Centre d'Information et de documentation du CRA Rhône-Alpes
CRA
Informations pratiques
-
Adresse
Centre d'information et de documentation
du CRA Rhône-Alpes
Centre Hospitalier le Vinatier
bât 211
95, Bd Pinel
69678 Bron CedexHoraires
Lundi au Vendredi
9h00-12h00 13h30-16h00Contact
Tél: +33(0)4 37 91 54 65
Mail
Fax: +33(0)4 37 91 54 37
-
Résultat de la recherche
1 recherche sur le mot-clé 'Viral vector'
Affiner la recherche Générer le flux rss de la recherche
Partager le résultat de cette recherche Faire une suggestion
Autism spectrum disorder: prospects for treatment using gene therapy / M. BENGER in Molecular Autism, 9 (2018)
[article]
Titre : Autism spectrum disorder: prospects for treatment using gene therapy Type de document : Texte imprimé et/ou numérique Auteurs : M. BENGER, Auteur ; M. KINALI, Auteur ; N. D. MAZARAKIS, Auteur Article en page(s) : 39p. Langues : Anglais (eng) Mots-clés : Animals Autism Spectrum Disorder/genetics/therapy Genetic Therapy/methods ASD models Autistic spectrum disorder Gene therapy Synaptic dysfunction Viral vector Index. décimale : PER Périodiques Résumé : Autism spectrum disorder (ASD) is characterised by the concomitant occurrence of impaired social interaction; restricted, perseverative and stereotypical behaviour; and abnormal communication skills. Recent epidemiological studies have reported a dramatic increase in the prevalence of ASD with as many as 1 in every 59 children being diagnosed with ASD. The fact that ASD appears to be principally genetically driven, and may be reversible postnatally, has raised the exciting possibility of using gene therapy as a disease-modifying treatment. Such therapies have already started to seriously impact on human disease and particularly monogenic disorders (e.g. metachromatic leukodystrophy, SMA type 1). In regard to ASD, technical advances in both our capacity to model the disorder in animals and also our ability to deliver genes to the central nervous system (CNS) have led to the first preclinical studies in monogenic ASD, involving both gene replacement and silencing. Furthermore, our increasing awareness and understanding of common dysregulated pathways in ASD have broadened gene therapy's potential scope to include various polygenic ASDs. As this review highlights, despite a number of outstanding challenges, gene therapy has excellent potential to address cognitive dysfunction in ASD. En ligne : https://dx.doi.org/10.1186/s13229-018-0222-8 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371
in Molecular Autism > 9 (2018) . - 39p.[article] Autism spectrum disorder: prospects for treatment using gene therapy [Texte imprimé et/ou numérique] / M. BENGER, Auteur ; M. KINALI, Auteur ; N. D. MAZARAKIS, Auteur . - 39p.
Langues : Anglais (eng)
in Molecular Autism > 9 (2018) . - 39p.
Mots-clés : Animals Autism Spectrum Disorder/genetics/therapy Genetic Therapy/methods ASD models Autistic spectrum disorder Gene therapy Synaptic dysfunction Viral vector Index. décimale : PER Périodiques Résumé : Autism spectrum disorder (ASD) is characterised by the concomitant occurrence of impaired social interaction; restricted, perseverative and stereotypical behaviour; and abnormal communication skills. Recent epidemiological studies have reported a dramatic increase in the prevalence of ASD with as many as 1 in every 59 children being diagnosed with ASD. The fact that ASD appears to be principally genetically driven, and may be reversible postnatally, has raised the exciting possibility of using gene therapy as a disease-modifying treatment. Such therapies have already started to seriously impact on human disease and particularly monogenic disorders (e.g. metachromatic leukodystrophy, SMA type 1). In regard to ASD, technical advances in both our capacity to model the disorder in animals and also our ability to deliver genes to the central nervous system (CNS) have led to the first preclinical studies in monogenic ASD, involving both gene replacement and silencing. Furthermore, our increasing awareness and understanding of common dysregulated pathways in ASD have broadened gene therapy's potential scope to include various polygenic ASDs. As this review highlights, despite a number of outstanding challenges, gene therapy has excellent potential to address cognitive dysfunction in ASD. En ligne : https://dx.doi.org/10.1186/s13229-018-0222-8 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=371