Titlebook: Genome Editing; The Next Step in Gen Toni Cathomen,Matthew Hirsch,Matthew Porteus Book 2016 The Editor(s) (if applicable) and The Author(s)

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Engineered Nucleases and Trinucleotide Repeat Diseases,late gene function and heritable traits in a number of species including human. Mutations at a subset of repeats, most of which are trinucleotide repeats, trigger devastating human neurological and skeletal disorders. In particular, at least a dozen neurological disorders share a common etiology—the

Harness

Using Engineered Nucleases to Create HIV-Resistant Cells, treatments, which require life-long adherence to antiretroviral drug regimens. Engineered nucleases have the capability to either disrupt a specific gene, or to promote precise gene edits or additions at the targeted gene. As one application for the gene disruption capabilities of the nucleases, HI

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Strategies to Determine Off-Target Effects of Engineered Nucleases,e-induced DNA breaks by either non-homologous end joining (NHEJ) or homology-directed repair (HDR) allows genome editing in a wide range of organisms and cell lines. However, if a nuclease cleaves at genomic locations other than the intended target, known as “off-target sites”, it can lead to mutati

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https://doi.org/10.1057/9780230504905thies or neuropathies are discussed. The genome editing tools available to create targeted genetic modifications are reviewed. Promising cell- and gene-based therapies are introduced in the context of the treatment of neuromuscular disorders in combination with genome editing therapies. Finally, spe

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https://doi.org/10.1007/978-3-031-46297-9rized here, from creation of transgenic organisms and in vivo gene therapy, to cellular reprogramming and precise genome editing by cassette exchange. The latest system, dual integrase cassette exchange (DICE), uses target phiC31 and Bxb1 . sequences precisely placed in genomes by homologous recombi