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Related Concept Videos

CRISPR01:59

CRISPR

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 Short...
CRISPR01:59

CRISPR

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 Short...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview

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Related Experiment Video

Updated: Jun 12, 2026

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

CRISPR-Based Gene Therapy for Brain Disease.

Fatemeh Khajouei1,2,3, Asma Ghaemi1, Khalil Abnous1

  • 1Pharmaceutical Research Center, Pharmaceutical Technology Institute, Mashhad University of Medical Sciences, Mashhad, Iran.

Molecular Neurobiology
|June 10, 2026
PubMed
Summary

CRISPR-Cas9 gene therapy offers a promising approach for neurological disorders by enabling precise gene editing. However, challenges like blood-brain barrier crossing and off-target effects need addressing for effective clinical application.

Keywords:
CRISPR-Cas9Gene editingNeurodegenerative disordersNeurological disorders

More Related Videos

CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer
09:39

CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer

Published on: June 9, 2018

Related Experiment Videos

Last Updated: Jun 12, 2026

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
09:00

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice

Published on: August 2, 2018

CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer
09:39

CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer

Published on: June 9, 2018

Area of Science:

  • Neuroscience
  • Genetics
  • Biotechnology

Background:

  • Neurological disorders are complex, often stemming from genetic mutations, with current treatments primarily managing symptoms.
  • Existing therapies for brain diseases necessitate more effective strategies beyond symptom alleviation.
  • Gene therapy, particularly CRISPR-Cas9 technology, presents a novel therapeutic avenue for genetic neurological conditions.

Purpose of the Study:

  • To review the application of CRISPR-Cas9 gene therapy in treating various neurological diseases.
  • To highlight the potential of CRISPR-Cas9 for addressing the genetic underpinnings of brain disorders.
  • To identify and discuss the challenges associated with clinical implementation of CRISPR-based therapies for neurological conditions.

Main Methods:

  • Literature review focusing on CRISPR-Cas9 applications in neurological disease research.
  • Analysis of studies investigating gene editing for conditions such as Alzheimer's, Parkinson's, epilepsy, stroke, and brain tumors.
  • Examination of challenges related to CRISPR-Cas9 delivery across the blood-brain barrier and potential side effects.

Main Results:

  • CRISPR-Cas9 technology demonstrates potential for precise gene editing in diverse cell types relevant to neurological disorders.
  • The review covers applications in multiple sclerosis, Alzheimer's, Parkinson's disease, epilepsy, stroke, and brain tumors.
  • Significant hurdles in clinical translation include overcoming the blood-brain barrier, managing off-target mutations, and ensuring safety.

Conclusions:

  • CRISPR-Cas9 gene therapy holds significant promise for treating a range of neurological disorders at their genetic source.
  • Successful clinical translation requires overcoming substantial technical and safety challenges, including delivery and immunogenicity.
  • Further research is essential to refine CRISPR-Cas9 technology for safe and effective neurological disease treatment.