Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Jonathan Battistini"'
Autor:
Beatrice Cardinali, Claudia Provenzano, Mariapaola Izzo, Christine Voellenkle, Jonathan Battistini, Georgios Strimpakos, Elisabetta Golini, Silvia Mandillo, Ferdinando Scavizzi, Marcello Raspa, Alessandra Perfetti, Denisa Baci, Dejan Lazarevic, Jose Manuel Garcia-Manteiga, Geneviève Gourdon, Fabio Martelli, Germana Falcone
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 27, Iss , Pp 184-199 (2022)
CRISPR/Cas9-mediated therapeutic gene editing is a promising technology for durable treatment of incurable monogenic diseases such as myotonic dystrophies. Gene-editing approaches have been recently applied to in vitro and in vivo models of myotonic
Externí odkaz:
https://doaj.org/article/5d433c05d99644d685e8e224594c7bdb
Autor:
Jose Manuel Garcia-Manteiga, Georgios Strimpakos, Christine Voellenkle, Geneviève Gourdon, Beatrice Cardinali, Germana Falcone, Silvia Mandillo, Claudia Provenzano, Marcello Raspa, Denisa Baci, Ferdinando Scavizzi, Mariapaola Izzo, Alessandra Perfetti, Dejan Lazarevic, Jonathan Battistini, Fabio Martelli, Elisabetta Golini
Publikováno v:
Molecular Therapy-Nucleic Acids
Molecular Therapy-Nucleic Acids, Elsevier, 2022, 27, pp.184-199. ⟨10.1016/j.omtn.2021.11.024⟩
Molecular Therapy. Nucleic Acids
MOLECULAR THERAPY-NUCLEIC ACIDS (2021). doi:10.1016/j.omtn.2021.11.024
info:cnr-pdr/source/autori:Cardinali B(1), Provenzano C(1), Izzo M(1), Voellenkle C(2), Battistini J(1), Strimpakos G(1), Golini E(1), Mandillo S(1), Scavizzi F(1), Raspa M(1), Perfetti A(2), Baci D(2), Lazarevic D(3), Garcia-Manteiga JM(3), Gourdon G(4), Martelli F(2), Falcone G(1)/titolo:Time-controlled and muscle-specific CRISPR%2FCas9 mediated deletion of CTG-repeat expansion in the DMPK gene/doi:10.1016%2Fj.omtn.2021.11.024/rivista:MOLECULAR THERAPY-NUCLEIC ACIDS/anno:2021/pagina_da:/pagina_a:/intervallo_pagine:/volume
Molecular Therapy: Nucleic Acids, Vol 27, Iss, Pp 184-199 (2022)
Molecular Therapy-Nucleic Acids, 2022, 27, pp.184-199. ⟨10.1016/j.omtn.2021.11.024⟩
Molecular Therapy-Nucleic Acids, Elsevier, 2022, 27, pp.184-199. ⟨10.1016/j.omtn.2021.11.024⟩
Molecular Therapy. Nucleic Acids
MOLECULAR THERAPY-NUCLEIC ACIDS (2021). doi:10.1016/j.omtn.2021.11.024
info:cnr-pdr/source/autori:Cardinali B(1), Provenzano C(1), Izzo M(1), Voellenkle C(2), Battistini J(1), Strimpakos G(1), Golini E(1), Mandillo S(1), Scavizzi F(1), Raspa M(1), Perfetti A(2), Baci D(2), Lazarevic D(3), Garcia-Manteiga JM(3), Gourdon G(4), Martelli F(2), Falcone G(1)/titolo:Time-controlled and muscle-specific CRISPR%2FCas9 mediated deletion of CTG-repeat expansion in the DMPK gene/doi:10.1016%2Fj.omtn.2021.11.024/rivista:MOLECULAR THERAPY-NUCLEIC ACIDS/anno:2021/pagina_da:/pagina_a:/intervallo_pagine:/volume
Molecular Therapy: Nucleic Acids, Vol 27, Iss, Pp 184-199 (2022)
Molecular Therapy-Nucleic Acids, 2022, 27, pp.184-199. ⟨10.1016/j.omtn.2021.11.024⟩
CRISPR/Cas9-mediated therapeutic gene editing is a promising technology for durable treatment of incurable monogenic diseases such as myotonic dystrophies. Gene-editing approaches have been recently applied to in vitro and in vivo models of myotonic
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::04839292564a0ed9916db5edb34c1f8c
https://hal.sorbonne-universite.fr/hal-03753518/document
https://hal.sorbonne-universite.fr/hal-03753518/document
Autor:
Beatrice Cardinali 1, Claudia Provenzano 1, Mariapaola Izzo 1, Jonathan Battistini 1, Georgios Strimpakos 1, Elisabetta Golini 1, Silvia Mandillo*1, Ferdinando Scavizzi 1, Marcello Raspa 1, Christine Voellenkle 2, Alessandra Perfetti 2, Denisa Baci 2, Fabio Martelli 2, Genevieve Gourdon 3, Germana Falcone 1
Publikováno v:
"Mechanistic Insights into Neurological Disorders and New Therapeutic Strategies", DSB Conference, CNR Rome, Italy, 07-08/07/2021
info:cnr-pdr/source/autori:Beatrice Cardinali 1, Claudia Provenzano 1, Mariapaola Izzo 1, Jonathan Battistini 1, Georgios Strimpakos 1, Elisabetta Golini 1, Silvia Mandillo*1, Ferdinando Scavizzi 1, Marcello Raspa 1, Christine Voellenkle 2, Alessandra Perfetti 2, Denisa Baci 2, Fabio Martelli 2, Genevieve Gourdon 3, Germana Falcone 1/congresso_nome:"Mechanistic Insights into Neurological Disorders and New Therapeutic Strategies"/congresso_luogo:DSB Conference, CNR Rome, Italy/congresso_data:07-08%2F07%2F2021/anno:2021/pagina_da:/pagina_a:/intervallo_pagine
info:cnr-pdr/source/autori:Beatrice Cardinali 1, Claudia Provenzano 1, Mariapaola Izzo 1, Jonathan Battistini 1, Georgios Strimpakos 1, Elisabetta Golini 1, Silvia Mandillo*1, Ferdinando Scavizzi 1, Marcello Raspa 1, Christine Voellenkle 2, Alessandra Perfetti 2, Denisa Baci 2, Fabio Martelli 2, Genevieve Gourdon 3, Germana Falcone 1/congresso_nome:"Mechanistic Insights into Neurological Disorders and New Therapeutic Strategies"/congresso_luogo:DSB Conference, CNR Rome, Italy/congresso_data:07-08%2F07%2F2021/anno:2021/pagina_da:/pagina_a:/intervallo_pagine
Background: Myotonic dystrophy type 1 (DM1) is a dominantly inherited neuromuscular disease caused by the abnormal expansion of CTG-triplets in the 3' untranslated region of the DMPK gene. While therapeutic approaches that neutralize the toxic DMPK t
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=cnr_________::01b75d694032588d55600578e19c1ada
https://publications.cnr.it/doc/458133
https://publications.cnr.it/doc/458133
Autor:
Mariapaola Izzo, Jonathan Battistini, Claudia Provenzano, Fabio Martelli, Beatrice Cardinali, Germana Falcone
Publikováno v:
International journal of molecular sciences (Online) 23 (2022). doi:10.3390/ ijms23094622
info:cnr-pdr/source/autori:Mariapaola Izzo (1), Jonathan Battistini (1), Claudia Provenzano (1), Fabio Martelli (2), Beatrice Cardinali (1), and Germana Falcone (1)/titolo:Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing/doi:10.3390%2F ijms23094622/rivista:International journal of molecular sciences (Online)/anno:2022/pagina_da:/pagina_a:/intervallo_pagine:/volume:23
info:cnr-pdr/source/autori:Mariapaola Izzo (1), Jonathan Battistini (1), Claudia Provenzano (1), Fabio Martelli (2), Beatrice Cardinali (1), and Germana Falcone (1)/titolo:Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing/doi:10.3390%2F ijms23094622/rivista:International journal of molecular sciences (Online)/anno:2022/pagina_da:/pagina_a:/intervallo_pagine:/volume:23
Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy affecting many different body tissues, predominantly skeletal and cardiac muscles and the central nervous system. The expansion of CTG repeats in the DM1 protein-kinase (DMPK) gene
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c71896fbe5c67172b6e72b91caca4c08
https://doi.org/10.3390/ijms23094622
https://doi.org/10.3390/ijms23094622