary Science, and Australian National Genomic Information Service (ANGIS). Available online (http://omia.angis.org. au/), accessed January 26, 2009.
-
Online Mendelian Inheritance in Animals (OMIA). Reprogen, University of Sydney Faculty of Veterinary Science, and Australian National Genomic Information Service (ANGIS). Available online (http://omia.angis.org. au/), accessed January 26, 2009.
-
-
-
-
2
-
-
85036794381
-
-
Scriver CR, Beaudet AL, Valle D, Sly WS, Childs B, Kinzler KW, Vogelstein B. 2001. Metabolic and Molecular Bases of Inherited Disease, 8th ed. New York: McGraw Hill. [Chapters on specific genetic diseases have sections on animal models if available; new edition due out soon.]
-
Scriver CR, Beaudet AL, Valle D, Sly WS, Childs B, Kinzler KW, Vogelstein B. 2001. Metabolic and Molecular Bases of Inherited Disease, 8th ed. New York: McGraw Hill. [Chapters on specific genetic diseases have sections on animal models if available; new edition due out soon.]
-
-
-
-
3
-
-
0024118185
-
Research on genetic diseases: Reciprocal benefits to animals and man
-
Patterson DF, Haskins ME, Jezyk PF, Giger U, Meyers-Wallen VN, Aguirre G, Fyfe JC, Wolfe JH. 1988. Research on genetic diseases: Reciprocal benefits to animals and man. JAVMA 193:1131-1144.
-
(1988)
JAVMA
, vol.193
, pp. 1131-1144
-
-
Patterson, D.F.1
Haskins, M.E.2
Jezyk, P.F.3
Giger, U.4
Meyers-Wallen, V.N.5
Aguirre, G.6
Fyfe, J.C.7
Wolfe, J.H.8
-
5
-
-
66149117418
-
Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems
-
Bauer TR Jr, Adler RL, Hickstein DD. 2009. Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems. ILAR J 50:168-186.
-
(2009)
ILAR J
, vol.50
, pp. 168-186
-
-
Bauer Jr, T.R.1
Adler, R.L.2
Hickstein, D.D.3
-
6
-
-
33645145491
-
Large animal models and gene therapy
-
Casal M, Haskins M. 2006. Large animal models and gene therapy. Eur J Hum Genet 14:266-272.
-
(2006)
Eur J Hum Genet
, vol.14
, pp. 266-272
-
-
Casal, M.1
Haskins, M.2
-
7
-
-
4544254425
-
Gene therapy for lysosomal storage diseases: The lessons and promise of animal models
-
Ellinwood NM, Vite CH, Haskins ME. 2004. Gene therapy for lysosomal storage diseases: The lessons and promise of animal models. J Gene Med 6:481-506.
-
(2004)
J Gene Med
, vol.6
, pp. 481-506
-
-
Ellinwood, N.M.1
Vite, C.H.2
Haskins, M.E.3
-
8
-
-
66149100946
-
Large animal models of neurological disorders for gene therapy
-
Gagliardi C, Bunnell BA. 2009. Large animal models of neurological disorders for gene therapy. ILAR J 50:128-143.
-
(2009)
ILAR J
, vol.50
, pp. 128-143
-
-
Gagliardi, C.1
Bunnell, B.A.2
-
9
-
-
44049090692
-
AAV-mediated gene transfer for the treatment of hemophilia B: Problems and prospects
-
Hasbrouck NC, High KA. 2008. AAV-mediated gene transfer for the treatment of hemophilia B: Problems and prospects. Gene Ther 15:870-875.
-
(2008)
Gene Ther
, vol.15
, pp. 870-875
-
-
Hasbrouck, N.C.1
High, K.A.2
-
10
-
-
66149133657
-
Gene therapy for lysosomal storage diseases (LSDs) in large animal models
-
Haskins M. 2009. Gene therapy for lysosomal storage diseases (LSDs) in large animal models. ILAR J 50:112-121.
-
(2009)
ILAR J
, vol.50
, pp. 112-121
-
-
Haskins, M.1
-
11
-
-
4444313651
-
Stem cell gene transfer: Efficacy and safety in large animal studies
-
Horn PA, Morris JC, Nef T, Kiem HP. 2004. Stem cell gene transfer: Efficacy and safety in large animal studies. Mol Ther 10:417-431.
-
(2004)
Mol Ther
, vol.10
, pp. 417-431
-
-
Horn, P.A.1
Morris, J.C.2
Nef, T.3
Kiem, H.P.4
-
12
-
-
66149144345
-
Gene therapy for inherited metabolic disorders in companion animals
-
Koeberl DD, Pinto C, Brown T, Chen YT. 2009. Gene therapy for inherited metabolic disorders in companion animals. ILAR J 50:122-127.
-
(2009)
ILAR J
, vol.50
, pp. 122-127
-
-
Koeberl, D.D.1
Pinto, C.2
Brown, T.3
Chen, Y.T.4
-
13
-
-
33845365042
-
Genetics, biology and clinical management of myeloid cell primary immune deficiencies: Chronic granulomatous disease and leukocyte adhesion deficiency
-
Malech HL, Hickstein DD. 2007. Genetics, biology and clinical management of myeloid cell primary immune deficiencies: Chronic granulomatous disease and leukocyte adhesion deficiency. Curr Opin Hematol 14:29-36.
-
(2007)
Curr Opin Hematol
, vol.14
, pp. 29-36
-
-
Malech, H.L.1
Hickstein, D.D.2
-
14
-
-
66149110999
-
Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von Willebrand disease, and factor VII deficiency
-
Nichols TC, Dillow AM, Franck HWG, Merricks EP, Raymer RA, Bellinger DA, Arruda VR, High KA. 2009. Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von Willebrand disease, and factor VII deficiency. ILAR J 50:144-167.
-
(2009)
ILAR J
, vol.50
, pp. 144-167
-
-
Nichols, T.C.1
Dillow, A.M.2
Franck, H.W.G.3
Merricks, E.P.4
Raymer, R.A.5
Bellinger, D.A.6
Arruda, V.R.7
High, K.A.8
-
15
-
-
33745178333
-
Gene therapy for inherited diseases of the central nervous system
-
Lynch D, ed, New York: Marcel Dekker. p
-
Pierson TM, Wolfe JH. 2005. Gene therapy for inherited diseases of the central nervous system. In: Lynch D, ed. Neurogenetics: Scientific and Clinical Advances. New York: Marcel Dekker. p 43-85.
-
(2005)
Neurogenetics: Scientific and Clinical Advances
, pp. 43-85
-
-
Pierson, T.M.1
Wolfe, J.H.2
-
16
-
-
2342452010
-
Preclinical animal models for hemophilia gene therapy: Predictive value and limitations
-
Rawle FE, Lillicrap D. 2004. Preclinical animal models for hemophilia gene therapy: Predictive value and limitations. Semin Thromb Hemost 30:205-213.
-
(2004)
Semin Thromb Hemost
, vol.30
, pp. 205-213
-
-
Rawle, F.E.1
Lillicrap, D.2
-
17
-
-
66149083628
-
Gene therapy in large animal models of human cardiovascular genetic disease
-
Sleeper MM, Bish LT, Sweeney HL. 2009. Gene therapy in large animal models of human cardiovascular genetic disease. ILAR J 50:199-205.
-
(2009)
ILAR J
, vol.50
, pp. 199-205
-
-
Sleeper, M.M.1
Bish, L.T.2
Sweeney, H.L.3
-
18
-
-
66149105139
-
AAV-mediated gene therapy for retinal disorders in large animal models
-
Stieger K, Lhériteau E, Moullier P, Rolling F. 2009. AAV-mediated gene therapy for retinal disorders in large animal models. ILAR J 50:206-224.
-
(2009)
ILAR J
, vol.50
, pp. 206-224
-
-
Stieger, K.1
Lhériteau, E.2
Moullier, P.3
Rolling, F.4
-
19
-
-
66149123674
-
Gene therapy in large animal models of muscular dystrophy
-
Wang Z, Chamberlain JS, Tapscott SJ, Storb R. 2009. Gene therapy in large animal models of muscular dystrophy. ILAR J 50:187-198.
-
(2009)
ILAR J
, vol.50
, pp. 187-198
-
-
Wang, Z.1
Chamberlain, J.S.2
Tapscott, S.J.3
Storb, R.4
-
20
-
-
0035032662
-
Gene therapy restores vision in a canine model of childhood blindness
-
Acland GM, Aguirre GD, Ray J, Zhang Q, Aleman TS, Cideciyan AV, Pearce-Kelling SE, Anand V, Zeng Y, Maguire AM, Jacobson SG, Hauswirth WW, Bennett J. 2001. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet 28:92-95.
-
(2001)
Nat Genet
, vol.28
, pp. 92-95
-
-
Acland, G.M.1
Aguirre, G.D.2
Ray, J.3
Zhang, Q.4
Aleman, T.S.5
Cideciyan, A.V.6
Pearce-Kelling, S.E.7
Anand, V.8
Zeng, Y.9
Maguire, A.M.10
Jacobson, S.G.11
Hauswirth, W.W.12
Bennett, J.13
-
21
-
-
1242274615
-
Leukocyte adhesion deficiency in children and Irish setter dogs
-
Bauer TR Jr, Gu YC, Creevy KE, Tuschong LM, Embree L, Holland SM, Sokolic RA, Hickstein DD. 2004. Leukocyte adhesion deficiency in children and Irish setter dogs. Pediatr Res 55:363-367.
-
(2004)
Pediatr Res
, vol.55
, pp. 363-367
-
-
Bauer Jr, T.R.1
Gu, Y.C.2
Creevy, K.E.3
Tuschong, L.M.4
Embree, L.5
Holland, S.M.6
Sokolic, R.A.7
Hickstein, D.D.8
-
22
-
-
51049084854
-
Leber congenital amaurosis: From darkness to spotlight
-
Kaplan J. 2008. Leber congenital amaurosis: From darkness to spotlight. Ophthalmic Genet 29:92-98.
-
(2008)
Ophthalmic Genet
, vol.29
, pp. 92-98
-
-
Kaplan, J.1
-
23
-
-
38949118647
-
Gene therapy for hæmophilia
-
Murphy SL, High KA. 2008. Gene therapy for hæmophilia. Br J Hæmatol 140:479-487.
-
(2008)
Br J Hæmatol
, vol.140
, pp. 479-487
-
-
Murphy, S.L.1
High, K.A.2
-
24
-
-
33646887376
-
Genotoxicity of retroviral integration in hematopoietic cells
-
Nienhuis AW, Dunbar CE, Sorrentino BP. 2006. Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 13:1031-1049.
-
(2006)
Mol Ther
, vol.13
, pp. 1031-1049
-
-
Nienhuis, A.W.1
Dunbar, C.E.2
Sorrentino, B.P.3
-
25
-
-
40349091535
-
In vivo models of hæmophilia: Status on current knowledge of clinical phenotypes and therapeutic interventions
-
Øvlisen K, Kristensen AT, Tranholm M. 2008. In vivo models of hæmophilia: Status on current knowledge of clinical phenotypes and therapeutic interventions. Hæmophilia 14:248-259.
-
(2008)
Hæmophilia
, vol.14
, pp. 248-259
-
-
Øvlisen, K.1
Kristensen, A.T.2
Tranholm, M.3
-
26
-
-
14844284635
-
Effective gene therapy for an inherited diffuse CNS disease in a large animal model
-
Vite CH, Niogi SN, McGowan JC, Passini MA, Drobatz KJ, Haskins ME, Wolfe JH. 2005. Effective gene therapy for an inherited diffuse CNS disease in a large animal model. Ann Neurol 57:355-364.
-
(2005)
Ann Neurol
, vol.57
, pp. 355-364
-
-
Vite, C.H.1
Niogi, S.N.2
McGowan, J.C.3
Passini, M.A.4
Drobatz, K.J.5
Haskins, M.E.6
Wolfe, J.H.7
-
27
-
-
39649091780
-
Molecular imaging of gene therapy for neurogenetic diseases
-
Kaplitt MG, During MG, eds, San Diego: Academic Press. p
-
Wolfe JH, Acton P, Poptani H, Vite CH. 2006. Molecular imaging of gene therapy for neurogenetic diseases. In: Kaplitt MG, During MG, eds. Gene Therapy in the Central Nervous System: From Bench to Bedside. San Diego: Academic Press. p 335-350.
-
(2006)
Gene Therapy in the Central Nervous System: From Bench to Bedside
, pp. 335-350
-
-
Wolfe, J.H.1
Acton, P.2
Poptani, H.3
Vite, C.H.4
-
28
-
-
52949154301
-
Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs
-
Rogers CS, Stoltz DA, Meyerholz DK, Ostedgaard LS, Rokhlina T, Taft PJ, Rogan MP, Pezzulo AA, Karp PH, Itani OA, Kabel AC, Wohlford-Lenane CL, Davis GJ, Hanfland RA, Smith TL, Samuel M, Wax D, Murphy CN, Rieke A, Whitworth K, Uc A, Starner TD, Brogden KA, Shilyansky J, McCray PB Jr, Zabner J, Prather RS, Welsh MJ. 2008. Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs. Science 321:1837-1841.
-
(2008)
Science
, vol.321
, pp. 1837-1841
-
-
Rogers, C.S.1
Stoltz, D.A.2
Meyerholz, D.K.3
Ostedgaard, L.S.4
Rokhlina, T.5
Taft, P.J.6
Rogan, M.P.7
Pezzulo, A.A.8
Karp, P.H.9
Itani, O.A.10
Kabel, A.C.11
Wohlford-Lenane, C.L.12
Davis, G.J.13
Hanfland, R.A.14
Smith, T.L.15
Samuel, M.16
Wax, D.17
Murphy, C.N.18
Rieke, A.19
Whitworth, K.20
Uc, A.21
Starner, T.D.22
Brogden, K.A.23
Shilyansky, J.24
McCray Jr, P.B.25
Zabner, J.26
Prather, R.S.27
Welsh, M.J.28
more..
-
29
-
-
45149105779
-
Toward a transgenic model of Huntington's disease in a non-human primate
-
Yang SH, Cheng PH, Banta H, Piotrowska-Nitsche K, Yang JJ, Cheng EC, Snyder B, Larkin K, Liu J, Orkin J, Fang ZH, Smith Y, Bachevalier J, Zola SM, Li SH, Li XJ, Chan AW. 2008. Toward a transgenic model of Huntington's disease in a non-human primate. Nature 453:921-924.
-
(2008)
Nature
, vol.453
, pp. 921-924
-
-
Yang, S.H.1
Cheng, P.H.2
Banta, H.3
Piotrowska-Nitsche, K.4
Yang, J.J.5
Cheng, E.C.6
Snyder, B.7
Larkin, K.8
Liu, J.9
Orkin, J.10
Fang, Z.H.11
Smith, Y.12
Bachevalier, J.13
Zola, S.M.14
Li, S.H.15
Li, X.J.16
Chan, A.W.17
-
30
-
-
33846015516
-
Transduction of brain by herpes simplex virus vectors
-
Berges BK, Wolfe JH, Fraser NW. 2007. Transduction of brain by herpes simplex virus vectors. Mol Ther 15:20-29.
-
(2007)
Mol Ther
, vol.15
, pp. 20-29
-
-
Berges, B.K.1
Wolfe, J.H.2
Fraser, N.W.3
-
31
-
-
36249015420
-
Gene delivery by lentivirus vectors
-
Cockrell AS, Kafri T. 2007. Gene delivery by lentivirus vectors. Mol Biotechnol 36:184-204.
-
(2007)
Mol Biotechnol
, vol.36
, pp. 184-204
-
-
Cockrell, A.S.1
Kafri, T.2
-
32
-
-
9244257887
-
Biology of adenovirus and its use as a vector for gene therapy
-
McConnell MJ, Imperiale MJ. 2004. Biology of adenovirus and its use as a vector for gene therapy. Hum Gene Ther 15:1022-1033.
-
(2004)
Hum Gene Ther
, vol.15
, pp. 1022-1033
-
-
McConnell, M.J.1
Imperiale, M.J.2
-
34
-
-
34447134838
-
Use of nonintegrating lentiviral vectors for gene therapy
-
Philpott NJ, Thrasher AJ. 2007. Use of nonintegrating lentiviral vectors for gene therapy. Hum Gene Ther 18:483-489.
-
(2007)
Hum Gene Ther
, vol.18
, pp. 483-489
-
-
Philpott, N.J.1
Thrasher, A.J.2
-
35
-
-
45749138773
-
Recombinant adeno-associated virus transduction and integration
-
Schultz BR, Chamberlain JS. 2008. Recombinant adeno-associated virus transduction and integration. Mol Ther 16:1189-1199.
-
(2008)
Mol Ther
, vol.16
, pp. 1189-1199
-
-
Schultz, B.R.1
Chamberlain, J.S.2
-
36
-
-
33746874152
-
Adeno-associated virus serotypes: Vector toolkit for human gene therapy
-
Wu Z, Asokan A, Samulski RJ. 2006. Adeno-associated virus serotypes: Vector toolkit for human gene therapy Mol Ther 14:316-327.
-
(2006)
Mol Ther
, vol.14
, pp. 316-327
-
-
Wu, Z.1
Asokan, A.2
Samulski, R.J.3
|