Titlebook: Gene Correction; Methods and Protocol Francesca Storici Book 2014 Springer Science+Business Media, LLC 2014 gene correction.gene repair.gen

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https://doi.org/10.1007/978-3-0348-9381-7specific integrase mediated repeated targeting (SIRT) method, which combines homologous recombination, site-specific integration, and bacterial recombineering to conduct targeted modifications of individual loci. Here we describe the recombineering designs and procedures for the introduction of epit

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https://doi.org/10.1007/978-1-349-06540-0of human development, disease pathogenesis, and the generation of screening systems to identify novel therapeutic agents. Autologous cell therapies based on patient-derived induced pluripotent stem cells also hold great promise for the treatment and correction of many inherited and acquired diseases

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Reflections on Research in Chemotherapy,ication for the treatment of many disorders. Among those, Duchenne muscular dystrophy (DMD) represents an ideal candidate for gene editing primarily due to the large size of dystrophin, the gene responsible for the disease, which limits the use of gene replacement approaches. Critical in the evaluat

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T. A. Khwaja,T. C. Hall,K. M. A. Sheikhctive approach for gene therapy. Small DNA fragments (SDFs) were used in SFHR to modify endogenous genomic DNA in both human and murine cells. The advantage of this gene targeting approach is to maintain the physiologic expression pattern of targeted genes without altering the regulatory sequences (

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Pharmacokinetics of Anticancer Drugs,ing a specific target sequence in vivo, driving that site into an exploitable repair pathway. Information is transferred to the site via participation in the pathway by the second component, a donor nucleic acid, resulting in a permanent change in the target sequence. We have developed biologically

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Bruce A. Chabner,David G. Johnsnous recombination machinery. The former includes a repertoire of sequence-specific binding molecules for targeted induction and appropriation of this machinery, such as by triplex-forming oligonucleotides (TFOs) or triplex-forming peptide nucleic acids (PNAs) and recombinagenic donor DNA, respectiv

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