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Related Concept Videos

CRISPR01:59

CRISPR

55.6K
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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RNA Editing02:23

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Updated: Nov 30, 2025

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
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Genome Editing for CNS Disorders.

Fábio Duarte1,2, Nicole Déglon1,2

  • 1Laboratory of Neurotherapies and NeuroModulation, Department of Clinical Neurosciences, Lausanne University Hospital and Lausanne University, Lausanne, Switzerland.

Frontiers in Neuroscience
|November 16, 2020
PubMed
Summary
This summary is machine-generated.

Gene editing offers promising therapies for central nervous system (CNS) disorders by correcting genetic defects. This review covers genome editing tools and preclinical CNS studies, addressing current challenges.

Keywords:
CNSCRISPR/CasTALEsZFsgenome editing

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Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Central nervous system (CNS) disorders pose significant societal and economic challenges.
  • Effective therapeutic strategies for CNS diseases are urgently needed.
  • Gene editing presents a potential curative approach by modifying endogenous genetic loci.

Purpose of the Study:

  • To review available genome editing tools for CNS applications.
  • To summarize preclinical in vivo studies of CNS genome editing.
  • To discuss limitations and alternative strategies in CNS genome editing.

Main Methods:

  • Review of scientific literature on genome editing technologies (ZFs, TALEs, CRISPR/Cas).
  • Analysis of preclinical in vivo studies focusing on CNS gene editing.
  • Evaluation of delivery systems for CNS access.

Main Results:

  • Emerging technologies like CRISPR/Cas have advanced genome manipulation for genetic diseases.
  • Efficient delivery systems are crucial for accessing the CNS.
  • Preclinical studies demonstrate the potential of genome editing in CNS disorders.

Conclusions:

  • Genome editing tools and delivery systems show promise for treating CNS disorders.
  • Overcoming current limitations is key to advancing clinical applications.
  • Further research into alternative approaches is warranted.