Functional biology of the neuronal ceroid lipofuscinoses (NCL) proteins - PubMed (original) (raw)
Review
. 2006 Oct;1762(10):920-33.
doi: 10.1016/j.bbadis.2006.05.007. Epub 2006 Jun 3.
Affiliations
- PMID: 16839750
- DOI: 10.1016/j.bbadis.2006.05.007
Free article
Review
Functional biology of the neuronal ceroid lipofuscinoses (NCL) proteins
Aija Kyttälä et al. Biochim Biophys Acta. 2006 Oct.
Free article
Abstract
Neuronal ceroid lipofucinoses (NCLs) are a group of severe neurodegenerative disorders characterized by accumulation of autofluorescent ceroid lipopigment in patients' cells. The different forms of NCL share many similar pathological features but result from mutations in different genes. The genes affected in NCLs encode both soluble and transmembrane proteins and are localized to ER or to the endosomes/lysosomes. Due to selective vulnerability of the central nervous system in the NCL disorders, the corresponding proteins are proposed to have important, tissue specific roles in the brain. The pathological similarities of the different NCLs have led not only to the grouping of these disorders but also to suggestion that the NCL proteins function in the same biological pathway. Despite extensive research, including the development of several model organisms for NCLs and establishment of high-throughput techniques, the precise biological function of many of the NCL proteins has remained elusive. The aim of this review is to summarize the current knowledge of the functions, or proposed functions, of the different NCL proteins.
Similar articles
- Correlations between genotype, ultrastructural morphology and clinical phenotype in the neuronal ceroid lipofuscinoses.
Mole SE, Williams RE, Goebel HH. Mole SE, et al. Neurogenetics. 2005 Sep;6(3):107-26. doi: 10.1007/s10048-005-0218-3. Epub 2005 Sep 28. Neurogenetics. 2005. PMID: 15965709 Review. - Molecular basis of the neuronal ceroid lipofuscinoses: mutations in CLN1, CLN2, CLN3, and CLN5.
Mole SE, Mitchison HM, Munroe PB. Mole SE, et al. Hum Mutat. 1999;14(3):199-215. doi: 10.1002/(SICI)1098-1004(1999)14:3<199::AID-HUMU3>3.0.CO;2-A. Hum Mutat. 1999. PMID: 10477428 Review. - The molecular genetic basis of the neuronal ceroid lipofuscinoses.
Gardiner RM. Gardiner RM. Neurol Sci. 2000;21(3 Suppl):S15-9. doi: 10.1007/s100720070035. Neurol Sci. 2000. PMID: 11073223 Review. - Cellular pathology and pathogenic aspects of neuronal ceroid lipofuscinoses.
Kida E, Golabek AA, Wisniewski KE. Kida E, et al. Adv Genet. 2001;45:35-68. doi: 10.1016/s0065-2660(01)45003-6. Adv Genet. 2001. PMID: 11332776 Review. - Neuronal ceroid lipofuscinoses and possible pathogenic mechanism.
Zhong N. Zhong N. Mol Genet Metab. 2000 Sep-Oct;71(1-2):195-206. doi: 10.1006/mgme.2000.3057. Mol Genet Metab. 2000. PMID: 11001811 Review.
Cited by
- TRAM1 is involved in disposal of ER membrane degradation substrates.
Ng CL, Oresic K, Tortorella D. Ng CL, et al. Exp Cell Res. 2010 Aug 1;316(13):2113-22. doi: 10.1016/j.yexcr.2010.04.010. Epub 2010 Apr 27. Exp Cell Res. 2010. PMID: 20430023 Free PMC article. - Exacerbating and reversing lysosomal storage diseases: from yeast to humans.
Rajakumar T, Munkacsi AB, Sturley SL. Rajakumar T, et al. Microb Cell. 2017 Aug 25;4(9):278-293. doi: 10.15698/mic2017.09.588. Microb Cell. 2017. PMID: 28913343 Free PMC article. Review. - Activation of PPARα Exhibits Therapeutic Efficacy in a Mouse Model of Juvenile Neuronal Ceroid Lipofuscinosis.
Jana M, Dutta D, Poddar J, Pahan K. Jana M, et al. J Neurosci. 2023 Mar 8;43(10):1814-1829. doi: 10.1523/JNEUROSCI.2447-21.2023. Epub 2023 Jan 25. J Neurosci. 2023. PMID: 36697260 Free PMC article. - Analysis of potential biomarkers and modifier genes affecting the clinical course of CLN3 disease.
Lebrun AH, Moll-Khosrawi P, Pohl S, Makrypidi G, Storch S, Kilian D, Streichert T, Otto B, Mole SE, Ullrich K, Cotman S, Kohlschütter A, Braulke T, Schulz A. Lebrun AH, et al. Mol Med. 2011;17(11-12):1253-61. doi: 10.2119/molmed.2010.00241. Epub 2011 Aug 18. Mol Med. 2011. PMID: 21863212 Free PMC article.
Publication types
MeSH terms
Substances
LinkOut - more resources
Full Text Sources
Miscellaneous