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Gene Therapy

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...

Microorganisms in Medicine and Therapeutics

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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Updated: Jan 14, 2026

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents

Published on: August 10, 2018

Developing a minimally invasive gene therapy for multiple sclerosis.

Paul J H Nijhuis¹, Maurits Romijn¹, Roy Honing¹

¹Laboratory of Neuroregeneration, The Netherlands Institute for Neuroscience, an Institute of the Royal Netherlands Academy of Arts and Sciences, Meibergdreef 47, 1105 BA Amsterdam, the Netherlands.

Molecular Therapy. Methods & Clinical Development

|October 22, 2025

Summary

This summary is machine-generated.

A novel adeno-associated virus (AAV.PHP.eB) vector successfully targeted specific central nervous system (CNS) cells in a multiple sclerosis (MS) model. This gene therapy approach shows promise for future myelin repair and inflammation reduction in MS patients.

Keywords:

AAV EAE MS PHP.eB cell-type specificity gene therapy intravenous multiple sclerosis promoters systemic

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

Area of Science:

Neuroscience
Immunology
Gene Therapy

Background:

Multiple sclerosis (MS) is a CNS demyelinating disease.
Current treatments focus on managing inflammation and symptoms.
Developing targeted gene therapies for myelin repair is a key research area.

Purpose of the Study:

To evaluate the efficacy of the AAV.PHP.eB vector for cell-type-specific gene delivery in the CNS.
To assess the vector's ability to target MS-relevant cell types in an experimental autoimmune encephalitis (EAE) model.
To explore the potential of AAV.PHP.eB for future gene therapy applications in MS.

Main Methods:

Utilized adeno-associated virus (AAV.PHP.eB) vector for gene delivery in mice.
Employed experimental autoimmune encephalitis (EAE) as a model for MS.
Investigated transgene expression using bioluminescence imaging and histological analysis.
Tested various promoters (CAG, MBP, Sox10, hSyn1, gfa2, gfaABC1D) for cell-type specificity.

Main Results:

AAV.PHP.eB successfully traversed the blood-brain barrier in the EAE model.
Demonstrated long-term, robust, and cell-type-specific transgene expression in the brain and spinal cord.
MBP and Sox10 promoters targeted oligodendroglia within demyelinating lesions.
gfaABC1D and hSyn1 promoters targeted astrocytes and neurons, respectively.
Neural promoters showed minimal peripheral activity, with gfa2 as an exception.

Conclusions:

AAV.PHP.eB enables targeted gene delivery to specific CNS cell types relevant to MS.
The vector's ability to target oligodendroglia, astrocytes, and neurons is promising for MS gene therapy.
This study provides a foundation for developing minimally invasive gene therapies for myelin repair and inflammation control in MS.