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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.

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Clinically relevant AAV8- <i>PEX1</i> gene therapy preserves retinal integrity and function long-term in a murine model of Zellweger spectrum disorder.

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Related Experiment Video

Updated: May 12, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
05:09

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

Published on: May 16, 2025

Progress in gene therapy for neurological disorders.

Michele Simonato1, Jean Bennett, Nicholas M Boulis

  • 1Section of Pharmacology, Department of Medical Sciences, University of Ferrara, Fossato di Mortara 17-19, 44121 Ferrara, Italy. michele.simonato@ unife.it

Nature Reviews. Neurology
|April 24, 2013
PubMed
Summary

Gene therapy offers a promising new treatment for neurological diseases, particularly those affecting the elderly. Advances in gene therapy techniques provide hope for curing nervous system disorders where traditional treatments have failed.

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High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies
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Last Updated: May 12, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
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Published on: May 16, 2025

Delivery of Therapeutic Agents Through Intracerebroventricular (ICV) and Intravenous (IV) Injection in Mice
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09:19

High Throughput Characterization of Adult Stem Cells Engineered for Delivery of Therapeutic Factors for Neuroprotective Strategies

Published on: January 4, 2015

Area of Science:

  • Neurology
  • Genetics
  • Biotechnology

Background:

  • Neurological diseases significantly impact populations, especially the elderly, often stemming from genetic mutations or environmental factors.
  • Current medical and surgical treatments are often ineffective, with limited pharmaceutical options to halt disease progression.
  • Gene therapy presents a novel and powerful therapeutic strategy for neurological disorders.

Purpose of the Study:

  • To review current gene therapy strategies for treating neurological diseases.
  • To discuss the advancements enabling gene therapy for nervous system disorders.
  • To explore future prospects of gene therapy in neurology.

Main Methods:

  • Review of scientific literature on gene therapy for neurological diseases.
  • Analysis of progress in understanding disease mechanisms, gene vector design, gene selection, and delivery methods.
  • Synthesis of findings to assess the potential of gene therapy.

Main Results:

  • Gene therapy shows significant potential for treating and potentially curing neurological diseases.
  • Improvements in understanding disease mechanisms and gene delivery technologies are driving progress.
  • Gene therapy is becoming a viable treatment option for neurologists, ophthalmologists, and neurosurgeons.

Conclusions:

  • Gene therapy holds considerable promise for addressing the unmet needs in treating neurological diseases.
  • Continued advancements in the field are expected to expand therapeutic applications.
  • Gene therapy represents a hopeful future for managing debilitating nervous system disorders.