Cited by 194Muscle Physiology and DisordersNeurogenetic and Muscular Disorders ResearchVirus-based gene therapy research

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Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

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Rodino‐Klapac, Louise R., Haidet, Amanda M., Kota, Janaiah, Handy, Chalonda, Kaspar, Brian K., Mendell, Jerry R.·2009·Muscle & Nerve

DOI 10.1002/mus.21244PMC PMC2717722

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Abstract

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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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.
ORCID: Rodino‐Klapac, Louise R.
Kota, Janaiah
Kaspar, Brian K.
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
DOI: 10.1002/mus.21244
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
Licence: onlinelibrary.wiley.com/termsAndConditions#vor (vor)
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 (clinical) › 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

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Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease

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