Cerebellar allografts survive and transiently alleviate ataxia in a transgenic model of spinocerebellar ataxia type-1

Experimental Neurology

Volumn 158, Issue 2, 1999, Pages 301-311

  Kaemmerer, William F ^a   Low, Walter C ^b  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF MINNESOTA   (United States)

Author keywords

CAG trinucleotide repeats; Cerebellar allografts; Neural transplants; Polyglutamine proteins; Spinocerebellar ataxia; Transgenic models

Indexed keywords

ALLOGRAFT; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BEHAVIOR; CEREBELLAR ATAXIA; CONTROLLED STUDY; DEGENERATIVE DISEASE; GRAFT SURVIVAL; HUNTINGTON CHOREA; MOTOR PERFORMANCE; MOUSE; MUSCLE ATROPHY; NONHUMAN; PRIORITY JOURNAL; PURKINJE CELL; TRANSGENIC MOUSE;

EID: 0032854754     PISSN: 00144886     EISSN: None     Source Type: Journal    
DOI: 10.1006/exnr.1999.7099     Document Type: Article

References (33)

1. Abdullah A., Trifiro M. A., Panet-Raymond V., Alvarado C., De Tourreil S., Frankel D., Schipper H. M., Pinsky L. Spinobulbar muscular atrophy: Polyglutamine expanded androgen receptor is proteolytically resistant in vitro and processed abnormally in transfected cells. Hum. Mol. Genet. 7:1998;379-384.
2. Alvarado-Mallart R.-M., Sotelo C. Cerebellar grafting in murine heredodegenerative ataxia. Adv. Neurol. 61:1993;181-192.
3. Burright E. N., Clark H. B., Servadio A., Matilla T., Feddersen R. M., Yunis W. S., Duvick L. A., Zoghbi H. Y., Orr H. T. SCA1 transgenic mice: A model for neurodegeneration caused by an expanded CAG trinucleotide repeat. Cell. 82:1995;937-948.
4. Caddy K. W. T., Biscoe T. J. Structural and quantitative studies in the normal C3H and Lurcher mutant mouse. Phil. Trans. R. Soc. Lond. B. Biol. Sci. 287:1979;167-201.
5. Clark H. B., Burright E. N., Yunis W. S., Larson S., Wilcox C., Hartman B., Matilla A., Zoghbi H. Y., Orr H. T. Purkinje cell expression of a mutant allele of SCA1 in transgenic mice leads to disparate effects on motor behaviors, followed by a progressive cerebellar dysfunction and histological alterations. J. Neurosci. 17:1997;7385-7395.
6. Cummings C. J., Mancini M. A., Antalffy B., De Franco D. B., Orr H. T., Zoghbi H. Y. Chaperone suppression of aggregation and altered subcellular proteosome localization imply protein misfolding in SCA1. Nature Genet. 19:1998;148-154.
7. Dunnett S. B., Carter R. J., Watts C., Torres E. M., Mahal A., Mangiarini L., Bates G., Morton A. J. Striatal transplantation in a transgenic mouse model of Huntington's disease. Exp. Neurol. 154:1998;31-40.
8. Gardette R., Alvarado-Mallart R. M., Crepel F., Sotelo C. Electrophysiological demonstration of a synaptic integration of transplanted Purkinje cells into the cerebellum of the adult Purkinje cell degeneration mutant mouse. Neuroscience. 24:1988;777-789.
9. Cell. 72:1993;971-983.
10. Ikeda H., Yamaguchi M., Sugai S., Aze Y., Narumiya S., Kakizuka A. Expanded polyglutamine in the Machado-Joseph disease protein induces cell death in vitro and in vivo. Nature Genet. 13:1996;196-202.
11. Keep M., Alvarado-Mallart R. M., Sotelo C. New insight on the factors orienting the axonal outgrowth of grafted Purkinje cells in the pcd cerebellum. Dev. Neurosci. 14:1992;153-165.
12. Kesslak J. P., Nieto-Sampedro M., Globus J., Cotman C. W. Transplants of purified astrocytes promote behavioral recovery after frontal cortex ablation. Exp. Neurology. 92:1986;377-390.
13. Koide R., Ikeuchi T., Onodera O., Tanaka H., Igarashi S., Endo K., Takahashi H., Kondo R., Ishikawa A., Hayashi T., Saito M., Tomoda A., Mike T., Naito H., Ikuta F., Tsuji S. Unstable expansion of CAG repeat in hereditary dentatorubral-pallidoluysian atrophy (DRPLA). Nature Genet. 6:1994;9-13.
14. Kopyov O. V., Jacques S., Kurth M., Philpott L. M., Lee A., Patterson M., Duma C., Liberman A., Eagle K. S. Fetal transplantation for Huntington's disease: Clinical studies. Freeman T. B., Kordower J. H. Fetal Transplantation in Neurological Disease. 1998;95-134 Humana Press, Totowa.
15. Kopyov O. V., Jacques S., Liberman A., Duma C. M., Eagle K. S. Safety of intrastriatal neurotransplantation for Huntington's disease patients. Exp. Neurol. 119:1998;97-108.
16. Kosby B. T., Zoghbi H. Y. The CAG/polyglutamine tract diseases: Gene products and molecular pathogenesis. Brain Pathol. 7:1997;927-942.
17. Laspada A. R., Wilson E. M., Lubahn D. B., Harding A. E., Fischbeck K. E. Androgen receptor gene mutations in X-linked spinal and bulbar muscular atrophy. Nature. 352:1991;77-79.
18. Low W. C., Triarhou L. C., Ghetti B. Cerebellar transplants into mutant mice with Purkinje and granule cell degeneration. Ann. N. Y. Acad. Sci. 495:1987;740-744.
19. Madrazo I., Franco-Bourland R. E., Castrejon H., Cuevas C., Ostrosky-Solis F. Fetal striatal homotransplantation for Huntington's disease: First two case reports. Neurol. Res. 17:1995;312-315.
20. Mangiarini L., Sathasivani K., Seller M., Cozens B., Harper A., Hetherington C., Lawton M., Trottier Y., Lehrach H., Davies S. W., Bates G. P. Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice. Cell. 87:1996;493-506.
21. Palfi S., Nguyen J. P., Brugieres P., Leguerinel C., Hantraye P., Remy P., Rostaing S., Defer G. L., Cesaro P., Keravel Y., Peschanski M. MRI-stereotactical approach for neural grafting in basal ganglia disorders. Exp. Neurol. 150:1998;272-281.
22. Philpott L. M., Kopyov O. V., Lee A. J., Jacques S., Duma C. M., Caine S., Yang M., Eagle K. S. Neuropsychological functioning following fetal striatal transplantation in Huntington's chorea: Three case presentations. Cell Transplant. 6:1997;203-212.
23. Scherzinger E., Lurz R., Turmaine M., Mangiarini L., Hollenbach B., Hasenbank R., Bates G. P., Davies S. W., Lehrach H., Wanker E. E. Huntingtin-encoded polyglutamine expansions form amyloid-like protein aggregates in vitro and in vivo. Cell. 90:1997;549-558.
24. Servadio A., Koshy B., Armstrong D., Antalffy B., Orr H. T., Zoghbi H. Y. Expression analysis of the ataxin-1 protein in tissues from normal and spinocerebellar ataxia type 1 individuals. Nature Genet. 10:1995;94-98.
25. Skinner P. J., Koshy B. T., Cummings C. J., Klement I. A., Helin K., Servadio A., Zoghbi H. Y., Orr H. T. Ataxin-1 with an expanded glutamine tract alters nuclear matrix-associated structures. Nature. 389:1997;971-974.
26. Sotelo C., Alvarado-Mallart R. M., Frain M., Vernet M. Molecular plasticity of adult Bergmann fibers is associated with radial migration of grafted Purkinje cells. J. Neurosci. 14:1994;124-133.
27. Sramka M., Rattaj M., Molina H., Vojtassak J., Belan V., Ruzicky E. Stereotactic technique and pathophysiological mechanisms of neurotransplantation in Huntington's chorea. Stereotact. Funct. Neurosurg. 58:1992;79-83.
28. Triarhou L. C. Neural Transplantation in Cerebellar Ataxia. 1997;Chapman & Hall, New York.
29. Triarhou L. C., Low W. C., Ghetti B. Intraparenchymal grafting of cerebellar cell suspensions to the deep cerebellar nuclei of pcd mutant mice, with particular emphasis on re-establishment of a Purkinje cell cortico-nuclear projection. Anat. Embryol. 185:1992;409-420.
30. Triarhou L. C., Zhang W., Lee W.-H. Graft-induced restoration of function in hereditary cerebellar ataxia. NeuroReport. 6:1995;1827-1832.
31. White J. K., Auerbach W., Duyao M. P., Vonsattel J. P., Gusella J. F., Joyner A. L., MacDonald M. E. Huntingtin is required for neurogenesis and is not impaired by the Huntington's disease CAG expansion. Nature Genet. 17:1997;404-410.
32. Zhang W., Lee W.-H., Triarhou L. C. Grafted cerebellar cells in a mouse model of hereditary ataxia express IGF-I system genes and partially restore behavioral function. Nature Med. 2:1996;65-71.
33. Zoghbi H. Y. Spinocerebellar ataxia type 1. Clin. Neurosci. 3:1995;5-11.