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 4 of 4 for “"in vivo gene delivery"”.
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Tissue-engineered liver microreactor as an in vitro surrogate assay for gene delivery
The lack of correlation between in vitro and in vivo gene delivery experiments presents a significant obstacle in the progress of gene therapy studies by preventing the extrapolation of successful cell culture results into animals. This phenomenon has also been documented in the specific case of …
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Gene delivery with amphiphilic lower generation polypropylenimine dendrimer
In this work a novel lower generation amphiphilic polypropylenimine (PPI) dendrimer with good aqueous solubility was developed and tested as a gene delivery agent. PPI dendrimer generation 3 (DAB-16-Am) was substituted with a low level of cetyl chains (less than 5 molar percent) by reaction with …
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Effect of uptake pathway on non-viral gene delivery in vitro and in vivo
Efficient non-viral gene delivery often involves the conjugation of a cell-specific ligand to the vector, which directs the vector to its intended target through binding to a cellular receptor followed by internalization via endocytosis. However, little is known in terms of how the various …
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Genetic Correction of Duchenne Muscular Dystrophy using Engineered Nucleases
… hereditary disorder caused by a loss of dystrophin, an essential musculoskeletal protein. Decades of promising research have yielded only modest gains in survival and quality of life for these patients and there have been no approved gene therapies for DMD to date. There are two significant …