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Talen based gene correction for epidermolysis bullosa pdf

 
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MessagePosté le: Ven 1 Déc - 05:16 (2017)    Sujet du message: Talen based gene correction for epidermolysis bullosa pdf Répondre en citant

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Deep sequencing-based genome-wide screening established a safety profile showing on-target activity and three off-target (OT) loci that, importantly, were at least 10 kb from a coding sequence. This study provides proof-of-concept for TALEN-mediated in situ correction of an endogenous patient-specific gene mutation and
17 May 2016 CRISPR/Cas | TALENs | epidermolysis bullosa | gene editing | . Cas9- and TALEN-mediated gene editing via NHEJ, we trans- targeted only the mutant sequence of COL7A1. Based on these experiments, we used CRISPR/Cas9 for further mutation site-spe- cific gene editing experiments in our DDEB
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2 Apr 2013 Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1). Gene augmentation therapies are promising, but run the risk of insertional mutagenesis. To abrogate this risk, we explored the
6 May 2014 The PDF of this article can be found at: f1000.com/reports/m/6/35/pdf . (A) Diagram of TALEN targeting COL7A1 mutation g.1837 C>T, which leads to a premature stop codon. . 22, Nystrom A, Bruckner-Tuderman L, Kern JS: Cell- and protein-based therapy approaches for epidermolysis bullosa.
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Transcription activator-like effector nucleases (TALEN) are restriction enzymes that can be engineered to cut specific sequences of DNA. They are made by fusing a TAL effector DNA-binding domain to a DNA cleavage domain (a nuclease which cuts DNA strands). Transcription activator-like effectors (TALEs) can be
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Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by a functional deficit of type VII collagen protein due to gene defects in the type VII collagen gene (COL7A1). Gene augmentation therapies are promising, but run the risk of insertional mutagenesis. To abrogate this risk, we explored the possibility of
11 May 2017 A rare hereditary mechanobullous disorder called epidermolysis bullosa (EB) causes blistering in the skin and the mucosal membranes. To date, nineteen EB-related genes have been discovered in human and other species. We describe here a novel EB variant in dogs. Two newborn littermates of Central

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