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Titlebook: Genome Editing in Neurosciences; Rudolf Jaenisch,Feng Zhang,Fred Gage Book‘‘‘‘‘‘‘‘ 2017 The Editor(s) (if applicable) and The Author(s) 20

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https://doi.org/10.1007/978-1-349-20987-3 gene function via tissue- or cell-specific mutagenesis remains challenging in zebrafish when the study of the function of certain loci might require tight spatiotemporal control of gene inactivation, which is particularly true in studying the function of a particular gene in post mitotic neurons, w
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https://doi.org/10.1007/978-3-8349-6807-4ogenic iPSC lines were created. We found that the disease phenotypes only manifested in the differentiated neural stem cell (NSC) stage, not in iPSCs. Transcriptomic analysis of HD iPSCs and HD NSCs compared to isogenic controls was utilized to understand the molecular basis for the CAG repeat expan
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Determinanten der Bildungsungleichheitrated transgenic marmosets with germ line transmission, opening new avenues in primate research..In this chapter, we describe recent advances in neuroscience and disease research using common marmosets, and we outline potential uses of genome editing in non-human primates toward the development of knock-in/knock-out marmosets.
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,Grundzüge der Kapitalstrukturtheorie,g or mutation-adjacent . exons are intentionally removed in order to restore protein reading frame, and thereby Dystrophin expression, in DMD patients (Beroud et al. Hum Mutat 28:196–202, 2007; Yokota et al. Expert Opin Biol Ther 7:831–842, 2007).
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Dissecting the Role of Synaptic Proteins with CRISPR,chnique in addition to previous genetic approaches vastly simplifies and accelerates the study of specific synaptic proteins. Here we illustrate different ways that CRISPR/Cas9 can be used in the study of synaptic properties.
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Therapeutic Gene Editing in Muscles and Muscle Stem Cells,g or mutation-adjacent . exons are intentionally removed in order to restore protein reading frame, and thereby Dystrophin expression, in DMD patients (Beroud et al. Hum Mutat 28:196–202, 2007; Yokota et al. Expert Opin Biol Ther 7:831–842, 2007).
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