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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 Recent progress and considerations for AAV gene therapies targeting the central nervous system / E. A. LYKKEN in Journal of Neurodevelopmental Disorders, 10-1 (December 2018)
[article]
Titre : Recent progress and considerations for AAV gene therapies targeting the central nervous system Type de document : Texte imprimé et/ou numérique Auteurs : E. A. LYKKEN, Auteur ; C. SHYNG, Auteur ; R. J. EDWARDS, Auteur ; A. ROZENBERG, Auteur ; S. J. GRAY, Auteur Année de publication : 2018 Article en page(s) : 16 p. Langues : Anglais (eng) Mots-clés : Aav9 Adeno-associated virus Cellular immunity Central nervous system Clinical trial Gene therapy Neutralizing antibody Index. décimale : PER Périodiques Résumé : BACKGROUND: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult to treat even when the underlying cause(s), such as genetic alterations, are understood. What treatments do exist are generally not curative and instead seek to improve quality of life for affected individuals. The advent of gene therapy via gene replacement offers the potential for transformative therapies to slow or even stop disease progression for current patients and perhaps minimize or prevent the appearance of symptoms in future patients. MAIN BODY: This review focuses on adeno-associated virus (AAV) gene therapies for diseases of the central nervous system. An overview of advances in AAV vector design for therapy is provided, along with a description of current strategies to develop AAV vectors with tailored tropism. Next, progress towards treatment of neurodegenerative diseases is presented at both the pre-clinical and clinical stages, focusing on a few select diseases to highlight broad categories of therapeutic parameters. Special considerations for more challenging cases are then discussed in addition to the immunological aspects of gene therapy. CONCLUSION: With the promising clinical trial results that have been observed for the latest AAV gene therapies and continued pre-clinical successes, the question is no longer whether a therapy can be developed for certain neurodevelopmental disorders, but rather, how quickly. En ligne : http://dx.doi.org/10.1186/s11689-018-9234-0 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=386
in Journal of Neurodevelopmental Disorders > 10-1 (December 2018) . - 16 p.[article] Recent progress and considerations for AAV gene therapies targeting the central nervous system [Texte imprimé et/ou numérique] / E. A. LYKKEN, Auteur ; C. SHYNG, Auteur ; R. J. EDWARDS, Auteur ; A. ROZENBERG, Auteur ; S. J. GRAY, Auteur . - 2018 . - 16 p.
Langues : Anglais (eng)
in Journal of Neurodevelopmental Disorders > 10-1 (December 2018) . - 16 p.
Mots-clés : Aav9 Adeno-associated virus Cellular immunity Central nervous system Clinical trial Gene therapy Neutralizing antibody Index. décimale : PER Périodiques Résumé : BACKGROUND: Neurodevelopmental disorders, as a class of diseases, have been particularly difficult to treat even when the underlying cause(s), such as genetic alterations, are understood. What treatments do exist are generally not curative and instead seek to improve quality of life for affected individuals. The advent of gene therapy via gene replacement offers the potential for transformative therapies to slow or even stop disease progression for current patients and perhaps minimize or prevent the appearance of symptoms in future patients. MAIN BODY: This review focuses on adeno-associated virus (AAV) gene therapies for diseases of the central nervous system. An overview of advances in AAV vector design for therapy is provided, along with a description of current strategies to develop AAV vectors with tailored tropism. Next, progress towards treatment of neurodegenerative diseases is presented at both the pre-clinical and clinical stages, focusing on a few select diseases to highlight broad categories of therapeutic parameters. Special considerations for more challenging cases are then discussed in addition to the immunological aspects of gene therapy. CONCLUSION: With the promising clinical trial results that have been observed for the latest AAV gene therapies and continued pre-clinical successes, the question is no longer whether a therapy can be developed for certain neurodevelopmental disorders, but rather, how quickly. En ligne : http://dx.doi.org/10.1186/s11689-018-9234-0 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=386