Corrigendum - Antisense-mediated Exon Skipping Decreases Tau Protein Expression: A Potential Therapy for Tauopathies (Molecular Therapy-Nucleic Acids (2014) 3 (e180) DOI: 10.1038/mtna.2014.30);Antisense-mediated exon skipping decreases Tau protein expression: A potential therapy for tauopathies

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages

  Sud, Reeteka ^a   Geller, Evan T ^a   Schellenberg, Gerard D ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Antisense oligonucleotides Therapeutic proof of concept

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; MESSENGER RNA; MORPHOLINO OLIGONUCLEOTIDE; TAU PROTEIN;

ALTERNATIVE RNA SPLICING; ANIMAL TISSUE; ARTICLE; BINDING SITE; CONTROLLED STUDY; DOWN REGULATION; ENHANCER REGION; EXON; EXON SKIPPING; FEASIBILITY STUDY; HUMAN; HUMAN TISSUE; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NEUROBLASTOMA CELL LINE; NONHUMAN; PRIORITY JOURNAL; PROTEIN AGGREGATION; PROTEIN EXPRESSION; START CODON; TAUOPATHY; TRANSLATION INITIATION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTISENSE THERAPY; CODON; DNA FLANKING REGION; FRAMESHIFT MUTATION; HUMAN CELL; NONSENSE MEDIATED MRNA DECAY; NUCLEOTIDE BINDING SITE; POINT MUTATION; PROGRAM FEASIBILITY; RNA SPLICING; STOP CODON;

EID: 84905270419     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2014.55     Document Type: Erratum

References (47)

1. Lee, VM, Goedert, M and Trojanowski, JQ (2001). Neurodegenerative tauopathies. Annu Rev Neurosci 24: 1121-1159. (Pubitemid 32695256)
2. Poorkaj, P, Bird, TD, Wijsman, E, Nemens, E, Garruto, RM, Anderson, L et al. (1998). Tau is a candidate gene for chromosome 17 frontotemporal dementia. Ann Neurol 43: 815-825. (Pubitemid 28280226)
3. Spillantini, MG, Murrell, JR, Goedert, M, Farlow, MR, Klug, A and Ghetti, B (1998). Mutation in the tau gene in familial multiple system tauopathy with presenile dementia. Proc Natl Acad Sci USA 95: 7737-7741. (Pubitemid 28293323)
4. Hutton, M, Lendon, CL, Rizzu, P, Baker, M, Froelich, S, Houlden, H et al. (1998). Association of missense and 5'-splice-site mutations in tau with the inherited dementia FTDP-17. Nature 393: 702-705.
5. Hong, M, Zhukareva, V, Vogelsberg-Ragaglia, V, Wszolek, Z, Reed, L, Miller, BI et al. (1998). Mutation-specific functional impairments in distinct tau isoforms of hereditary FTDP-17. Science 282: 1914-1917. (Pubitemid 28555275)
6. D'Souza, I, Poorkaj, P, Hong, M, Nochlin, D, Lee, VM, Bird, TD et al. (1999). Missense and silent tau gene mutations cause frontotemporal dementia with parkinsonism-chromosome 17 type, by affecting multiple alternative RNA splicing regulatory elements. Proc Natl Acad Sci USA 96: 5598-5603. (Pubitemid 29234664)
7. Höglinger, GU, Melhem, NM, Dickson, DW, Sleiman, PM, Wang, LS, Klei, L et al.; PSP Genetics Study Group. (2011). Identification of common variants influencing risk of the tauopathy progressive supranuclear palsy. Nat Genet 43: 699-705.
8. Baker, M, Litvan, I, Houlden, H, Adamson, J, Dickson, D, Perez-Tur, J et al. (1999). Association of an extended haplotype in the tau gene with progressive supranuclear palsy. Hum Mol Genet 8: 711-715. (Pubitemid 29139987)
9. Simón-Sánchez, J, Schulte, C, Bras, JM, Sharma, M, Gibbs, JR, Berg, D et al. (2009). Genome-wide association study reveals genetic risk underlying Parkinson's disease. Nat Genet 41: 1308-1312.
10. Harada, A, Oguchi, K, Okabe, S, Kuno, J, Terada, S, Ohshima, T et al. (1994). Altered microtubule organization in small-calibre axons of mice lacking tau protein. Nature 369: 488-491. (Pubitemid 24210568)
11. Ikegami, S, Harada, A and Hirokawa, N (2000). Muscle weakness, hyperactivity, and impairment in fear conditioning in tau-deficient mice. Neurosci Lett 279: 129-132. (Pubitemid 30069810)
12. Takei, Y, Teng, J, Harada, A and Hirokawa, N (2000). Defects in axonal elongation and neuronal migration in mice with disrupted tau and map1b genes. J Cell Biol 150: 989-1000.
13. Higuchi, M, Ishihara, T, Zhang, B, Hong, M, Andreadis, A, Trojanowski, J et al. (2002). Transgenic mouse model of tauopathies with glial pathology and nervous system degeneration. Neuron 35: 433-446. (Pubitemid 34879944)
14. Ishihara, T, Hong, M, Zhang, B, Nakagawa, Y, Lee, MK, Trojanowski, JQ et al. (1999). Agedependent emergence and progression of a tauopathy in transgenic mice overexpressing the shortest human tau isoform. Neuron 24: 751-762.
15. Lewis, J, McGowan, E, Rockwood, J, Melrose, H, Nacharaju, P, Van Slegtenhorst, M et al. (2000). Neurofibrillary tangles, amyotrophy and progressive motor disturbance in mice expressing mutant (P301L) tau protein. Nat Genet 25: 402-405. (Pubitemid 32983430)
16. Götz, J, Chen, F, Barmettler, R and Nitsch, RM (2001). Tau filament formation in transgenic mice expressing P301L tau. J Biol Chem 276: 529-534. (Pubitemid 32050347)
17. Clavaguera, F, Bolmont, T, Crowther, RA, Abramowski, D, Frank, S, Probst, A et al. (2009). Transmission and spreading of tauopathy in transgenic mouse brain. Nat Cell Biol 11: 909-913.
18. Iba, M, Guo, JL, McBride, JD, Zhang, B, Trojanowski, JQ and Lee, VM (2013). Synthetic tau fibrils mediate transmission of neurofibrillary tangles in a transgenic mouse model of Alzheimer's-like tauopathy. J Neurosci 33: 1024-1037.
19. Roberson, ED, Scearce-Levie, K, Palop, JJ, Yan, F, Cheng, IH, Wu, T et al. (2007). Reducing endogenous tau ameliorates amyloid beta-induced deficits in an Alzheimer's disease mouse model. Science 316: 750-754. (Pubitemid 46717684)
20. Ittner, LM, Ke, YD, Delerue, F, Bi, M, Gladbach, A, van Eersel, J et al. (2010). Dendritic function of tau mediates amyloid-beta toxicity in Alzheimer's disease mouse models. Cell 142: 387-397.
21. Shipton, OA, Leitz, JR, Dworzak, J, Acton, CE, Tunbridge, EM, Denk, F et al. (2011). Tau protein is required for amyloid {beta}-induced impairment of hippocampal long-term potentiation. J Neurosci 31: 1688-1692.
22. Vossel, KA, Zhang, K, Brodbeck, J, Daub, AC, Sharma, P, Finkbeiner, S et al. (2010). Tau reduction prevents Abeta-induced defects in axonal transport. Science 330: 198.
23. Aartsma-Rus, A, van Vliet, L, Hirschi, M, Janson, AA, Heemskerk, H, de Winter, CL et al. (2009). Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms. Mol Ther 17: 548-553.
24. Lu, QL, Mann, CJ, Lou, F, Bou-Gharios, G, Morris, GE, Xue, SA et al. (2003). Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat Med 9: 1009-1014. (Pubitemid 37021780)
25. Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K et al. (2011). Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet 378: 595-605.
26. Goemans, NM, Tulinius, M, van den Akker, JT, Burm, BE, Ekhart, PF, Heuvelmans, N et al. (2011). Systemic administration of PRO051 in Duchenne's muscular dystrophy. N Engl J Med 364: 1513-1522.
27. Andreadis, A, Brown, WM and Kosik, KS (1992). Structure and novel exons of the human tau gene. Biochemistry 31: 10626-10633.
28. Goedert, M, Wischik, CM, Crowther, RA, Walker, JE and Klug, A (1988). Cloning and sequencing of the cDNA encoding a core protein of the paired helical filament of Alzheimer disease: identification as the microtubule-associated protein tau. Proc Natl Acad Sci USA 85: 4051-4055.
29. Goedert, M, Spillantini, MG, Potier, MC, Ulrich, J and Crowther, RA (1989). Cloning and sequencing of the cDNA encoding an isoform of microtubule-associated protein tau containing four tandem repeats: differential expression of tau protein mRNAs in human brain. EMBO J 8: 393-399. (Pubitemid 19274956)
30. D'Souza, I and Schellenberg, GD (2000). Determinants of 4-repeat tau expression. Coordination between enhancing and inhibitory splicing sequences for exon 10 inclusion. J Biol Chem 275: 17700-17709. (Pubitemid 30430819)
31. D'Souza, I and Schellenberg, GD (2006). Arginine/serine-rich protein interaction domaindependent modulation of a tau exon 10 splicing enhancer: altered interactions and mechanisms for functionally antagonistic FTDP-17 mutations Delta280K AND N279K. J Biol Chem 281: 2460-2469. (Pubitemid 43845705)
32. D'Souza, I and Schellenberg, GD (2002). tau Exon 10 expression involves a bipartite intron 10 regulatory sequence and weak 5' and 3' splice sites. J Biol Chem 277: 26587-26599. (Pubitemid 34967157)
33. Smith, CJ, Anderton, BH, Davis, DR and Gallo, JM (1995). Tau isoform expression and phosphorylation state during differentiation of cultured neuronal cells. FEBS Lett 375: 243-248.
34. Poorkaj, P, Kas, A, D'Souza, I, Zhou, Y, Pham, Q, Stone, M et al. (2001). A genomic sequence analysis of the mouse and human microtubule-associated protein tau. Mamm Genome 12: 700-712. (Pubitemid 32800560)
35. McMillan, P, Korvatska, E, Poorkaj, P, Evstafjeva, Z, Robinson, L, Greenup, L et al. (2008). Tau isoform regulation is region-and cell-specific in mouse brain. J Comp Neurol 511: 788-803.
36. Yasuda, M, Takamatsu, J, D'Souza, I, Crowther, RA, Kawamata, T, Hasegawa, M et al. (2000). A novel mutation at position +12 in the intron following exon 10 of the tau gene in familial frontotemporal dementia (FTD-Kumamoto). Ann Neurol 47: 422-429. (Pubitemid 30194597)
37. Kalbfuss, B, Mabon, SA and Misteli, T (2001). Correction of alternative splicing of tau in frontotemporal dementia and parkinsonism linked to chromosome 17. J Biol Chem 276: 42986-42993.
38. DeVos, SL, Goncharoff, DK, Chen, G, Kebodeaux, CS, Yamada, K, Stewart, FR et al. (2013). Antisense reduction of tau in adult mice protects against seizures. J Neurosci 33: 12887-12897.
39. Alvarez-Erviti, L, Seow, Y, Yin, H, Betts, C, Lakhal, S and Wood, MJ (2011). Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes. Nat Biotechnol 29: 341-345.
40. Ding, H, Inoue, S, Ljubimov, AV, Patil, R, Portilla-Arias, J, Hu, J et al. (2010). Inhibition of brain tumor growth by intravenous poly (?-L-malic acid) nanobioconjugate with pHdependent drug release [corrected]. Proc Natl Acad Sci USA 107: 18143-18148.
41. Rosi, NL, Giljohann, DA, Thaxton, CS, Lytton-Jean, AK, Han, MS and Mirkin, CA (2006). Oligonucleotide-modified gold nanoparticles for intracellular gene regulation. Science 312: 1027-1030.
42. Kordasiewicz, HB, Stanek, LM, Wancewicz, EV, Mazur, C, McAlonis, MM, Pytel, KA et al. (2012). Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 74: 1031-1044.
43. Smith, RA, Miller, TM, Yamanaka, K, Monia, BP, Condon, TP, Hung, G et al. (2006). Antisense oligonucleotide therapy for neurodegenerative disease. J Clin Invest 116: 2290-2296. (Pubitemid 44162339)
44. Schwartz, S, Hall, E and Ast, G (2009). SROOGLE: webserver for integrative, user-friendly visualization of splicing signals. Nucleic Acids Res 37(Web Server issue): W189-W192. 45. van Meerloo, J, Kaspers, GJ and Cloos, J (2011). Cell sensitivity assays: the MTT assay. Methods Mol Biol 731: 237-245.
45. Bustin, SA, Benes, V, Garson, JA, Hellemans, J, Huggett, J, Kubista, M et al. (2009). The MIQE guidelines: minimum information for publication of quantitative real-time PCR experiments. Clin Chem 55: 611-622.
46. Livak, KJ and Schmittgen, TD (2001). Analysis of relative gene expression data using realtime quantitative PCR and the 2(-Delta Delta C(T)) Method. Methods 25: 402-408. (Pubitemid 34164012)
47. Hanger, DP, Gibb, GM, de Silva, R, Boutajangout, A, Brion, JP, Revesz, T et al. (2002). The complex relationship between soluble and insoluble tau in tauopathies revealed by efficient dephosphorylation and specific antibodies. FEBS Lett 531: 538-542. (Pubitemid 35336082)