Structural modification of small interfering ribonucleic acids for efficient gene delivery = 효율적인 유전자 전달을 위한 소간섭 리보핵산의 구조적 변형에 관한 연구

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Gene silencing by RNA interference (RNAi) was discovered in C. elegans in 1998 as a response to long double-stranded (ds) RNA. The RNAi effect has been successfully extended to plants, Drosophila, and fungi. However, the introduction of long dsRNA into mammalian cells activates antiviral defense responses and nonspecific gene silencing. In 2001, RNAi-mediated gene silencing in mammalian cells was first reported by short, synthetic dsRNA, known as small interfering RNA (siRNA), which opened the door to RNAi technology for gene therapy. The synthetic siRNA has 19 ~ 21 base pairs with a 2-nucleotide overhang at both 3`-ends. The ability to easily design and customize siRNA sequences for any genes has made it more attractive for siRNA therapeutics. An siRNA is incorporated and unwound in RNA-induced silencing complex (RISC), and retains one strand of the siRNA duplex in cells. The RISC encapsulating a single strand siRNA guides the endonucleolytic cleavage of complementary mRNA, resulting in sequence-specific gene silencing. To achieve siRNA therapy, it is essential to transport exogenous siRNA into the cytoplasm of target cells due to endogenous RNAi pathway. To this end, viral vectors have been used for siRNA delivery owing to their ability to efficiently attach and transport own genomic materials into cells. They have shown high transfection efficiency. However, their clinical application has been restricted by the potential risks such as mutagenicity, inflammatory, and immunogenicity. These in-creasing concerns have led to the development of synthetic non-viral vectors. A variety of synthetic vectors based on cationic polymers, peptides, and lipids have been studied to form cationic nanocomplexes with poly-anionic nucleic acids by electrostatic interactions, which can facilitate cellular uptake through endocytosis. In general, plasmid DNA can be effectively condensed into stable complexes with sizes of approximately 200 nm upon contact with cationic carriers. ...
Advisors
Nam, Yoon-Sungresearcher남윤성
Description
한국과학기술원 : 생명과학과,
Publisher
한국과학기술원
Issue Date
2013
Identifier
513589/325007  / 020098106
Language
eng
Description

학위논문(박사) - 한국과학기술원 : 생명과학과, 2013.2, [ xi, 110 p. ]

Keywords

Small interfering RNA (siRNA); RNA interference (RNAi); Engineered siRNA structures; siRNA delivery; 소간섭 리보핵산; 리보핵산 간섭; 소간섭 리보핵산 구조변형체; 소간섭 리보핵산 전달; 나노복합체; Nanocomplex

URI
http://hdl.handle.net/10203/179808
Link
http://library.kaist.ac.kr/search/detail/view.do?bibCtrlNo=513589&flag=dissertation
Appears in Collection
BS-Theses_Ph.D.(박사논문)
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