関連する実験動画

Updated: Jun 20, 2025

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
07:44

CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

Published on: September 14, 2019

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スプリットインテイン媒介タンパク質のトランススプライシングにより,大型のディストロフィンが発現する

Hichem Tasfaout1,2, Christine L Halbert3,4, Timothy S McMillen5

  • 1Department of Neurology, University of Washington School of Medicine, Seattle, WA, USA. tasfaout@uw.edu.

Nature
|July 17, 2024
PubMed
まとめ

アデノ関連ウイルス (AAV) のベクトルを用いたデュシェンヌ筋縮症の遺伝子治療は,現在,大型のディストロフィンを発現させることができる. このスプリット・インテイン・メソッドは,AAV包装の限界を克服し,マウスのマイクロディストロフィンよりも機能的優位性を示しています.

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RNA Splicing01:32

RNA Splicing

Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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Alternative RNA Splicing02:18

Alternative RNA Splicing

Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
There are five types of alternative RNA splicing that vary in the ways the pre-mRNA segments are removed or retained in the mature mRNA. The first...
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Tagging and Fusion Proteins

Proteins are involved in several cellular processes and biochemical reactions. Analyzing a specific protein of interest requires it to be isolated from the other proteins in the cell. This is achieved by overexpressing the specific gene in a suitable host to produce large quantities of the target protein. A tag or label is recombined with the gene to produce a fusion protein containing the target protein and the tag. The tags on these fusion proteins can then be used for easy detection and...
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