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This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact

CRISPR/Cas9 --- genome edited plants --- biosafety --- agriculture --- policy and legislation

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La dystrophie musculaire de Duchenne (DMD) est une maladie dégénérative héréditaire des muscles striés chez l’homme affectant essentiellement les garçons. Cette atteinte résulte généralement de la délétion d’un ou plusieurs exons du gène de la dystrophine entrainant un décalage du cadre de lecture. Ceci a pour conséquence l’absence de synthèse d’une dystrophine fonctionnelle, protéine impliquée dans l’intégrité structurale des fibres musculaires. A ce jour, il n’existe aucun traitement curatif et les patients meurent précocement par insuffisance cardiorespiratoire vers l’âge de 30 ans.
Dans le monde animal, une maladie musculaire comparable est la myopathie dystrophique liée au chromosome X. D’après la littérature, de nombreuses races canines sont prédisposées par la maladie pour lesquelles différentes mutations sur le gène de la dystrophine ont été répertoriées. Ces pathologies, cliniquement et histologiquement similaires, font du modèle canin le modèle animal le plus proche de l’enfant dystrophique pour évaluer l’efficacité thérapeutique de la maladie de Duchenne, maladie génétique la plus répandue chez l’homme.
En septembre 2018, des chercheurs ont appliqué pour la première fois le système CRISPR/Cas9 chez quatre chiens dystrophiques par l’administration de virus adéno-associés AAV9. Des ARNgs ciblant l’exon 51 ont été utilisés pour restaurer le cadre de lecture du gène de la dystrophine chez des chiens dystrophiques. Les résultats ont mis en évidence une augmentation de la production de dystrophine dans l’ensemble des muscles, y compris dans le cœur, chez les chiens ayant reçu une injection intraveineuse. Bien que cette étude soit encourageante, d’autres travaux incluant une population plus large sur une plus longue durée devront être réalisés afin d’évaluer l’efficacité et l’innocuité de ce système pour permettre une éventuelle application chez l’enfant dystrophique.

CRISPR/Cas9 --- maladie de Duchenne --- modèle canin --- Sciences du vivant > Génétique & processus génétiques

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This eBook is a collection of articles from a Frontiers Research Topic. Frontiers Research Topics are very popular trademarks of the Frontiers Journals Series: they are collections of at least ten articles, all centered on a particular subject. With their unique mix of varied contributions from Original Research to Review Articles, Frontiers Research Topics unify the most influential researchers, the latest key findings and historical advances in a hot research area! Find out more on how to host your own Frontiers Research Topic or contribute to one as an author by contacting the Frontiers Editorial Office: frontiersin.org/about/contact

Trypanosoma cruzi --- Chagas disease --- parasite-host interaction --- gene function --- CRISPR/Cas9 technique

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Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs. The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult. Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology.

Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells

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Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs. The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult. Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology.

Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells