Get Genome Editing: The Next Step in Gene Therapy PDF

By Toni Cathomen, Matthew Hirsch, Matthew Porteus

ISBN-10: 1493935070

ISBN-13: 9781493935079

ISBN-10: 1493935097

ISBN-13: 9781493935093

This accomplished quantity explores human genetic engineering its pre-clinical and medical purposes, present advancements, and as therapy for hereditary ailments. It offers and evaluates the newest advances within the knowing of mammalian host DNA fix mechanisms, reminiscent of double-strand holiday caused gene focusing on and mutagenesis, the improvement of zinc-finger nucleases, genome modifying for neuromuscular illnesses, section integrases, triplex forming oligonucleotides and peptide nucleic acids, aptamer-guided gene concentrating on, AAV gene modifying through DSB fix, engineered nucleases and trinucleotide repeat ailments, and production of HIV-resistant cells. The expertly authored chapters contextualize present advancements in the historical past of genome enhancing whereas additionally discussing the present and power safeguard matters of this quickly becoming field.

Genome modifying: the next move in Gene Therapy, the newest quantity within the American Society of Gene and cellphone treatment series, deftly illuminates the opportunity of genetic engineering expertise to eliminate today’s deadliest and so much prolific illnesses. it's perfect studying for clinicians and researchers in genetics and immunology.

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Extra resources for Genome Editing: The Next Step in Gene Therapy

Example text

TALEs are composed of N-terminal secretion and translocation signals, a central domain with DNA binding capability and a C-terminal acidic activator domain coupled to nuclear localization signals that enable translocation into nucleus [2] (Fig. 1). Protein engineering strategies have been especially focused on the identification of N- or C-terminal truncations aimed at creating artificial TALE-based DNA binding domains that combine minimal size with efficient DNA binding activity. In the next paragraphs we will describe and summarize the development of different TALE scaffolds that have been engineered as versatile carriers for various effector domains.

The first ZFNs for a genomic target displayed significant toxicity when expressed at high levels [38], due to promiscuous binding and cleavage [39], and this issue has continued to trouble many new designs. Increasing the number of fingers in each monomer is one approach to ameliorating this effect, but that is not always sufficient [40, 41]. The toxicity is frequently a property of one ZFN within a pair, and it appears to be due to inadequate specificity, leading to homodimerization and cleavage at unintended, off-target sites [39].

The latter approach seems to work because of the high intrinsic positive charge on the ZFNs, and it is applicable to a range of cell types, albeit with variable efficiencies. Long, doublestranded donor DNAs can be introduced on plasmid or viral vectors, while short, single-stranded donors are usually simply added to the medium [54, 55, 58, 59]. For situations in which manipulation of whole organisms is the goal, genome alterations in the germ line must be achieved. The cells in which the germ line is most accessible are typically in the very early embryo.

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Genome Editing: The Next Step in Gene Therapy by Toni Cathomen, Matthew Hirsch, Matthew Porteus


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