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Related Concept Videos

CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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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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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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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: Jan 23, 2026

CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
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Editing the Central Nervous System Through CRISPR/Cas9 Systems.

Agustin Cota-Coronado1, Néstor Fabián Díaz-Martínez2, Eduardo Padilla-Camberos1

  • 1Biotecnología Médica y Farmacéutica CONACYT, Centro de Investigación y Asistencia en Tecnología y Diseño del Estado de Jalisco (CIATEJ), Guadalajara, Mexico.

Frontiers in Molecular Neuroscience
|June 14, 2019
PubMed
Summary

Gene editing tools like CRISPR/Cas9 are advancing gene therapy for neurodegenerative diseases. This review explores systems engineered to cross the blood-brain barrier for treating Parkinson's and Alzheimer's disease.

Keywords:
CRISPR/Cas9adenovirus-associated virusblood–brain barriercentral nervous systemd-Cas9“Trojan horse” peptides

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Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Gene therapy offers promising treatments for neurodegenerative diseases.
  • Advancements in gene editing tools, including CRISPR/Cas9, have narrowed the translational gap.
  • Targeting the central nervous system (CNS) presents unique challenges due to the blood-brain barrier (BBB).

Purpose of the Study:

  • To review current strategies for engineering CRISPR/Cas9 systems capable of crossing the BBB.
  • To explore the clinical potential of these systems for treating neurodegenerative conditions.
  • To highlight advancements in gene therapy for Parkinson's disease and Alzheimer's disease (AD).

Main Methods:

  • Review of existing literature on CRISPR/Cas9 delivery systems.
  • Analysis of methods designed to overcome the BBB.
  • Evaluation of studies focusing on neurodegenerative disease models.

Main Results:

  • CRISPR/Cas9 technology enables precise gene editing for potential therapeutic applications.
  • Several strategies are being developed to enhance CNS penetration of gene editing complexes.
  • Engineered systems show promise for targeting neurodegeneration in preclinical models.

Conclusions:

  • Improved gene editing tools are facilitating gene therapy for CNS disorders.
  • Overcoming the BBB is critical for effective gene therapy in neurodegenerative diseases.
  • Future clinical applications of CRISPR/Cas9 hold potential for treating Parkinson's and Alzheimer's disease.