A
Alberto Malerba
Researcher at Royal Holloway, University of London
Publications - 46
Citations - 1064
Alberto Malerba is an academic researcher from Royal Holloway, University of London. The author has contributed to research in topics: Duchenne muscular dystrophy & Muscular dystrophy. The author has an hindex of 16, co-authored 39 publications receiving 816 citations. Previous affiliations of Alberto Malerba include University of London.
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Journal ArticleDOI
Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy.
Caroline Le Guiner,Laurent Servais,Marie Montus,Thibaut Larcher,Bodvael Fraysse,Sophie Moullec,M. Allais,Virginie François,Maeva Dutilleul,Alberto Malerba,Taeyoung Koo,Jean-Laurent Thibaut,B. Matot,Marie Devaux,Johanne Le Duff,Jack-Yves Deschamps,Inès Barthélémy,Inès Barthélémy,Stéphane Blot,Stéphane Blot,Isabelle Testault,Karim Wahbi,Stéphane Ederhy,Samia Martin,Philippe Veron,Christophe Georger,Takis Athanasopoulos,Takis Athanasopoulos,Takis Athanasopoulos,Carole Masurier,Federico Mingozzi,Pierre G. Carlier,Bernard Gjata,Jean-Yves Hogrel,Oumeya Adjali,Fulvio Mavilio,Thomas Voit,Philippe Moullier,Philippe Moullier,George Dickson +39 more
TL;DR: It is shown that locoregional and systemic delivery of a rAAV2/8 vector expressing a canine microdystrophin (cMD1) is effective in restoring dyStrophin expression and stabilizing clinical symptoms in studies performed on a total of 12 treated golden retriever muscular dystrophy dogs.
Journal ArticleDOI
Chronic systemic therapy with low-dose morpholino oligomers ameliorates the pathology and normalizes locomotor behavior in mdx mice.
Alberto Malerba,Paul S. Sharp,Ian R. Graham,Virginia Arechavala-Gomeza,Keith Foster,Francesco Muntoni,Dominic J. Wells,George Dickson +7 more
TL;DR: For the first time, a chronic long-term administration of LDs of unmodified PMO, equivalent to doses in use in DMD boys, is safe, significantly ameliorates the muscular dystrophic phenotype and improves the activity of dystrophin-deficient mice, thus encouraging the further clinical translation of this approach in humans.
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Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9.
Taeyoung Koo,Ngoc Lu-Nguyen,Alberto Malerba,Eunji Kim,Daesik Kim,Ornella Cappellari,Hee-Yeon Cho,George Dickson,Linda Popplewell,Jin-Soo Kim +9 more
TL;DR: It is shown that CjCas9 derived from Campylobacter jejuni can be used as a gene-editing tool to correct an out-of-frame DMD exon in DMD knockout mice, and muscle strength was enhanced in the Cj Cas9-treated muscles, without off-target mutations, indicating high efficiency and specificity of CJCas9.
Journal ArticleDOI
Antisense-induced Myostatin Exon Skipping Leads to Muscle Hypertrophy in Mice Following Octa guanidine Morpholino Oligomer Treatment
TL;DR: The substantial myostatin exon skipping observed after systemic injection of Vivo-PMO into normal mice led to a significant increase in soleus muscle mass as compared to the controls injected with normal saline suggesting that this approach could be feasible to ameliorate muscle-wasting pathologies.
Journal ArticleDOI
PABPN1 gene therapy for oculopharyngeal muscular dystrophy
Alberto Malerba,Pierre Klein,Houria Bachtarzi,Susan Jarmin,Gonzalo Córdova,Arnaud Ferry,Arnaud Ferry,V. Strings,M. Polay Espinoza,Kamel Mamchaoui,S. C. Blumen,J. Lacau St Guily,Vincent Mouly,M. Graham,Gillian Butler-Browne,David Suhy,Capucine Trollet,George Dickson +17 more
TL;DR: The treatment of a mouse model of OPMD with an adeno-associated virus-based gene therapy combining complete knockdown of endogenous PABPN1 and its replacement by a wild-type PABBN1 substantially reduces the amount of insoluble aggregates, decreases muscle fibrosis, reverts muscle strength to the level of healthy muscles and normalizes the muscle transcriptome.