Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease
Open accessAbstract
In most cases, pharmacologic strategies to treat genetic muscle disorders and certain acquired disorders, such as sporadic inclusion body myositis, have produced modest clinical benefits. In these conditions, inhibition of the myostatin pathway represents an alternative strategy to improve functional outcomes. Preclinical data that support this approach clearly demonstrate the potential for blocking the myostatin pathway. Follistatin has emerged as a powerful antagonist of myostatin that can increase muscle mass and strength. Follistatin was first isolated from the ovary and is known to suppress follicle-stimulating hormone. This raises concerns for potential adverse effects on the hypothalamic-pituitary-gonadal axis and possible reproductive capabilities. In this review we demonstrate a strategy to bypass off-target effects using an alternatively spliced cDNA of follistatin (FS344) delivered by adeno-associated virus (AAV) to muscle. The transgene product is a peptide of 315 amino acids that is secreted from the muscle and circulates in the serum, thus avoiding cell-surface binding sites. Using this approach our translational studies show increased muscle size and strength in species ranging from mice to monkeys. Adverse effects are avoided, and no organ system pathology or change in reproductive capabilities has been seen. These findings provide the impetus to move toward gene therapy clinical trials with delivery of AAV-FS344 to increase size and function of muscle in patients with neuromuscular disease.
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FST acts as a multi-faceted hepatokine involved in reproduction, muscle growth, cancer development, and metabolism.
“FST acts as a multi-faceted hepatokine that is involved in reproduction [181], muscle growth [182], cancer development [183], and metabolism [184].”
From Hepatokines and metabolism: Deciphering communication from the liver (2021) · cites this paper as [182]
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- Authors
- Rodino‐Klapac, L. R., Haidet, A. M., Kota, J., Handy, C., Kaspar, B. K., & Mendell, J. R.
- Affiliations
- Nationwide Children's Hospital (US)
- Corresponding author
- Mendell, Jerry R.
- Venue
- Muscle & Nerve
- Publisher
- Wiley
- Published
- 2009-02-10
- Volume
- 39
- Issue
- 3
- Pages
- 283-296
- ISSN
- 0148-639X (ISSN-L)
- 1097-4598
- PMC
- PMC2717722
- OpenAlex
- W2108645449
- Cited by
- 194
- References
- 83
- Citations by year
- 2026: 6 · 2025: 5 · 2024: 4 · 2023: 8 · 2022: 8 · 2021: 9 · 2020: 13 · 2019: 12 · 2018: 14 · 2017: 7 · 2016: 14 · 2015: 10 · 2014: 10 · 2013: 10 · 2012: 21
- FWCI
- 1.63
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Muscle & Nerve (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (F31NS058224)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Muscle Physiology and Disorders
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- myostatin inhibition, follistatin, adeno-associated virus gene therapy, myopathy, neuromuscular disorders, sporadic inclusion body myositis, muscle mass and strength, hypothalamic-pituitary-gonadal axis, reproductive safety, alternative splicing
- MeSH terms
- Dependovirus, Animals, Humans, Mice, Mutant Strains, Muscles, Muscular Diseases, Genetic Therapy, Alternative Splicing, DNA, Complementary, Follistatin, Mice, Myostatin
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
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