Erratum: Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-Deficient mdx Mice (Peptide Nucleic Acid Promotes Systemic Dystrophin Expression and Functional Rescue in Dystrophin-deficient mdx Mice (2015) 4 (1–8), (S2162253116300403), (10.1038/mtna.2015.27));Peptide nucleic acid promotes systemic dystrophin expression and functional rescue in dystrophin-deficient mdx mice

Molecular Therapy Nucleic Acids

Volumn 4, Issue , 2015, Pages e255-

  Gao, Xianjun ^a   Shen, Xiaoyong ^a   Dong, Xue ^a   Ran, Ning ^a   Han, Gang ^a   Cao, Limin ^a   Gu, Ben ^a   Yin, HaiFang ^a  

^a TIANJIN MEDICAL UNIVERSITY   (China)

Author keywords

antisense oligonucleotide; Duchenne muscular dystrophy; dystrophin; exon skipping; peptide nucleic acid

Indexed keywords

ALANINE AMINOTRANSFERASE; ALPHA SARCOGLYCAN; ANTISENSE OLIGONUCLEOTIDE; ASPARTATE AMINOTRANSFERASE; BETA DYSTROGLYCAN; BETA SARCOGLYCAN; CD3 ANTIGEN; CD68 ANTIGEN; DYSTROPHIN; DYSTROPHIN ASSOCIATED PROTEIN COMPLEX; NEURONAL NITRIC OXIDE SYNTHASE; PEPTIDE NUCLEIC ACID;

ACIDITY; ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; AQUEOUS SOLUTION; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CD3+ T LYMPHOCYTE; CONTROLLED STUDY; CYTOPLASM; DIAPHRAGM; DRUG MEGADOSE; DRUG SAFETY; DRUG SOLUBILITY; EXON; EXON SKIPPING; FEASIBILITY STUDY; GASTROCNEMIUS MUSCLE; GRIP STRENGTH; HEART MUSCLE; MOUSE; NONHUMAN; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROTEIN EXPRESSION; QUADRICEPS FEMORIS MUSCLE; REPEATED DRUG DOSE; SARCOLEMMA; SINGLE DRUG DOSE; TIBIALIS ANTERIOR MUSCLE;

EID: 84943403142     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1016/j.omtn.2020.08.027     Document Type: Erratum

References (33)

1. Hoffman, EP, Brown, RH Jr, and Kunkel, LM. (1987). Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 51: 919-928
2. Bushby, K, Finkel, R, Birnkrant, DJ, Case, LE, Clemens, PR, Cripe, L, et al.; DMD Care Considerations Working Group. (2010). Diagnosis, and management of duchenne muscular dystrophy, part 1: diagnosis, and pharmacological, and psychosocial management. Lancet Neurol 9: 77-93
3. Bushby, K, Finkel, R, Birnkrant, DJ, Case, LE, Clemens, PR, Cripe, L, et al.; DMD Care Considerations Working Group. (2010). Diagnosis, and management of duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 9: 177-189
4. Hamada, S, Ishikawa, Y, Aoyagi, T, Ishikawa, Y, Minami, R, and Bach, JR. (2011). Indicators for ventilator use in duchenne muscular dystrophy. Respir Med 105: 625-629
5. Lostal, W, Kodippili, K, Yue, Y, and Duan, D. (2014). Full-length dystrophin reconstitution with adeno-associated viral vectors. Hum Gene Ther 25: 552-562
6. Lai, Y, Yue, Y, Liu, M, Ghosh, A, Engelhardt, JF, Chamberlain, JS, et al. (2005). Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol 23: 1435-1439
7. Fabb, SA, Wells, DJ, Serpente, P, and Dickson, G. (2002). Adeno-associated virus vector gene transfer, and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex, and inhibits myofibre degeneration in nude/mdx mice. Hum Mol Genet 11: 733-741
8. Bostick, B, Shin, JH, Yue, Y, Wasala, NB, Lai, Y, and Duan, D. (2012). AAV microdystrophin gene therapy alleviates stress-induced cardiac death but not myocardial fibrosis in >21-m-old mdx mice, an end-stage model of duchenne muscular dystrophy cardiomyopathy. J Mol Cell Cardiol 53: 217-222
9. Howard, MT, Anderson, CB, Fass, U, Khatri, S, Gesteland, RF, Atkins, JF, et al. (2004). Readthrough of dystrophin stop codon mutations induced by aminoglycosides. Ann Neurol 55: 422-426
10. Kayali, R, Ku, JM, Khitrov, G, Jung, ME, Prikhodko, O, and Bertoni, C. (2012). Read-through compound 13 restores dystrophin expression, and improves muscle function in the mdx mouse model for Duchenne muscular dystrophy. Hum Mol Genet 21: 4007-4020
11. Tinsley, J, Deconinck, N, Fisher, R, Kahn, D, Phelps, S, Gillis, JM, et al. (1998). Expression of full-length utrophin prevents muscular dystrophy in mdx mice. Nat Med 4: 1441-1444
12. Perkins, KJ, and Davies, KE. (2002). The role of utrophin in the potential therapy of duchenne muscular dystrophy. Neuromuscul Disord 12(suppl. 1): S78-S89
13. Gillis, JM. (2000). An attempt of gene therapy in duchenne muscular dystrophy: overexpression of utrophin in transgenic mdx mice. Acta Neurol Belg 100: 146-150
14. 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
15. Lu, QL, Rabinowitz, A, Chen, YC, Yokota, T, Yin, H, Alter, J, et al. (2005). Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 102: 198-203
16. Alter, J, Lou, F, Rabinowitz, A, Yin, H, Rosenfeld, J, Wilton, SD, et al. (2006). Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide, and improves dystrophic pathology. Nat Med 12: 175-177
17. Wu, B, Xiao, B, Cloer, C, Shaban, M, Sali, A, Lu, P, et al. (2011). One-year treatment of morpholino antisense oligomer improves skeletal, and cardiac muscle functions in dystrophic mdx mice. Mol Ther 19: 576-583
18. Aartsma-Rus, A, Kaman, WE, Bremmer-Bout, M, Janson, AA, Den dunnen, JT, van Ommen, GJ, et al. (2004). Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther 11: 1391-1398
19. Orum, H, and Wengel, J. (2001). Locked nucleic acids: a promising molecular family for gene-function analysis, and antisense drug development. Curr Opin Mol Ther 3: 239-243
20. Nielsen, PE, Egholm, M, Berg, RH, and Buchardt, O. (1991). Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Science 254: 1497-1500
21. Bushby, K, Finkel, R, Wong, B, Barohn, R, Campbell, C, Comi, GP, et al.; PTC124-GD- 007-DMD STUDY GROUP. (2014). Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve 50: 477-487
22. Aartsma-Rus, A, Van Deutekom, JC, Fokkema, IF, Van Ommen, GJ, and Den dunnen, JT. (2006). Entries in the Leiden duchenne muscular dystrophy mutation database: an overview of mutation types, and paradoxical cases that confirm the reading-frame rule. Muscle Nerve 34: 135-144
23. Mendell, JR, Rodino-Klapac, LR, Sahenk, Z, Roush, K, Bird, L, Lowes, LP, et al.; Eteplirsen Study Group. (2013). Eteplirsen for the treatment of duchenne muscular dystrophy. Ann Neurol 74: 637-647
24. 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
25. 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
26. Yin, H, Lu, Q, and Wood, M. (2008). Effective exon skipping, and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice. Mol Ther 16: 38-45
27. Cao, L, Han, G, Gu, B, and Yin, H. (2014). Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in duchenne muscular dystrophy. PLoS One 9: e111079
28. Blake, DJ, Weir, A, Newey, SE, and Davies, KE. (2002). Function, and genetics of dystrophin, and dystrophin-related proteins in muscle. Physiol Rev 82: 291-329
29. Thompson, MW, Murphy, EG, and McAlpine, PJ. (1967). An assessment of the creatine kinase test in the detection of carriers of duchenne muscular dystrophy. J Pediatr 71: 82-93
30. Lu, QL, Cirak, S, and Partridge, T. (2014). What can we learn from clinical trials of exon skipping for DMD?. Mol Ther Nucleic Acids 3: e152
31. Goyenvalle, A, Griffith, G, Babbs, A, El Andaloussi, S, Ezzat, K, Avril, A, et al. (2015). Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo- DNA oligomers. Nat Med 21: 270-275
32. Yin, H, Betts, C, Saleh, AF, Ivanova, GD, Lee, H, Seow, Y, et al. (2010). Optimization of peptide nucleic acid antisense oligonucleotides for local, and systemic dystrophin splice correction in the mdx mouse. Mol Ther 18: 819-827
33. Gao, X, Zhao, J, Han, G, Zhang, Y, Dong, X, Cao, L, et al. (2014). Effective dystrophin restoration by a novel muscle-homing peptide-morpholino conjugate in dystrophin-deficient mdx mice. Mol Ther 22: 1333-1341