Follistatin-344
Research-Grade
Follistatin-344 (FS344) is the full-length isoform of human follistatin, a single-chain glycoprotein expressed in nearly all tissues. Its primary biological role is to bind and neutralize members of the TGF-β superfamily, most notably myostatin (GDF-8) and activin A — both of which act as powerful negative regulators of skeletal muscle mass. In animal models, overexpression of follistatin produces dramatic muscle hypertrophy even in myostatin-knockout backgrounds, indicating it inhibits additional anti-growth signals beyond myostatin alone. This has made it a major focus of gene therapy research: Nationwide Children's Hospital has conducted phase I/II AAV-follistatin gene therapy trials in Becker muscular dystrophy and inclusion body myositis with encouraging safety and preliminary efficacy data. As an injectable peptide, FS344 is available only as a research compound. Its large molecular size and glycosylation make bioavailability and stability more challenging than typical small peptides. Most practical use is via gene therapy vectors rather than direct peptide injection.
Specifications
| Origin / Manufacturer | Synthetic / Recombinant |
| Active Components | Follistatin-344 recombinant proteinMannitol (lyoprotectant)Bacteriostatic water (for reconstitution) |
| Storage | Lyophilized: −20°C. Reconstituted: 2–8°C |
| Shelf Life | Lyophilized 12+ months at −20°C; reconstituted 7 days refrigerated |
| Form Factor | Lyophilized powder (1 mg vial) |
Clinical Evidence
Clinical report reference
Clinical report reference
Clinical report reference
Frequently Asked Questions
Sources & References
Every clinical claim on this page traces to a primary peer-reviewed source.
- 1Mendell JR, Sahenk Z, Malik V, et al.. A phase 1/2a follistatin gene therapy trial for Becker muscular dystrophy. Molecular Therapy. 2015;23(1):192-201. doi:10.1038/mt.2014.200 PMID:25322757
- 2Lee SJ, McPherron AC. Regulation of myostatin activity and muscle growth. Proceedings of the National Academy of Sciences. 2001;98(16):9306-9311. PMID:11459935
- 3Rodino-Klapac LR, Haidet AM, Kota J, et al.. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease. Muscle & Nerve. 2009;39(3):283-296. doi:10.1002/mus.21244 PMID:19208403
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