{"product_id":"genetic-knockout-of-cathepsin-d-using-zinc-finger-nucleases-delivered-by-aav-vectors-von-maryna-psol","title":"Genetic knockout of Cathepsin D using zinc-finger nucleases delivered by AAV vectors","description":"\u003cp\u003eProject Report from the year 2013 in the subject Biology - Neurobiology, grade: 1.0, University of Göttingen, language: English, abstract: Genetic engineering is known as a powerful technique for basic research and clinical applications. Recent progress in development of zinc-finger nucleases (ZFNs), which combine the DNA cleavage ability of Fok1 restriction enzyme with highly specific recognition properties of zinc-finger motifs, allows to improve efficiency and to broaden the field of use of genome editing. Here, we demonstrate our initial results in generating novel tools for Cathepsin D gene knockout in neurons based on ZFNs technology and mediated by adeno-associated virus (AAV) vectors. Pairs of AAV-ZFNs were produced and demonstrated the robust expression of nucleases in neuronal cell culture. Observed toxicity most likely was associated with heterodimerization but not homodimerization of ZFNs; cytotoxicity was greatly reduced when ZFN were provided at lower concentrations. Future studies evaluating efficiency of Ctsd knockout, off-target effects on molecular level and long-term outcomes in vivo can be performed.\u003c\/p\u003e\u003cdiv class=\"aw-variant-hidden-subtitle-div\" id=\"aw-variant-subtitle-9783656525035\"\u003e\u003ch3\u003e\u003c\/h3\u003e\u003c\/div\u003e","brand":"Libri","offers":[{"title":"Softcover - 9783656525035","offer_id":39446965846109,"sku":"9783656525035","price":17.95,"currency_code":"EUR","in_stock":true}],"thumbnail_url":"\/\/cdn.shopify.com\/s\/files\/1\/0940\/0622\/files\/12dab521-23cd-4cc2-8a10-b059ec10883b.jpg?v=1778302710","url":"https:\/\/shop.autorenwelt.de\/products\/genetic-knockout-of-cathepsin-d-using-zinc-finger-nucleases-delivered-by-aav-vectors-von-maryna-psol","provider":"Autorenwelt Shop","version":"1.0","type":"link"}