Approaches to gene modulation therapy for ALS
Open accessAbstract
Amyotrophic lateral sclerosis (ALS) is a devastating motor neuron disease for which there is currently no robust therapy. Recent progress in understanding ALS disease mechanisms and genetics in combination with innovations in gene modulation strategies creates promising new options for the development of ALS therapies. In recent years, six gene modulation therapies have been tested in ALS patients. These target gain-of-function pathology of the most common ALS genes, SOD1, C9ORF72, FUS, and ATXN2, using adeno-associated virus (AAV)-mediated microRNAs and antisense oligonucleotides (ASOs). Here, we review the latest clinical and preclinical advances in gene modulation approaches for ALS, including gene silencing, gene correction, and gene augmentation. These techniques have the potential to positively impact the direction of future research trials and transform ALS treatments for this grave disease.
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There is no specific treatment available for ALS, although novel treatments include gene therapy and stem cell therapy.
“There is no specific treatment available for ALS, although the more popular novel treatments include gene therapy (7) and stem cell therapy (8).”
From Modulation of the gut–brain axis via the gut microbiota: a new era in treatment of amyotrophic lateral sclerosis (2023) · cites this paper as [7]
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References 173 · Cited by 38
Part of this paper’s reference list could not be split into single entries.
Research works printed but not linked: 1
- Belikova AM, Zarytova VF, Grineva NI. Synthesis of ribo- nucleosides and diribonucleoside phosphates containing 2-chloroethylamine and nitrogen mustard residues. Tetrahe- dron Lett. 1967;37:3557-62.
- Authors
- Meijboom, K. E., & Brown, R. H.
- Affiliations
- University of Massachusetts Chan Medical School (US)
- Corresponding author
- Brown, Robert H.
- Venue
- Neurotherapeutics
- Publisher
- Elsevier BV
- Published
- 2022-07
- Volume
- 19
- Issue
- 4
- Pages
- 1159-1179
- ISSN
- 1878-7479 (ISSN-L)
- PMC
- PMC9587165
- OpenAlex
- W4294839703
- Cited by
- 38
- References
- 173
- Citations by year
- 2026: 8 · 2025: 9 · 2024: 7 · 2023: 11 · 2022: 3
- FWCI
- 1.08
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Neurotherapeutics (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- NIH Clinical Center (R01 NS111990)
- The Angel Fund for ALS Research
- ALSOne
- ALS Finding a Cure
- The Cellucci Fund for ALS Research
- The Pierre de Bourgknecht ALS Research Fund
- Max Rosenfeld Fund
- Topics
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Keywords
- amyotrophic lateral sclerosis, antisense oligonucleotides, adeno-associated virus vectors, microRNA, gene silencing, gene correction, gene augmentation, SOD1, C9orf72, FUS, ATN2, motor neuron disease, clinical trials
- MeSH terms
- Superoxide Dismutase-1, C9orf72 Protein, Amyotrophic Lateral Sclerosis, Humans, Oligonucleotides, Antisense, MicroRNAs
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