Zobrazeno 1 - 10
of 78
pro vyhledávání: '"Matteo Bovolenta"'
Autor:
Juliette Lemoine, Auriane Dubois, Alan Dorval, Abbass Jaber, Ganesh Warthi, Kamel Mamchaoui, Tao Wang, Guillaume Corre, Matteo Bovolenta, Isabelle Richard
Publikováno v:
Scientific Reports, Vol 14, Iss 1, Pp 1-12 (2024)
Abstract Duchenne Muscular dystrophy (DMD), a yet-incurable X-linked recessive disorder that results in muscle wasting and loss of ambulation is due to mutations in the dystrophin gene. Exonic duplications of dystrophin gene are a common type of muta
Externí odkaz:
https://doaj.org/article/6161c960f714429d90dfa15c23adb38e
Autor:
Marika Faiella, Giada Botti, Alessandro Dalpiaz, Lorenzo Gnudi, Aurélie Goyenvalle, Barbara Pavan, Daniela Perrone, Matteo Bovolenta, Elena Marchesi
Publikováno v:
Pharmaceutics, Vol 16, Iss 8, p 1023 (2024)
Delivery represents a major hurdle to the clinical advancement of oligonucleotide therapeutics for the treatment of disorders such as Duchenne muscular dystrophy (DMD). In this preliminary study, we explored the ability of 2′-O-methyl-phosphorothio
Externí odkaz:
https://doaj.org/article/f564dd01072d4f529e4c43386b9bc6fe
Autor:
Giampaolo Morciano, Gaia Pedriali, Massimo Bonora, Rita Pavasini, Elisa Mikus, Simone Calvi, Matteo Bovolenta, Magdalena Lebiedzinska-Arciszewska, Mirko Pinotti, Alberto Albertini, Mariusz R. Wieckowski, Carlotta Giorgi, Roberto Ferrari, Lorenzo Galluzzi, Gianluca Campo, Paolo Pinton
Publikováno v:
Cell Reports, Vol 35, Iss 2, Pp 108983- (2021)
Summary: Preclinical models of ischemia/reperfusion injury (RI) demonstrate the deleterious effects of permeability transition pore complex (PTPC) opening in the first minutes upon revascularization of the occluded vessel. The ATP synthase c subunit
Externí odkaz:
https://doaj.org/article/827b58fa4eb044499de5d9de3ee09408
Autor:
Elena Marchesi, Rita Cortesi, Lorenzo Preti, Paola Rimessi, Maddalena Sguizzato, Matteo Bovolenta, Daniela Perrone
Publikováno v:
International Journal of Molecular Sciences, Vol 23, Iss 8, p 4270 (2022)
Our groups previously reported that conjugation at 3′-end with ursodeoxycholic acid (UDCA) significantly enhanced in vitro exon skipping properties of ASO 51 oligonucleotide targeting the human DMD exon 51. In this study, we designed a series of li
Externí odkaz:
https://doaj.org/article/b17d8f2ac4b74ac193dc561235532d8f
Autor:
Araksya Izmiryan, Clarisse Ganier, Matteo Bovolenta, Alain Schmitt, Fulvio Mavilio, Alain Hovnanian
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 554-567 (2018)
Recessive dystrophic epidermolysis bullosa is a rare and severe genetic skin disease resulting in blistering of the skin and mucosa. Recessive dystrophic epidermolysis bullosa (RDEB) is caused by a wide variety of mutations in COL7A1-encoding type VI
Externí odkaz:
https://doaj.org/article/b2ffef89e71c4eed88e785f863c41701
Autor:
Elena Marchesi, Matteo Bovolenta, Lorenzo Preti, Massimo L. Capobianco, Kamel Mamchaoui, Monica Bertoldo, Daniela Perrone
Publikováno v:
Molecules, Vol 26, Iss 24, p 7662 (2021)
Steric blocking antisense oligonucleotides (ASO) are promising tools for splice modulation such as exon-skipping, although their therapeutic effect may be compromised by insufficient delivery. To address this issue, we investigated the synthesis of a
Externí odkaz:
https://doaj.org/article/4032b6268bc14cc3a6b11fd7e41653ff
Autor:
Annalisa Lattanzi, Stephanie Duguez, Arianna Moiani, Araksya Izmiryan, Elena Barbon, Samia Martin, Kamel Mamchaoui, Vincent Mouly, Francesco Bernardi, Fulvio Mavilio, Matteo Bovolenta
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 7, Iss C, Pp 11-19 (2017)
Exonic duplications account for 10%–15% of all mutations in Duchenne muscular dystrophy (DMD), a severe hereditary neuromuscular disorder. We report a CRISPR (clustered regularly interspaced short palindromic repeat)/Cas9-based strategy to correct
Externí odkaz:
https://doaj.org/article/88c9ce137af0434d9ad8ef36a9936d97
Autor:
Nicole Ziliotto, Giovanna Marchetti, Chiara Scapoli, Matteo Bovolenta, Silvia Meneghetti, Andrea Benazzo, Barbara Lunghi, Dario Balestra, Lorenza Anna Laino, Nicolò Bozzini, Irene Guidi, Fabrizio Salvi, Sofia Straudi, Donato Gemmati, Erica Menegatti, Paolo Zamboni, Francesco Bernardi
Publikováno v:
Frontiers in Genetics, Vol 10 (2019)
In light of the complex nature of multiple sclerosis (MS) and the recently estimated contribution of low-frequency variants into disease, decoding its genetic risk components requires novel variant prioritization strategies. We selected, by reviewing
Externí odkaz:
https://doaj.org/article/c58b97f7c4414c37b117da9028f228eb
Autor:
Matteo Bovolenta, Daniela Erriquez, Emanuele Valli, Simona Brioschi, Chiara Scotton, Marcella Neri, Maria Sofia Falzarano, Samuele Gherardi, Marina Fabris, Paola Rimessi, Francesca Gualandi, Giovanni Perini, Alessandra Ferlini
Publikováno v:
PLoS ONE, Vol 7, Iss 9, p e45328 (2012)
The 2.2 Mb long dystrophin (DMD) gene, the largest gene in the human genome, corresponds to roughly 0.1% of the entire human DNA sequence. Mutations in this gene cause Duchenne muscular dystrophy and other milder X-linked, recessive dystrophinopathie
Externí odkaz:
https://doaj.org/article/9cf116aa6d174028b9a3d4f2ba59d040
Autor:
Silvia Pignani, Alessio Branchini, Federico Zappaterra, Francesco Bernardi, Matteo Bovolenta, Antonia Follenzi, Elena Barbon, Mirko Pinotti
Publikováno v:
Biochim Biophys Acta Gene Regul Mech
Biochim Biophys Acta Gene Regul Mech, 2019, 1862, pp.619-624. ⟨10.1016/j.bbagrm.2019.04.002⟩
Biochim Biophys Acta Gene Regul Mech, 2019, 1862, pp.619-624. ⟨10.1016/j.bbagrm.2019.04.002⟩
International audience; Engineered transcription factors (TF) have expanded our ability to modulate gene expression and hold great promise as bio-therapeutics. The first-generation TF, based on Zinc Fingers or Transcription-Activator-like Effectors (