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Related Concept Videos

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...

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Related Experiment Video

Updated: May 27, 2026

Direct Injection of a Lentiviral Vector Highlights Multiple Motor Pathways in the Rat Spinal Cord
07:57

Direct Injection of a Lentiviral Vector Highlights Multiple Motor Pathways in the Rat Spinal Cord

Published on: March 15, 2019

Basic research on cerebellar gene therapy using lentiviral vectors.

Hirokazu Hirai1

  • 1Department of Neurophysiology, Gunma University Graduate School of Medicine, Maebashi, Gunma, Japan. hirai@gunma-u.ac.jp

Cerebellum (London, England)
|November 29, 2011
PubMed
Summary

Lentiviral vectors enable efficient gene delivery into postmitotic neurons, offering a promising tool for cerebellar research. This method facilitates the study of cerebellar diseases and gene function in Purkinje cells.

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CRISPR-mediated Loss of Function Analysis in Cerebellar Granule Cells Using In Utero Electroporation-based Gene Transfer
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Published on: June 9, 2018

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Last Updated: May 27, 2026

Direct Injection of a Lentiviral Vector Highlights Multiple Motor Pathways in the Rat Spinal Cord
07:57

Direct Injection of a Lentiviral Vector Highlights Multiple Motor Pathways in the Rat Spinal Cord

Published on: March 15, 2019

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
  • Molecular Biology

Background:

  • Postmitotic neurons present challenges for gene delivery.
  • Lentiviral vectors offer efficient gene introduction with low toxicity to neurons.
  • These vectors demonstrate sustained transgene expression in neurons.

Purpose of the Study:

  • To present a method for efficient in vivo gene expression in cerebellar Purkinje cells using lentiviral vectors.
  • To highlight the utility of this method in investigating cerebellar pathophysiology.
  • To showcase applications in studying gene function and neurological disorders.

Main Methods:

  • Development of a lentiviral vector-based gene delivery system.
  • Application of the method for in vivo gene expression in cerebellar Purkinje cells.
  • Utilizing the system to study cerebellum-specific genes and spinocerebellar ataxia models.

Main Results:

  • Successful and efficient foreign gene expression in cerebellar Purkinje cells.
  • Demonstrated continuous transgene expression for extended periods.
  • Enabled the generation and analysis of spinocerebellar ataxia mouse models.
  • Facilitated rescue of ataxic phenotypes through gene introduction.

Conclusions:

  • Lentiviral vectors provide a robust platform for transgene expression in the cerebellum.
  • The developed method is effective for studying cerebellar function and disease.
  • This approach holds potential for gene therapy and understanding neurological disorders.