Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
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Showing 1 to 10 of 10 for “"Gene correction"”.
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Sequence-Specific Gene Correction of Cystic Fibrosis Airway Basal Cells
… resulting from mutations in the <em>CFTR </em>gene which encodes a protein involved in regulating anion trans-epithelial transport. A three-base deletion in <em>CFTR </em>(termed as ΔF508 mutation), wherein CFTR protein is misfolded leading to its pre-mature degradation in the endoplasmic …
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Ipsc Based Gene Correction and Disease Model of A New Class of Lgmd Due to Poglut1 Mutation
… and rescue experiments. Using a CRISPR based gene targeting method, we aimed to correct the point mutation and restore POGLUT1 function, thus restoring Notch signaling activity. Following correction, iPSC-derived gene corrected myogenic cells were differentiated and compared to healthy control …
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Site-Specific Genome Engineering in Mouse Primary Fibroblasts
… can dramatically stimulate the frequency of gene targeting at a given locus. Recently, there has been an outpouring of studies performing site-specific genome engineering in human cell lines and primary cells, including embryonic stem cells, induced pluripotent stem cells and CD34+ …
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Site-Specific Genome Engineering in Mouse Primary Fibroblasts
… can dramatically stimulate the frequency of gene targeting at a given locus. Recently, there has been an outpouring of studies performing site-specific genome engineering in human cell lines and primary cells, including embryonic stem cells, induced pluripotent stem cells and CD34+ …
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Correction of Cystic Fibrosis-Specific Induced Pluripotent Stem Cells
… advances in reprogramming and the ability to genetically modify the resulting induced pluripotent stem cells (iPSCs) have provided an alternative to conventional gene therapies thus far attempted for CF. Here we describe the <em>in vitro</em> generation of iPSCs starting from fibroblast cells …
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Protein Engineering via in Vitro Coevolution
… potential for applications such as targeted gene correction in gene therapy and gene alteration in systems biology and metabolic engineering. However, the limited repertoire of recognition sequences in naturally available homing endonucleases severely hampers their usefulness. I have …
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Modelling and correction of retinal pathologies caused by mitochondrial DNA mutations via genetic engineering
… to progressive visual loss. Although advances in genetic engineering have enabled the modelling and correction of nuclear DNA mutations in the retina, challenges remain in modifying mtDNA. The generation of accurate models that mimic retinal mtDNA diseases and development of curative gene …
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Genetic Correction of Duchenne Muscular Dystrophy using Engineered Nucleases
… these patients and there have been no approved gene therapies for DMD to date. There are two significant hurdles to creating effective gene therapies for DMD; it is difficult to deliver a replacement dystrophin gene due to its large size and current strategies to restore the native dystrophin …
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Engineering of transcription activator-like effector nucleases (TALENs) for targeted genome editing
… is to edit or program genomic sequences and to generate desired phenotypes. Although virus-based strategies have long been developed to for efficient gene insertion, the random or semi-random integration can disrupt certain endogenous genes and cause unpredictable phenotypes. In contrast, …
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CORRECTION OF THE GENETIC DEFECT IN INDUCED PLURIPOTENT STEM CELL LINES THROUGH CHROMOSOME TRANSPLANTATION
… and biomedical advancements, such as regenerative medicine, disease modelling, and drug screening. A pivotal milestone in this field was the generation of induced pluripotent stem cells (iPSCs) by Yamanaka and Takahashi in 2006, through the use of four reprogramming factors (Oct4, Sox2, …