Titlebook: Viral Vectors for Gene Therapy; Methods and Protocol Fredric P. Manfredsson,Matthew J. Benskey Book 2019 Springer Science+Business Media, L

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Design, Construction, and Application of Transcription Activation-Like Effectorsquences following a simple cipher. Customized TALE proteins can be used in a variety of molecular applications that include gene editing and transcriptional modulation. Presently, we provide a brief primer on the design and construction of TALEs. TALE proteins can be fused to a variety of different

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AAV Production Using Baculovirus Expression Vector Systemic or cancer diseases. The most efficient gene transfer tools are currently derived from viruses. Among them, the recombinant adeno-associated viruses (AAVs) are vectors of choice for many fundamental and therapeutic applications. The increasing number of clinical trials involving AAVs demonstrates

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Multimodal Production of Adeno-Associated Viruscelerated pace. Nevertheless, despite its popularity, AAV is a relatively cumbersome virus to produce; however, significant efforts have been invested to develop, optimize, and simplify methodology that allows the generation of high-quality AAV with significantly increased production yields. Here we

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A Scalable Lentiviral Vector Production and Purification Method Using Mustang Q Chromatography and Ttions in the genome. This status can be attributed primarily to their ability to transduce dividing as well as quiescent cells. When coupled with internal promotor selection to drive expression in one cell type but not another, the ease with which the vectors can be pseudotyped to either restrict or

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Construction of Oncolytic Herpes Simplex Virus with Therapeutic Genes of InterestC) has been accelerating translational research of oncolytic HSV (oHSV) as a promising therapeutic for refractory cancers such as glioblastoma, the deadliest primary malignancy in the brain. The large genome size of HSV readily allows arming of oHSV by incorporating therapeutic transgenes within the

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Poxviruses as Gene Therapy Vectors: Generating Poxviral Vectors Expressing Therapeutic Transgenes and for vaccine development. More importantly, the oncolytic properties of poxviruses have led to their development as cancer therapeutics. Two poxviruses, vaccinia virus (VACV) and myxoma virus (MYXV), have been extensively studied as virotherapeutics with promising results. Vaccinia virus vectors

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AAV-Mediated Gene Delivery to the Mouse Liverrently the most popular gene delivery system for targeting the liver, reflecting high transduction efficiency in vivo and the availability of a toolkit of multiple different capsids with high liver tropism. While AAV vectors confer stable gene transfer in the relatively quiescent adult liver, the pr