Malerba A - Search Results

1

Nuclear poly(A)-binding protein aggregates misplace a pre-mRNA outside of SC35 speckle causing its abnormal splicing.

Klein P, Oloko M, Roth F, Montel V, Malerba A, Jarmin S, Gidaro T, Popplewell L, Perie S, Lacau St Guily J, de la Grange P, Antoniou MN, Dickson G, Butler-Browne G, Bastide B, Mouly V, Trollet C. Klein P, et al. Among authors: malerba a. Nucleic Acids Res. 2016 Dec 15;44(22):10929-10945. doi: 10.1093/nar/gkw703. Epub 2016 Aug 9. Nucleic Acids Res. 2016. PMID: 27507886 Free PMC article.

2

Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.

Malerba A, Thorogood FC, Dickson G, Graham IR. Malerba A, et al. Hum Gene Ther. 2009 Sep;20(9):955-65. doi: 10.1089/hum.2008.157. Hum Gene Ther. 2009. PMID: 19469709

3

Gene therapy for muscular dystrophy: current progress and future prospects.

Trollet C, Athanasopoulos T, Popplewell L, Malerba A, Dickson G. Trollet C, et al. Among authors: malerba a. Expert Opin Biol Ther. 2009 Jul;9(7):849-66. doi: 10.1517/14712590903029164. Expert Opin Biol Ther. 2009. PMID: 19527108 Review.

4

Antisense-induced myostatin exon skipping leads to muscle hypertrophy in mice following octa-guanidine morpholino oligomer treatment.

Kang JK, Malerba A, Popplewell L, Foster K, Dickson G. Kang JK, et al. Among authors: malerba a. Mol Ther. 2011 Jan;19(1):159-64. doi: 10.1038/mt.2010.212. Epub 2010 Oct 5. Mol Ther. 2011. PMID: 20924365 Free PMC article.

5

Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.

Malerba A, Sharp PS, Graham IR, Arechavala-Gomeza V, Foster K, Muntoni F, Wells DJ, Dickson G. Malerba A, et al. Mol Ther. 2011 Feb;19(2):345-54. doi: 10.1038/mt.2010.261. Epub 2010 Nov 23. Mol Ther. 2011. PMID: 21102560 Free PMC article.

6

Bioinformatic and functional optimization of antisense phosphorodiamidate morpholino oligomers (PMOs) for therapeutic modulation of RNA splicing in muscle.

Popplewell LJ, Graham IR, Malerba A, Dickson G. Popplewell LJ, et al. Among authors: malerba a. Methods Mol Biol. 2011;709:153-78. doi: 10.1007/978-1-61737-982-6_10. Methods Mol Biol. 2011. PMID: 21194027

7

Delivery of AAV2/9-microdystrophin genes incorporating helix 1 of the coiled-coil motif in the C-terminal domain of dystrophin improves muscle pathology and restores the level of α1-syntrophin and α-dystrobrevin in skeletal muscles of mdx mice.

Koo T, Malerba A, Athanasopoulos T, Trollet C, Boldrin L, Ferry A, Popplewell L, Foster H, Foster K, Dickson G. Koo T, et al. Among authors: malerba a. Hum Gene Ther. 2011 Nov;22(11):1379-88. doi: 10.1089/hum.2011.020. Epub 2011 May 25. Hum Gene Ther. 2011. PMID: 21453126 Free PMC article.

8

Long-term systemic administration of unconjugated morpholino oligomers for therapeutic expression of dystrophin by exon skipping in skeletal muscle: implications for cardiac muscle integrity.

Malerba A, Boldrin L, Dickson G. Malerba A, et al. Nucleic Acid Ther. 2011 Aug;21(4):293-8. doi: 10.1089/nat.2011.0306. Nucleic Acid Ther. 2011. PMID: 21851223

9

Optimizing antisense oligonucleotides using phosphorodiamidate morpholino oligomers.

Popplewell LJ, Malerba A, Dickson G. Popplewell LJ, et al. Among authors: malerba a. Methods Mol Biol. 2012;867:143-67. doi: 10.1007/978-1-61779-767-5_10. Methods Mol Biol. 2012. PMID: 22454060

10

Dual Myostatin and Dystrophin Exon Skipping by Morpholino Nucleic Acid Oligomers Conjugated to a Cell-penetrating Peptide Is a Promising Therapeutic Strategy for the Treatment of Duchenne Muscular Dystrophy.

Malerba A, Kang JK, McClorey G, Saleh AF, Popplewell L, Gait MJ, Wood MJ, Dickson G. Malerba A, et al. Mol Ther Nucleic Acids. 2012 Dec 18;1(12):e62. doi: 10.1038/mtna.2012.54. Mol Ther Nucleic Acids. 2012. PMID: 23250360 Free PMC article.

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