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Related Concept Videos

Alternative RNA Splicing02:18

Alternative RNA Splicing

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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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RNA Splicing01:32

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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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CRISPR01:59

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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CRISPR and crRNAs02:53

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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What is Gene Expression?01:42

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Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
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Functional Assessment of BRCA1 variants using CRISPR-Mediated Base Editors
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CRISPR-SKIP: programmable gene splicing with single base editors.

Michael Gapinske1, Alan Luu2,3, Jackson Winter1

  • 1Department of Bioengineering, University of Illinois at Urbana-Champaign, 1406 West Green Street, Urbana, 61801-2918, IL, USA.

Genome Biology
|August 16, 2018
PubMed
Summary
This summary is machine-generated.

CRISPR-SKIP precisely programs exon skipping by mutating DNA bases in splice sites. This versatile gene editing tool offers enhanced precision for gene therapy and synthetic biology applications.

Keywords:
Alternative splicingBRCA2Base editingCRISPR-Cas9Exon skippingGene editingGene isoformPIK3CARELASynthetic biology

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • CRISPR gene editing enables targeted DNA modification but can lead to off-target mutations.
  • Cellular DNA repair mechanisms introduce variability in gene editing outcomes.
  • High-precision gene editing technologies are crucial for advanced applications.

Purpose of the Study:

  • To develop a precise gene editing method for programming exon skipping.
  • To leverage single-base editors for enhanced accuracy in genetic engineering.
  • To introduce a versatile tool for gene therapy and synthetic biology.

Main Methods:

  • Utilized cytidine deaminase single-base editors.
  • Developed a method termed CRISPR-SKIP.
  • Targeted DNA bases within splice acceptor sites to induce mutations.
  • Programmed exon skipping through targeted DNA base modification.

Main Results:

  • CRISPR-SKIP enables precise programming of exon skipping.
  • The method demonstrates high precision by targeting specific DNA bases.
  • Successfully mutated target DNA bases within splice acceptor sites.
  • Validated the versatility of CRISPR-SKIP for genetic engineering.

Conclusions:

  • CRISPR-SKIP offers a simple and precise method for exon skipping.
  • The technology has broad applicability in gene therapy.
  • CRISPR-SKIP is a valuable tool for synthetic biology advancements.