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Mechamism Studies Of Targeted Gene Repair Mediated By Single-Stranded Oligonucleotides

Posted on:2004-01-25Degree:DoctorType:Dissertation
Country:ChinaCandidate:X S WuFull Text:PDF
GTID:1100360185473354Subject:Biochemistry and Molecular Biology
Abstract/Summary:PDF Full Text Request
Diseases are caused by disorders of gene structure or gene expression in essence. Currently, gene therapy has become the most potential treatment in biomedical field, following the development in recognition of molecular process of diseases and technologies in molecular biological field. The ideal process of gene therapy contains the transferring effective vectors into target cells, vectors'specific integration into chromosome DNA, and controllable expression of target genes. Couples of strategies, including gene addition mediated by virol vectors, gene replacement supported by homologous recombination, and nucleic acid therapeutics, were established for these purposes. Among them, a newly developing technology, called targeted gene repair mediated by oligonucleotides, is a better way for genetic disorders than traditional strategies. It can repair the mistaken site at the same position in the chromosome, and so it maintains the recovered gene expression in the natural context. Moreover, because the synthesized oligonucleotides are free from immunity, they can be administered several times as normal drugs.At present time, studies about targeted gene repair are carried out at multi-levels, which include bacteria, fungi, plants and animals. But investigators found that oligonucleotide's action was not stable. The results couldn't be repeated precisely. Because of unknowing the mechanism, people could hardly improve the frequency of gene repair. So it is essential to reveal the mechamism of targeted gene repair mediated by oligonucleotides.For reasons mentioned above, we developed fluorescent reporter system in...
Keywords/Search Tags:chimera, RDO, gene therapy, targeted gene repair
PDF Full Text Request
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