{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,26]],"date-time":"2026-08-26T10:22:43Z","timestamp":1787739763910,"version":"build-2784847793"},"reference-count":45,"publisher":"Elsevier BV","issue":"9","license":[{"start":{"date-parts":[[2013,9,1]],"date-time":"2013-09-01T00:00:00Z","timestamp":1377993600000},"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":1132,"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":[[2013,9]]},"DOI":"10.1038\/mt.2013.96","type":"journal-article","created":{"date-parts":[[2013,6,4]],"date-time":"2013-06-04T12:25:29Z","timestamp":1370348729000},"page":"1661-1667","source":"Crossref","is-referenced-by-count":65,"title":["Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease"],"prefix":"10.1016","volume":"21","author":[{"given":"Darin J","family":"Falk","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cathryn S","family":"Mah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meghan S","family":"Soustek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kun-Ze","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mai K","family":"ElMallah","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denise A","family":"Cloutier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David D","family":"Fuller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J","family":"Byrne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"R1","key":"10.1038\/mt.2013.96_bib1","doi-asserted-by":"crossref","first-page":"R61","DOI":"10.1093\/hmg\/ddr174","article-title":"Pompe disease gene therapy","volume":"20","author":"Byrne","year":"2011","journal-title":"Hum Mol Genet"},{"key":"10.1038\/mt.2013.96_bib2","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.ymgme.2011.02.004","article-title":"Pompe disease: design, methodology, and early findings from the Pompe Registry","volume":"103","author":"Byrne","year":"2011","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2013.96_bib3","doi-asserted-by":"crossref","first-page":"628","DOI":"10.1097\/MOP.0b013e3282f161f2","article-title":"Enzyme reconstitution\/replacement therapy for lysosomal storage diseases","volume":"19","author":"Burrow","year":"2007","journal-title":"Curr Opin Pediatr"},{"key":"10.1038\/mt.2013.96_bib4","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1007\/s11910-007-0024-4","article-title":"Acid alpha-glucosidase deficiency (Pompe disease)","volume":"7","author":"Fukuda","year":"2007","journal-title":"Curr Neurol Neurosci Rep"},{"key":"10.1038\/mt.2013.96_bib5","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1016\/j.ymgme.2007.07.009","article-title":"Pompe disease: current state of treatment modalities and animal models","volume":"92","author":"Geel","year":"2007","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2013.96_bib6","doi-asserted-by":"crossref","first-page":"145","DOI":"10.2174\/1566524024605789","article-title":"Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease)","volume":"2","author":"Raben","year":"2002","journal-title":"Curr Mol Med"},{"key":"10.1038\/mt.2013.96_bib7","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1016\/j.ymgme.2011.12.012","article-title":"Mannose-6-phosphate pathway: a review on its role in lysosomal function and dysfunction","volume":"105","author":"Coutinho","year":"2012","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2013.96_bib8","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1097\/01.gim.0000218152.87434.f3","article-title":"Pompe disease diagnosis and management guideline","volume":"8","author":"Kishnani","year":"2006","journal-title":"Genet Med"},{"key":"10.1038\/mt.2013.96_bib9","doi-asserted-by":"crossref","first-page":"9419","DOI":"10.1073\/pnas.0902534106","article-title":"Neural deficits contribute to respiratory insufficiency in Pompe disease","volume":"106","author":"DeRuisseau","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2013.96_bib10","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.2013.96_bib11","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1097\/00005072-196501000-00008","article-title":"Pompe's disease (diffuse glycogenosis) with neuronal storage","volume":"24","author":"Mancall","year":"1965","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/mt.2013.96_bib12","doi-asserted-by":"crossref","first-page":"412","DOI":"10.1097\/00005072-197107000-00008","article-title":"Nervous system in Pompe's disease. Ultrastructure and biochemistry","volume":"30","author":"Gambetti","year":"1971","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/mt.2013.96_bib13","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1007\/BF00687878","article-title":"Pompe's disease: an inborn lysosomal disorder with storage of glycogen. A study of brain and striated muscle","volume":"23","author":"Martin","year":"1973","journal-title":"Acta Neuropathol"},{"key":"10.1038\/mt.2013.96_bib14","first-page":"379","article-title":"Infantile-onset glycogen storage disease type II (Pompe disease): report of a case with genetic diagnosis and pathological findings","volume":"27","author":"Teng","year":"2004","journal-title":"Chang Gung Med J"},{"key":"10.1038\/mt.2013.96_bib15","doi-asserted-by":"crossref","first-page":"312","DOI":"10.1177\/030098588302000307","article-title":"Generalized glycogen storage disease in Japanese quail (Coturnix coturnix japonica)","volume":"20","author":"Matsui","year":"1983","journal-title":"Vet Pathol"},{"key":"10.1038\/mt.2013.96_bib16","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1097\/NEN.0b013e3181815994","article-title":"Temporal neuropathologic and behavioral phenotype of 6neo\/6neo Pompe disease mice","volume":"67","author":"Sidman","year":"2008","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/mt.2013.96_bib17","doi-asserted-by":"crossref","first-page":"502","DOI":"10.1038\/mt.2009.305","article-title":"Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease","volume":"18","author":"Mah","year":"2010","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.96_bib18","doi-asserted-by":"crossref","first-page":"31","DOI":"10.3389\/fphys.2011.00031","article-title":"Hypoglossal neuropathology and respiratory activity in pompe mice","volume":"2","author":"Lee","year":"2011","journal-title":"Front Physiol"},{"key":"10.1038\/mt.2013.96_bib19","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1089\/hgtb.2012.009","article-title":"Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9","volume":"23","author":"ElMallah","year":"2012","journal-title":"Hum Gene Ther Methods"},{"key":"10.1038\/mt.2013.96_bib20","doi-asserted-by":"crossref","first-page":"21","DOI":"10.1038\/mt.2011.214","article-title":"Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice","volume":"20","author":"Qiu","year":"2012","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.96_bib21","doi-asserted-by":"crossref","first-page":"244","DOI":"10.1016\/j.jneumeth.2009.06.016","article-title":"Retrograde labeling of phrenic motoneurons by intrapleural injection","volume":"182","author":"Mantilla","year":"2009","journal-title":"J Neurosci Methods"},{"key":"10.1038\/mt.2013.96_bib22","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1186\/1479-0556-6-13","article-title":"Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice","volume":"6","author":"Pacak","year":"2008","journal-title":"Genet Vaccines Ther"},{"key":"10.1038\/mt.2013.96_bib23","doi-asserted-by":"crossref","first-page":"501","DOI":"10.1038\/sj.mt.6300100","article-title":"Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors","volume":"15","author":"Mah","year":"2007","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.96_bib24","series-title":"American Society of Gene Therapy 9th Annual Meeting","article-title":"rAAV2\/9 mediated gene delivery of acid \u03b1-glucosidase corrects the cardiac phenotype in a mouse model of Pompe disease","author":"Pacak","year":"2006"},{"key":"10.1038\/mt.2013.96_bib25","doi-asserted-by":"crossref","first-page":"1405","DOI":"10.1038\/sj.gt.3302550","article-title":"Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors","volume":"12","author":"Mah","year":"2005","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2013.96_bib26","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1089\/hum.2005.16.68","article-title":"Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II","volume":"16","author":"Cresawn","year":"2005","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2013.96_bib27","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1542\/peds.112.2.332","article-title":"The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature","volume":"112","author":"van den Hout","year":"2003","journal-title":"Pediatrics"},{"key":"10.1038\/mt.2013.96_bib28","first-page":"45","article-title":"Role of autophagy in the pathogenesis of Pompe disease","volume":"26","author":"Raben","year":"2007","journal-title":"Acta Myol"},{"issue":"5 Pt 1","key":"10.1038\/mt.2013.96_bib29","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1006\/mthe.2002.0580","article-title":"Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors","volume":"5","author":"Fraites","year":"2002","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.96_bib30","doi-asserted-by":"crossref","first-page":"3007","DOI":"10.1242\/dev.01169","article-title":"Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease","volume":"131","author":"Rucker","year":"2004","journal-title":"Development"},{"issue":"suppl. 10","key":"10.1038\/mt.2013.96_bib31","doi-asserted-by":"crossref","first-page":"S347","DOI":"10.1097\/CCM.0b013e3181b6e760","article-title":"Prolonged mechanical ventilation alters diaphragmatic structure and function","volume":"37","author":"Powers","year":"2009","journal-title":"Crit Care Med"},{"key":"10.1038\/mt.2013.96_bib32","doi-asserted-by":"crossref","first-page":"894","DOI":"10.1212\/WNL.19.9.894","article-title":"Pompe's disease","volume":"19","author":"Hogan","year":"1969","journal-title":"Neurology"},{"key":"10.1038\/mt.2013.96_bib33","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1016\/j.pediatrneurol.2010.02.006","article-title":"Acute progression of neuromuscular findings in infantile Pompe disease","volume":"42","author":"Burrow","year":"2010","journal-title":"Pediatr Neurol"},{"key":"10.1038\/mt.2013.96_bib34","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1007\/s10545-010-9075-9","article-title":"Pathophysiology of neuropathic lysosomal storage disorders","volume":"33","author":"Bellettato","year":"2010","journal-title":"J Inherit Metab Dis"},{"key":"10.1038\/mt.2013.96_bib35","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/j.ymthe.2004.07.016","article-title":"Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6","volume":"10","author":"Blankinship","year":"2004","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2013.96_bib36","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1089\/hum.2009.126","article-title":"Long-term luciferase expression monitored by bioluminescence imaging after adeno-associated virus-mediated fetal gene delivery in rhesus monkeys (Macaca mulatta)","volume":"21","author":"Tarantal","year":"2010","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2013.96_bib37","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mt.2013.96_bib38","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1089\/hum.2009.131","article-title":"Efficient retrograde transport of adeno-associated virus type 8 to spinal cord and dorsal root ganglion after vector delivery in muscle","volume":"21","author":"Zheng","year":"2010","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2013.96_bib39","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1038\/gt.2012.27","article-title":"Axonal transport of adeno-associated viral vectors is serotype-dependent","volume":"20","author":"Salegio","year":"2013","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2013.96_bib40","doi-asserted-by":"crossref","first-page":"2407","DOI":"10.1073\/pnas.0810682106","article-title":"Efficient gene therapy-based method for the delivery of therapeutics to primate cortex","volume":"106","author":"Kells","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2013.96_bib41","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1016\/j.virol.2005.06.051","article-title":"A combination of mutations enhances the neurotropism of AAV-2","volume":"341","author":"Xu","year":"2005","journal-title":"Virology"},{"key":"10.1038\/mt.2013.96_bib42","doi-asserted-by":"crossref","first-page":"1548","DOI":"10.1073\/pnas.86.5.1548","article-title":"Dynein is the motor for retrograde axonal transport of organelles","volume":"86","author":"Schnapp","year":"1989","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2013.96_bib43","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1016\/S1046-2023(02)00220-7","article-title":"Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors","volume":"28","author":"Zolotukhin","year":"2002","journal-title":"Methods"},{"key":"10.1038\/mt.2013.96_bib44","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.2013.96_bib45","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1177\/002215540505300108","article-title":"High-resolution light microscopy (HRLM) and digital analysis of Pompe disease pathology","volume":"53","author":"Lynch","year":"2005","journal-title":"J Histochem Cytochem"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616320007?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616320007?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,10,15]],"date-time":"2018-10-15T14:26:45Z","timestamp":1539613605000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616320007"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,9]]},"references-count":45,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2013,9]]}},"alternative-id":["S1525001616320007"],"URL":"https:\/\/doi.org\/10.1038\/mt.2013.96","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2013,9]]}}}