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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...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
Recombinant DNA01:09

Recombinant DNA

Overview
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Related Experiment Video

Updated: Jun 22, 2026

A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears
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A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears

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Future applications: gene therapy.

R M Richardson1, V Varenika, J R Forsayeth

  • 1Laboratory for Molecular Therapeutics, Department of Neurological Surgery, University of California San Francisco, 1855 Folsom Street, Room 226, San Francisco, CA 94103, USA. richardsonma@neurosurg.ucsf.edu

Neurosurgery Clinics of North America
|June 27, 2009
PubMed
Summary

Gene therapy using viral vectors is a safe and promising frontier in restorative neurosurgery for brain disorders. Future clinical trials will expand gene therapy applications for neurodegenerative diseases, stroke, and traumatic brain injury.

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Last Updated: Jun 22, 2026

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Suspension Culture Production and Purification of Adeno-Associated Virus by Iodixanol Density Gradient Centrifugation for In Vivo Applications
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Suspension Culture Production and Purification of Adeno-Associated Virus by Iodixanol Density Gradient Centrifugation for In Vivo Applications

Published on: February 9, 2024

Area of Science:

  • Neurosurgery
  • Gene Therapy
  • Neurology

Background:

  • Gene therapy represents a promising frontier in restorative neurosurgery for brain disorders.
  • Current experimental gene therapy initiatives are active in clinical trials.
  • Direct intracerebral delivery of viral vectors has shown safety and tolerability.

Purpose of the Study:

  • To highlight the current status and future potential of gene therapy in neurosurgery.
  • To discuss the application of viral vectors for gene transfer in the brain.
  • To explore the relevance of gene therapy for treating neurodegenerative diseases, stroke, and traumatic brain injury.

Main Methods:

  • Utilizing viral vectors for direct intracerebral gene delivery.
  • Conducting clinical trials to assess safety and efficacy.
  • Investigating gene transfer for enzymatic or neurotrophic activity.

Main Results:

  • Gene therapy treatments delivered via intracerebral viral vectors are safe and well tolerated in current trials.
  • Significant experimental gene therapy initiatives are underway.
  • Established safety and tolerability of current gene therapy approaches.

Conclusions:

  • Gene therapy is a safe and effective approach for brain disorders.
  • Future clinical trials will likely expand gene therapy applications for various neurological conditions.
  • Viral vector-mediated gene transfer holds significant therapeutic potential for neurodegenerative diseases, stroke, and traumatic brain injury.