シングル siRNA ナノカプセルは,強化されたRNAi配送のためのものです
Ming Yan1, Min Liang, Jing Wen
1Department of Microbiology, Immunology, and Molecular Genetics, and California NanoSystems Institute (CNSI), University of California, Los Angeles, California 90095, USA.
Journal of the American Chemical Society
|August 8, 2012
まとめ
この研究は,単一の小型の干渉RNA (siRNA) 分子を運ぶための新しいナノカプセルを導入し,疾患の治療における潜在的な治療応用のための安定性と細胞吸収を強化します.
科学分野:
- バイオテクノロジー バイオテクノロジー
- ナノテクノロジー ナノテクノロジー
- 分子生物学は分子生物学である.
背景:
- 合成小干渉RNA (siRNA) は治療効果が有望だが,安定性と発送において課題に直面している.
- 標的組織と細胞にRNA干渉 (RNAi) 化合物を効果的に投与することは,臨床トランスレーションの重要な障害となっています.
研究 の 目的:
- シングル siRNA 分子のための新しいナノカプセルベースの配送システムを開発し,検証する.
- CCR5をHIV治療のモデルとして使用して,特定の遺伝子標的をダウンレギュレーションするこのシステムの有効性を実証する.
主な方法:
- 単一のsiRNA分子を分解可能なポリマーナノカプセル内に封じ込め (約. 直径20nm,陽性表面電荷).
- CCR5-siRNAナノカプセルを293T細胞に投与し,CCR5RNAのダウンレギュレーションを評価する.
- RNaseとヒト血清の存在下におけるナノカプセルの安定性とsiRNAの完全性の評価.
- 自由 siRNA とリポフェクトアミン媒介配送による遺伝子静止効率の比較.
主要な成果:
- ナノカプセルは,CCR5-siRNAを投与し,293T細胞におけるCCR5RNA発現を8%まで低調させることに成功した.
- ナノカプセルは,siRNAをRNaseと血清による分解から保護し,1時間間の完全性を維持しました.
- 血清では,ナノカプセルで発送されたsiRNAは<15%のCCR5-mCherry発現を達成し,リポフェクトアミン (55%) を大幅に上回った.
結論:
- 新しい単一のsiRNAナノカプセル配送プラットフォームが開発されました.
- この技術は,安定性の向上と効果的な遺伝子サイレンシングを提供し,現在のsiRNA治療法の主要な制限に対処します.
- ナノカプセルプラットフォームは,様々なヒト疾患における治療開発の可能性を秘めています.
関連する概念動画
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
01:23RNA Interference
RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
This process occurs naturally in cells, often through the activity of genomically-encoded microRNAs. Researchers can take advantage of this mechanism by introducing synthetic RNAs to deactivate specific genes for research or therapeutic purposes. For example, RNAi could be used...
02:30siRNA - Small Interfering RNAs
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
02:15Experimental RNAi
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
02:30Small interfering RNAs (siRNA)
Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...
In the cytoplasm, siRNA is processed from a double-stranded RNA, which comes from either endogenous DNA transcription or exogenous sources like a virus. This double-stranded RNA is then cleaved by the ATP-dependent...