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. 2009;50(2):107-11.
doi: 10.1093/ilar.50.2.107.

Gene therapy in large animal models of human genetic diseases. Introduction

John H Wolfe  1
Affiliations

Gene therapy in large animal models of human genetic diseases. Introduction

John H Wolfe. ILAR J. 2009.
No abstract available

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References

Bibliography

General References for Animal Models of Human Genetic Diseases
    1. Online Mendelian Inheritance in Animals (OMIA) Reprogen, University of Sydney Faculty of Veterinary Science, and Australian National Genomic Information Service (ANGIS) [accessed January 26, 2009]. Available online ( http://omia.angis.org.au/)
    1. Scriver CR, Beaudet AL, Valle D, Sly WS, Childs B, Kinzler KW, Vogelstein B. Metabolic and Molecular Bases of Inherited Disease. 8. New York: McGraw Hill; 2001. [Chapters on specific genetic diseases have sections on animal models if available; new edition due out soon.]
    1. Patterson DF, Haskins ME, Jezyk PF, Giger U, Meyers-Wallen VN, Aguirre G, Fyfe JC, Wolfe JH. Research on genetic diseases: Reciprocal benefits to animals and man. JAVMA. 1988;193:1131–1144. - PubMed
A Selection of Reviews on Gene Therapy for Human Genetic Diseases in Large Animals
    1. Allocca M, Tessitore A, Cotugno G, Auricchio A. AAV-mediated gene transfer for retinal diseases. Expert Opin Biol Ther. 2006;6:1279–1294. - PubMed
    1. Bauer TR, Jr, Adler RL, Hickstein DD. Potential large animal models for gene therapy of human genetic diseases of immune and blood cell systems. ILAR J. 2009;50:168–186. - PMC - PubMed
    1. Casal M, Haskins M. Large animal models and gene therapy. Eur J Hum Genet. 2006;14:266–272. - PubMed
    1. Ellinwood NM, Vite CH, Haskins ME. Gene therapy for lysosomal storage diseases: The lessons and promise of animal models. J Gene Med. 2004;6:481–506. - PubMed
    1. Gagliardi C, Bunnell BA. Large animal models of neurological disorders for gene therapy. ILAR J. 2009;50:128–143. - PMC - PubMed
References for Specific Models or Processes
    1. 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. Gene therapy restores vision in a canine model of childhood blindness. Nat Genet. 2001;28:92–95. - PubMed
    1. Bauer TR, Jr, Gu YC, Creevy KE, Tuschong LM, Embree L, Holland SM, Sokolic RA, Hickstein DD. Leukocyte adhesion deficiency in children and Irish setter dogs. Pediatr Res. 2004;55:363–367. - PubMed
    1. Kaplan J. Leber congenital amaurosis: From darkness to spotlight. Ophthalmic Genet. 2008;29:92–98. - PubMed
    1. Murphy SL, High KA. Gene therapy for hæmophilia. Br J Hæmatol. 2008;140:479–487. - PMC - PubMed
    1. Nienhuis AW, Dunbar CE, Sorrentino BP. Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther. 2006;13:1031–1049. - PubMed
Transgenic Large Animal Models of Human Disease
    1. 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, Hanfl 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. Disruption of the CFTR gene produces a model of cystic fibrosis in newborn pigs. Science. 2008;321:1837–1841. - PMC - PubMed
    1. 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. Toward a transgenic model of Huntington’s disease in a non-human primate. Nature. 2008;453:921–924. - PMC - PubMed
General Reviews on the Most Commonly Used Vectors
    1. Berges BK, Wolfe JH, Fraser NW. Transduction of brain by herpes simplex virus vectors. Mol Ther. 2007;15:20–29. - PubMed
    1. Cockrell AS, Kafri T. Gene delivery by lentivirus vectors. Mol Biotechnol. 2007;36:184–204. - PubMed
    1. McConnell MJ, Imperiale MJ. Biology of adenovirus and its use as a vector for gene therapy. Hum Gene Ther. 2004;15:1022–1033. - PubMed
    1. Oehmig A, Fraefel C, Breakefield XO. Update on herpesvirus amplicon vectors. Mol Ther. 2004;10:630–643. - PubMed
    1. Philpott NJ, Thrasher AJ. Use of nonintegrating lentiviral vectors for gene therapy. Hum Gene Ther. 2007;18:483–489. - PubMed

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