{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T08:48:21Z","timestamp":1790153301705,"version":"4.0.1"},"reference-count":44,"publisher":"Elsevier BV","issue":"9","license":[{"start":{"date-parts":[[2010,9,1]],"date-time":"2010-09-01T00:00:00Z","timestamp":1283299200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":2228,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2010,9]]},"DOI":"10.1038\/mt.2010.110","type":"journal-article","created":{"date-parts":[[2010,6,15]],"date-time":"2010-06-15T14:15:42Z","timestamp":1276611342000},"page":"1584-1591","source":"Crossref","is-referenced-by-count":21,"title":["Evaluation of Systemic Follistatin as an Adjuvant to Stimulate Muscle Repair and Improve Motor Function in Pompe Mice"],"prefix":"10.1016","volume":"18","author":[{"given":"Joseph W","family":"Foley","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott D","family":"Bercury","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrick","family":"Finn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seng H","family":"Cheng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ronald K","family":"Scheule","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robin J","family":"Ziegler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2010.110_bib1","series-title":"The Metabolic and Molecular Bases of Inherited Disease, Vol. III","first-page":"3389","article-title":"Glycogen storage disease type ii: acid \u03b1-glucosidase (acid maltase) deficiency","author":"Hirschhorn","year":"2001"},{"key":"10.1038\/mt.2010.110_bib2","doi-asserted-by":"crossref","first-page":"S35","DOI":"10.1016\/j.jpeds.2004.01.053","article-title":"Pompe disease in infants and children","volume":"144","author":"Kishnani","year":"2004","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2010.110_bib3","doi-asserted-by":"crossref","first-page":"19086","DOI":"10.1074\/jbc.273.30.19086","article-title":"Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II","volume":"273","author":"Raben","year":"1998","journal-title":"J Biol Chem"},{"key":"10.1038\/mt.2010.110_bib4","doi-asserted-by":"crossref","first-page":"700","DOI":"10.1002\/ana.20807","article-title":"Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease","volume":"59","author":"Fukuda","year":"2006","journal-title":"Ann Neurol"},{"key":"10.1038\/mt.2010.110_bib5","doi-asserted-by":"crossref","first-page":"546","DOI":"10.4161\/auto.4591","article-title":"Deconstructing Pompe disease by analyzing single muscle fibers: to see a world in a grain of sand","volume":"3","author":"Raben","year":"2007","journal-title":"Autophagy"},{"key":"10.1038\/mt.2010.110_bib6","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1089\/hum.2008.010","article-title":"Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice","volume":"19","author":"Ziegler","year":"2008","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.110_bib7","doi-asserted-by":"crossref","first-page":"418","DOI":"10.1038\/nature01154","article-title":"Functional improvement of dystrophic muscle by myostatin blockade","volume":"420","author":"Bogdanovich","year":"2002","journal-title":"Nature"},{"key":"10.1038\/mt.2010.110_bib8","doi-asserted-by":"crossref","first-page":"2924","DOI":"10.1172\/JCI28520","article-title":"Muscular atrophy of caveolin-3-deficient mice is rescued by myostatin inhibition","volume":"116","author":"Ohsawa","year":"2006","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2010.110_bib9","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1096\/fj.07-8673com","article-title":"Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice","volume":"22","author":"Nakatani","year":"2008","journal-title":"FASEB J"},{"key":"10.1038\/mt.2010.110_bib10","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1089\/hum.2007.159","article-title":"Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice","volume":"19","author":"Qiao","year":"2008","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.110_bib11","doi-asserted-by":"crossref","first-page":"4318","DOI":"10.1073\/pnas.0709144105","article-title":"Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors","volume":"105","author":"Haidet","year":"2008","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.110_bib12","first-page":"14","article-title":"Myostatin inhibition by a follistatin-derived peptide ameliorates the pathophysiology of muscular dystrophy model mice","volume":"27","author":"Tsuchida","year":"2008","journal-title":"Acta Myol"},{"key":"10.1038\/mt.2010.110_bib13","doi-asserted-by":"crossref","first-page":"2519","DOI":"10.1073\/pnas.0408729102","article-title":"Muscle regeneration in the prolonged absence of myostatin","volume":"102","author":"Wagner","year":"2005","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.110_bib14","doi-asserted-by":"crossref","first-page":"1463","DOI":"10.1038\/sj.mt.6300182","article-title":"Antagonism of myostatin enhances muscle regeneration during sarcopenia","volume":"15","author":"Siriett","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib15","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1016\/S0006-291X(02)02953-4","article-title":"Inhibition of myostatin in adult mice increases skeletal muscle mass and strength","volume":"300","author":"Whittemore","year":"2003","journal-title":"Biochem Biophys Res Commun"},{"key":"10.1038\/mt.2010.110_bib16","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.ydbio.2004.01.046","article-title":"Follistatin complexes Myostatin and antagonises Myostatin-mediated inhibition of myogenesis","volume":"270","author":"Amthor","year":"2004","journal-title":"Dev Biol"},{"key":"10.1038\/mt.2010.110_bib17","doi-asserted-by":"crossref","first-page":"3586","DOI":"10.1210\/en.2006-0089","article-title":"Biological activity of follistatin isoforms and follistatin-like-3 is dependent on differential cell surface binding and specificity for activin, myostatin, and bone morphogenetic proteins","volume":"147","author":"Sidis","year":"2006","journal-title":"Endocrinology"},{"key":"10.1038\/mt.2010.110_bib18","doi-asserted-by":"crossref","first-page":"e789","DOI":"10.1371\/journal.pone.0000789","article-title":"Quadrupling muscle mass in mice by targeting TGF-beta signaling pathways","volume":"2","author":"Lee","year":"2007","journal-title":"PLoS ONE"},{"key":"10.1038\/mt.2010.110_bib19","doi-asserted-by":"crossref","first-page":"E157","DOI":"10.1152\/ajpendo.00193.2009","article-title":"Follistatin induces muscle hypertrophy through satellite cell proliferation and inhibition of both myostatin and activin","volume":"297","author":"Gilson","year":"2009","journal-title":"Am J Physiol Endocrinol Metab"},{"key":"10.1038\/mt.2010.110_bib20","doi-asserted-by":"crossref","first-page":"6ra15","DOI":"10.1126\/scitranslmed.3000112","article-title":"Follistatin gene delivery enhances muscle growth and strength in nonhuman primates","volume":"1","author":"Kota","year":"2009","journal-title":"Sci Transl Med"},{"key":"10.1038\/mt.2010.110_bib21","doi-asserted-by":"crossref","first-page":"4218","DOI":"10.1073\/pnas.85.12.4218","article-title":"Primary structure of the human follistatin precursor and its genomic organization","volume":"85","author":"Shimasaki","year":"1988","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.110_bib22","doi-asserted-by":"crossref","first-page":"5052","DOI":"10.1210\/en.2005-0833","article-title":"Differential biosynthesis and intracellular transport of follistatin isoforms and follistatin-like-3","volume":"146","author":"Saito","year":"2005","journal-title":"Endocrinology"},{"key":"10.1038\/mt.2010.110_bib23","doi-asserted-by":"crossref","first-page":"1773","DOI":"10.1089\/10430349950017455","article-title":"Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors","volume":"10","author":"Pastore","year":"1999","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2010.110_bib24","doi-asserted-by":"crossref","first-page":"1347","DOI":"10.1172\/JCI200316887","article-title":"Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer","volume":"111","author":"Mingozzi","year":"2003","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2010.110_bib25","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.ymthe.2003.11.015","article-title":"AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice","volume":"9","author":"Ziegler","year":"2004","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib26","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1016\/j.ymthe.2005.04.024","article-title":"Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II","volume":"12","author":"Franco","year":"2005","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib27","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1038\/sj.mt.6300066","article-title":"Correction of the biochemical and functional deficits in fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A","volume":"15","author":"Ziegler","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib28","doi-asserted-by":"crossref","first-page":"954","DOI":"10.1038\/mt.2009.37","article-title":"Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease","volume":"17","author":"Zhu","year":"2009","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib29","doi-asserted-by":"crossref","first-page":"1975","DOI":"10.2353\/ajpath.2006.051316","article-title":"Age dependent effect of myostatin blockade on disease severity in a murine model of limb-girdle muscular dystrophy","volume":"168","author":"Parsons","year":"2006","journal-title":"Am J Pathol"},{"key":"10.1038\/mt.2010.110_bib30","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/j.jpeds.2006.02.035","article-title":"Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease","volume":"149","author":"Kishnani","year":"2006","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2010.110_bib31","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1212\/01.wnl.0000251268.41188.04","article-title":"Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease","volume":"68","author":"Kishnani","year":"2007","journal-title":"Neurology"},{"key":"10.1038\/mt.2010.110_bib32","doi-asserted-by":"crossref","first-page":"1033","DOI":"10.1007\/s00246-008-9267-3","article-title":"Cardiac remodeling after enzyme replacement therapy with acid alpha-glucosidase for infants with Pompe disease","volume":"29","author":"Levine","year":"2008","journal-title":"Pediatr Cardiol"},{"key":"10.1038\/mt.2010.110_bib33","doi-asserted-by":"crossref","first-page":"210","DOI":"10.1097\/GIM.0b013e31819d0996","article-title":"Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease","volume":"11","author":"Nicolino","year":"2009","journal-title":"Genet Med"},{"key":"10.1038\/mt.2010.110_bib34","first-page":"155","article-title":"Results from a randomized, double-blind, multicenter, multinational, placebo-controlled study of the safety and efficacy of Myozyme, recombinant human acid alpha-glucosidase (rhGAA), for the treatment of Pompe disease in juveniles and adults","volume":"71","author":"van der Ploeg","year":"2008","journal-title":"Neurology"},{"key":"10.1038\/mt.2010.110_bib35","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1002\/ana.20019","article-title":"Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up","volume":"55","author":"Winkel","year":"2004","journal-title":"Ann Neurol"},{"key":"10.1038\/mt.2010.110_bib36","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1016\/j.nmd.2008.04.009","article-title":"Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease","volume":"18","author":"van Capelle","year":"2008","journal-title":"Neuromuscul Disord"},{"key":"10.1038\/mt.2010.110_bib37","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.1016\/j.jbiomech.2004.05.040","article-title":"Effects of non-contractile inclusions on mechanical performance of skeletal muscle","volume":"38","author":"Drost","year":"2005","journal-title":"J Biomech"},{"key":"10.1038\/mt.2010.110_bib38","doi-asserted-by":"crossref","first-page":"1835","DOI":"10.1073\/pnas.0604893104","article-title":"Lack of myostatin results in excessive muscle growth but impaired force generation","volume":"104","author":"Amthor","year":"2007","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2010.110_bib39","doi-asserted-by":"crossref","first-page":"6","DOI":"10.1186\/1755-8417-1-6","article-title":"Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts","volume":"1","author":"Cardone","year":"2008","journal-title":"Pathogenetics"},{"key":"10.1038\/mt.2010.110_bib40","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1002\/mus.10381","article-title":"Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy","volume":"27","author":"Winkel","year":"2003","journal-title":"Muscle Nerve"},{"key":"10.1038\/mt.2010.110_bib41","doi-asserted-by":"crossref","first-page":"1208","DOI":"10.1038\/labinvest.3700484","article-title":"Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease","volume":"86","author":"Thurberg","year":"2006","journal-title":"Lab Invest"},{"key":"10.1038\/mt.2010.110_bib42","doi-asserted-by":"crossref","first-page":"1400","DOI":"10.1038\/mt.2008.115","article-title":"Systemic Insulin-like growth factor-1 reverses hypoalgesia and improves mobility in a mouse model of diabetic peripheral neuropathy","volume":"16","author":"Chu","year":"2008","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2010.110_bib43","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1507\/endocrj.KR07E-001","article-title":"Therapeutic potential of follistatin to promote tissue regeneration and prevent tissue fibrosis","volume":"54","author":"Aoki","year":"2007","journal-title":"Endocr J"},{"key":"10.1038\/mt.2010.110_bib44","doi-asserted-by":"crossref","first-page":"G89","DOI":"10.1152\/ajpgi.2000.278.1.G89","article-title":"Gene expression of activin, activin receptors, and follistatin in intestinal epithelial cells","volume":"278","author":"Sonoyama","year":"2000","journal-title":"Am J Physiol Gastrointest Liver Physiol"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616308334?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616308334?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2019,5,30]],"date-time":"2019-05-30T03:35:07Z","timestamp":1559187307000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616308334"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,9]]},"references-count":44,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2010,9]]}},"alternative-id":["S1525001616308334"],"URL":"https:\/\/doi.org\/10.1038\/mt.2010.110","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2010,9]]}}}