12 Outlook for future treatment

Advances in Genetics

Volumn 45, Issue , 2001, Pages 217-224

  Zhong, Nanbert ^a,b   Wisniewski, Krystyna E ^a,b  

^a NEW YORK STATE INSTITUTE FOR BASIC RESEARCH IN DEVELOPMENTAL DISABILITIES   (United States)

^b SUNY DOWNSTATE MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

MEMBRANE PROTEIN;

CLINICAL TRIAL; ENZYMOLOGY; GENE THERAPY; GENETICS; HUMAN; LYSOSOME; METABOLISM; MOLECULAR CLONING; NEURONAL CEROID LIPOFUSCINOSIS; REVIEW;

CLINICAL TRIALS; CLONING, MOLECULAR; GENE THERAPY; HUMANS; LYSOSOMES; MEMBRANE PROTEINS; NEURONAL CEROID-LIPOFUSCINOSES;

EID: 0035237099     PISSN: 00652660     EISSN: None     Source Type: Book Series    
DOI: 10.1016/s0065-2660(01)45013-9     Document Type: Article

References (28)

1. Aberg L. Heiskala H. Vanhanen S.L. Himberg J.J. Hosking G. Yuen A. Santavuori P. Lamotrigine therapy in infantile ceroid lipofuscinosis (INCL) Neuropediatrics 28 1997 77 79
2. Aberg L. Kirveskari E. Santavuori P. Lamotrigine therapy in juvenile ceroid lipofuscinosis Epilepsia 40 1999 796 799
3. Azizi S.A. Stokes D. Augelli B.J. Digirolamo C. Prockop D.J. Engraftment and migration of human bone marrow stromal cells implanted in the brains of albino rats-similarities to astrocyte grafts Proc. Natl. Acad. Sci. (USA) 95 1998 3908 3913
4. Bennett M.J. Gayton A.R. Rittey C.D. Hosking G.P. Juvenile neuronal ceroid-lipofuscinosis: Developmental progress after supplementation with polyunsaturated fatty acids Dev. Med. Child. Neurol. 36 1994 630 638
5. Bennett M.J. Boriack R.L. Birch D.G. In-utero and post-delivery supplementation of motor neuron degeneration mutant mice with polyunsaturated fatty acids does not alter the clinical or pathological course Neuropediatrics 28 1997 82 84
6. Cooper J.D. Messer A. Feng A.K. Chua-Couzens J. Mobley W.C. Apparent loss and hypertrophy of interneurons in a mouse model of neuronal ceroid lipofuscinosis: Evidence for partial response to insulin-like growth factor-1 treatment J. Neurosci. 19 1999 2556 2567
7. Daly T.M. Vogler C. Levy B. Haskins M.E. Sands M.S. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease Proc. Natl. Acad. Sci. (USA) 96 1999 2296 2300
8. Davidson B.L. Stein C.S. Heth J.A. Martins I. Kotin R.M. Derksen T.A. Zabner J. Ghods A. Chiorini J.A. Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system Proc. Natl. Acad. Sci. (USA) 97 2000 3428 3432
9. Deeg H.J. Shulman H.M. Albrechtsen D. Graham T.C. Storb R. Koppang N. Batten's disease: Failure of allogeneic bone marrow transplantation to arrest disease progression in a canine model Clin. Genet. 37 1990 264 270
10. Dunn W.A. Raizada M.K. Vogt E.S. Brown E.A. Growth factor-induced growth in primary neuronal cultures of dogs with neuronal ceroid lipofuscinosis Int. J. Dev. Neurosci. 12 1994 185 196
11. Janes R.W. Munroe P.B. Mitchison H.M. Gardiner R.M. Mole S.E. Wallace B.A. A model for Batten disease protein CLN3: Functional implications from homology and mutations FEBS Lett. 399 1996 75 77
12. Katz M.L. Rice L.M. Gao C.L. Dietary carnitine supplements slow disease progression in a putative mouse model for hereditary ceroid-lipofuscinosis J. Neurosci. Res. 50 1997 123 132
13. Kessler P.D. Podsakoff G.M. Chen X. McQuiston S.A. Colosis P.C. Matelis L.A. Kurtzman G.J. Byrne B.J. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein Proc. Natl. Acad. Sci. (USA) 93 1996 14082 14087
14. Lake B.D. Henderson D.C. Oakhill A. Vellodi A. Bone marrow transplantation in Batten disease Am. J. Med. Genet. 57 1995 369 373
15. Lake B.D. Steward C.G. Oakhill A. Wilson J. Perham T.G. Bone marrow transplantation in late infantile Batten disease and juvenile Batten disease Neuropediatrics 28 1997 80 81
16. Li M. Pevny L. Lovell-Badge R. Smith A. Generation of purified neural precursors from embryonic stem cells by lineage selection Curr. Biol. 8 1998 971 974
17. Pearce D.A. Carr C.J. Das B. Sherman F. Phenotypic reversal of the defects in yeast by chloroquine: A model for Batten disease Proc. Natl. Acad. Sci. (USA) 96 1999 11341 11345
18. Pereira R.F. O'Hara M.D. Laptev A.V. Halford K.W. Pollard M.D. Class R. Simon D. Livezey K. Prockop D.J. Marrow stromal cells as a source of progenitor cells for nonhematopoietic tissues in transgenic mice with a phenotype of osteogenesis imperfecta Proc. Natl. Acad. Sci. (USA) 95 1998 1142 1147
19. Phylactou L.A. Kilpatrick M.W. Wood M.J.A. Ribozymes as therapeutic tools for genetic disease Hum. Mol. Genet. 7 1998 1649 1653
20. Ranta S. Zhang Y. Ross B. Lonka L. Takkunen E. Messer A.M. Sharp J. Wheeler R. Kusumi K. Mole S. Liu W. Soares M.B. de Fatima Bonaldo M. Hirvasniemi A. de la Chapelle A. Gilliam T.C. Lehesjoki A. The neuronal ceroid lipofuscinoses in human EPMR and mnd mutant mice are associated with mutations in CLN8 Nat. Genet. 23 1999 233 2236
21. Santavuori P. Moren R. Experience of antioxidant treatment in neuronal ceroid-lipofuscinosis of spielmeyer-sjogren type Neuropadiatrics 8 1977 333 344
22. Sharp J.D. Wheeler R.B. Lake B.D. Fox M. Gardiner R.M. Williams R.E. Genetic and physical mapping of the CLN6 gene on chromosome 15q21–23 Mol. Genet. Metab. 66 1999 329 331
23. Sleat D.E. Sohar I. Pullarkat P.S. Lobel P. Pullarkat R.K. Specific alterations in levels of mannose 6-phosphoryled glycoproteins in different neuronal ceroid lipofuscinoses Biochem. J. 334 1998 547 551
24. Westermarck T. Santavuori P. Principle of antioxidant therapy in neuronal ceroid lipofuscinosis Med. Biol. 62 1984 148 151
25. Westlake, V. J., Jolly, R. D., Jones, B. R., Mellor, D. J., Machon, R., Zanjani, E. D., and Krivit, W. Hematopoietic cell transplantation in fetal lambs with ceroid-lipofuscinosis. Am. J. Med. Genet. 57 , 365–368.
26. Zhong N. Ju W. Moroziewicz D. Jurkiewez A. Wisniewski K. Brown W.T. Molecular pathogenic studies of Batten disease: Identification and characterization of Battenin-interactive proteins J. Mol. Diag. 1 1999 G23 (Abstr.)
27. Zhong N. Ju W. Moroziewicz D. Jurkewez A. Wisniewski K. Brown W.T. CLN-encoded proteins do not interact with each other Neurogenetics 86 2000 in press
28. Zhong N. Ju W. Moroziewicz D. Jurkewez A. Wisniewski K. Brown W.T. Proteins BIPs and BIPf interact with Battenin/Battenim and may play central roles in the pathegenesis of NCL The 8th International Congress on Neuronal Ceroid Lipofuscinoses Sept. 20–24, 2000, Oxford, UK (Abstr.) 2000