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Related Experiment Video

Updated: Aug 29, 2025

Intramuscular Injections Along the Motor End Plates: A Minimally Invasive Approach to Shuttle Tracers Directly into Motor Neurons

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Published on: July 13, 2015

Approaches to Gene Modulation Therapy for ALS.

Katharina E Meijboom¹, Robert H Brown²

¹Department of Neurology, University of Massachusetts Medical School, Worcester, MA, 01605, USA.

Neurotherapeutics : the Journal of the American Society for Experimental Neurotherapeutics

|September 6, 2022

Summary

Gene modulation therapies offer new hope for amyotrophic lateral sclerosis (ALS) treatment by targeting key genes. Advances in gene silencing, correction, and augmentation are paving the way for future clinical trials and improved patient outcomes.

Keywords:

Adeno-associated virus (AAV)Amyotrophic lateral sclerosis (ALS)Antisense oligonucleotides (ASOs)CRISPR/Cas9 Gene therapy MicroRNA

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Last Updated: Aug 29, 2025

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ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

ALS - Motor Neuron Disease: Mechanism and Development of New Therapies

Published on: July 29, 2007

Area of Science:

Neuroscience
Genetics
Biotechnology

Background:

Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease with limited therapeutic options.
Understanding ALS genetics and disease mechanisms is crucial for developing effective treatments.

Purpose of the Study:

To review recent clinical and preclinical advances in gene modulation therapies for ALS.
To highlight the potential of gene silencing, correction, and augmentation strategies for ALS treatment.

Main Methods:

Review of clinical trials and preclinical studies involving gene modulation for ALS.
Focus on adeno-associated virus (AAV)-mediated microRNAs and antisense oligonucleotides (ASOs).
Targeting common ALS genes such as SOD1, C9ORF72, FUS, and ATXN2.

Main Results:

Six gene modulation therapies have been tested in ALS patients.
These therapies target the gain-of-function pathology of specific ALS-associated genes.
Gene modulation approaches show promise in preclinical and early clinical settings.

Conclusions:

Gene modulation strategies represent a promising frontier for ALS therapy development.
These approaches have the potential to significantly impact future research and clinical practice for ALS.
Innovations in gene therapy could transform treatment outcomes for this devastating disease.