ArticleMolecular therapy : the journal of the American Society of Gene Therapy2017
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Registry-linked trialOpen access · bronzeAbstract read
In one paragraph
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT07443826 (CALM-AF-AI), which is not on this map. Cited by 63 papers.
0numbers the graph read from it
0cells of the map it votes in
63citing papers in PubMed
6.5field-weighted citation impact, top 2% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
NCT07443826 phase1 / phase2recruitingnot on this mapstarted 2026, after this paper: background citation
CALM-AF-AI: Counteracting Age-related Loss of Muscle With AAV-Follistatin Combined With Angiogenesis-Inducing VEGF Plasmid Gene Therapy
Who cites it
63 citing papers in PubMed, 104 citations in OpenAlex.
- The NORAD-Pumilio regulatory axis in the evolution of inclusion body myositis.Journal of neuropathology and experimental neurology · 2026Article
- Sarcopenia and Skeletal Muscle Dysfunction in Liver Cirrhosis: Clinical Perspectives of Myostatin Inhibition.Biomedicines · 2026Review
- Alterations in systemic and skeletal muscle myostatin regulation are most prominent at disease onset and associate with muscle-related outcomes in idiopathic inflammatory myopathies.Arthritis research & therapy · 2026Article
- Follistatin Mitigates Atherosclerosis Through Activation of Arginine Metabolism and Adipose Browning.Cells · 2026Article
- Targeted Gene and Genome-Editing Strategies for Epilepsy: Experimental Advances and Translational Challenges.International journal of molecular sciences · 2026Review
- AAVrh74.tMCK.NT-3 Surrogate Gene Therapy in a Mouse Model of CMT2A.International journal of molecular sciences · 2026Article
- Grip strength as a systems biomarker of aging: neuromuscular junction constraints and biomarker decoupling in gene therapy.Frontiers in aging · 2026Article
- Integrative Approaches to Myopathies and Muscular Dystrophies: Molecular Mechanisms, Diagnostics, and Future Therapies.International journal of molecular sciences · 2025Review
- Sarcopenia in chronic obstructive pulmonary disease: mechanisms, diagnosis, and management strategies.Annals of medicine and surgery (2012) · 2025Review
- AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis.Gene therapy · 2025Article
- Multiplex detection of seven transgenes for human gene doping analysis.Scientific reports · 2025Article
- Therapeutic applications and challenges in myostatin inhibition for enhanced skeletal muscle mass and functions.Molecular and cellular biochemistry · 2025Review
- Inclusion body myositis and immunosenescence: current evidence and future perspectives.Rheumatology (Oxford, England) · 2025Review
- Targeting Soluble TGF-β Factors: Advances in Precision Therapy for Pulmonary Arterial Hypertension.JACC. Basic to translational science · 2024Review
- Dual FKRP/FST gene therapy normalizes ambulation, increases strength, decreases pathology, and amplifies gene expression in LGMDR9 mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Sporadic Inclusion Body Myositis at the Crossroads between Muscle Degeneration, Inflammation, and Aging.International journal of molecular sciences · 2024Review
- The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.Neurology and therapy · 2024Article
- Sarcopenia in chronic kidney disease: from bench to bedside.The Korean journal of internal medicine · 2023Review
- Inclusion body myositis: from genetics to clinical trials.Journal of neurology · 2023Review
- The elusive role of myostatin signaling for muscle regeneration and maintenance of muscle and bone homeostasis.Osteoporosis and sarcopenia · 2023Review
3 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
19 authors at 2 institutions in 1 country.
Jerry R MendellCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA. Electronic address: jerry.mendell@nationwidechildrens.org.
Zarife SahenkCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.
Samiah Al-ZaidyCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Louise R Rodino-KlapacCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Linda P LowesCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Lindsay N AlfanoCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Katherine BerryCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Natalie MillerCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA; Clinical Therapies, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Mehmet YalvacCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Igor DvorchikBiostatics Research Core, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Melissa Moore-ClingenpeelBiostatics Research Core, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Kevin M FlaniganCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA; Department of Neurology, The Ohio State University, Columbus, OH 43210, USA.
Kathleen ChurchCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Kim ShontzCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Choumpree CurryCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Sarah LewisCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Markus McCollyCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Mark J HoganVascular and Interventional Radiology, Department of Radiology, Nationwide Children's Hospital, Columbus, OH 43205, USA.
Brian K KasparCenter for Gene Therapy, Nationwide Children's Hospital, Columbus, OH 43205, USA; Department of Pediatrics, The Ohio State University, Columbus, OH 43205, USA.
Nationwide Children's Hospital · USThe Ohio State University · US
Funding
Training and Education CoreU54HD066409 · NICHD · RESEARCH INST NATIONWIDE CHILDREN'S HOSP · PI MARTIN, PAUL TAYLOR · 2010 to 2014
$6.7MNICHD NIH HHS U54 HD066409
Abstract
Sporadic inclusion body myositis, a variant of inflammatory myopathy, has features distinct from polymyositis/dermatomyositis. The disease affects men more than women, most commonly after age 50. Clinical features include weakness of the quadriceps, finger flexors, ankle dorsiflexors, and dysphagia. The distribution of weakness is similar to Becker muscular dystrophy, where we previously reported improvement following intramuscular injection of an isoform of follistatin (FS344) by AAV1. For this clinical trial, rAAV1.CMV.huFS344, 6 × 10
Indexed as
Genetic TherapyAgedAMP-Activated Protein KinasesAnimalsBiomarkersBiopsyDependovirusFollistatinFollow-Up StudiesGene DosageGenetic VectorsHumansMaleMiceMiddle AgedMuscle, SkeletalAMP-Activated Protein KinasesBiomarkersFollistatinTOR Serine-Threonine Kinases6-min walk distance6-min walk testadeno-associated virusFollistatingene therapysporadic inclusion body myositis
Identifiers
What OpenQuestion holds
Textmetadata
Read underepoch 390
Registered trials
NCT07443826ClinicalTrials.gov
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.