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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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...
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Updated: Mar 29, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
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Systemic Gene Therapy for Targeting the CNS.

Sara E Gombash1,2, Kevin D Foust3

  • 1Department of Translational Science& Molecular Medicine, Michigan State University, Grand Rapids, MI, USA.

Methods in Molecular Biology (Clifton, N.J.)
|November 28, 2015
PubMed
Summary

Systemic gene delivery effectively targets the central nervous system in mice for disease modeling and treatment. These straightforward intravenous techniques require minimal equipment for both neonates and adults.

Keywords:
Adeno-associated virus type 9Intravenous injectionSuperficial temporal veinSystemic gene therapyTail vein

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Area of Science:

  • Neuroscience and Genetics
  • Biomedical Research

Background:

  • Systemic gene delivery is crucial for studying and treating widespread diseases.
  • Recent advancements show systemic agents can effectively target the central nervous system (CNS).
  • This approach has shown significant success in rodent models of neurological disorders.

Purpose of the Study:

  • To detail intravenous gene delivery methods for neonate and adult mice.
  • To provide easily learnable techniques with minimal equipment needs.

Main Methods:

  • Intravenous delivery of agents in young and adult mice.
  • Focus on practical, low-equipment procedures.

Main Results:

  • Successful systemic gene delivery to the CNS in mice.
  • Demonstration of techniques applicable to both neonate and adult subjects.
  • Confirmation of ease of learning and minimal equipment requirements.

Conclusions:

  • Intravenous gene delivery is a viable and accessible method for CNS targeting in mice.
  • These techniques facilitate neurological disease modeling and therapeutic development.
  • The described methods offer a practical approach for researchers.