{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T11:37:54Z","timestamp":1791027474812,"version":"4.1.0"},"reference-count":56,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2012,9,19]],"date-time":"2012-09-19T00:00:00Z","timestamp":1348012800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01 HL081122"],"award-info":[{"award-number":["R01 HL081122"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000050","name":"National Heart, Lung, and Blood Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000050","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005202","name":"Muscular Dystrophy Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005202","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["faseb.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["The FASEB Journal"],"published-print":{"date-parts":[[2013,1]]},"abstract":"<jats:p>\n                    Pompe disease has resisted enzyme replacement therapy with acid \u03b1\u2010glucosidase (GAA), which has been attributed to inefficient cation\u2010independent mannose\u20106\u2010phosphate receptor (CI\u2010MPR) mediated uptake. We evaluated \u03b22\u2010agonist drugs, which increased CI\u2010MPR expression in GAA knockout (KO) mice. Clenbuterol along with a low\u2010dose adeno\u2010associated virus vector increased Rotarod latency by 75% at 4 wk, in comparison with vector alone (\n                    <jats:italic>P<\/jats:italic>\n                    &lt;2\u00d710\n                    <jats:sup>\u20135<\/jats:sup>\n                    ). Glycogen content was lower in skeletal muscles, including soleus (\n                    <jats:italic>P<\/jats:italic>\n                    &lt;0.01), extensor digitorum longus (EDL;\n                    <jats:italic>P<\/jats:italic>\n                    &lt; 0.001), and tibialis anterior (\n                    <jats:italic>P<\/jats:italic>\n                    &lt;0.05) following combination therapy, in comparison with vector alone. Glycogen remained elevated in the muscles following clenbuterol alone, indicating an adjunctive effect with gene therapy. Elderly GAA\u2010KO mice treated with combination therapy demonstrated 2\u2010fold increased wirehang latency, in comparison with vector or clenbuterol alone (\n                    <jats:italic>P<\/jats:italic>\n                    &lt;0.001). The glycogen content of skeletal muscle decreased following combination therapy in elderly mice (\n                    <jats:italic>P<\/jats:italic>\n                    &lt;0.05). Finally, CI\u2010MPR\u2010KO\/GAA\u2010KO mice did not respond to combination therapy, indicating that clenbuterol's effect depended on CI\u2010MPR expression. In summary, adjunctive \u03b22\u2010agonist treatment increased CI\u2010MPR expression and enhanced efficacy from gene therapy in Pompe disease, which has implications for other lysosomal storage disorders that involve primarily the brain.\u2014Li, S., Sun, B., Nilsson, M. I., Bird, A., Tarnopolsky, M. A., Thurberg, B. L., Bali, D., Koeberl, D. D. Adjunctive \u03b22\u2010agonists reverse neuromuscular involvement in murine Pompe disease.\n                    <jats:italic>FASEB J.<\/jats:italic>\n                    27, 34\u201344 (2013).\n                    <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" xlink:href=\"http:\/\/www.fasebj.org\">www.fasebj.org<\/jats:ext-link>\n                  <\/jats:p>","DOI":"10.1096\/fj.12-207472","type":"journal-article","created":{"date-parts":[[2012,9,19]],"date-time":"2012-09-19T22:29:51Z","timestamp":1348093791000},"page":"34-44","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":36,"title":["Adjunctive \u03b22\u2010agonists reverse neuromuscular involvement in murine Pompe disease"],"prefix":"10.1096","volume":"27","author":[{"given":"Songtao","family":"Li","sequence":"first","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Baodong","family":"Sun","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mats I.","family":"Nilsson","sequence":"additional","affiliation":[{"name":"Department of Pediatrics McMaster University Medical Center Hamilton Ontario Canada"},{"name":"Department of Medicine McMaster University Medical Center Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Bird","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark A.","family":"Tarnopolsky","sequence":"additional","affiliation":[{"name":"Department of Pediatrics McMaster University Medical Center Hamilton Ontario Canada"},{"name":"Department of Medicine McMaster University Medical Center Hamilton Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Beth L.","family":"Thurberg","sequence":"additional","affiliation":[{"name":"Department of Pathology Genzyme Corp. Framingham Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Deeksha","family":"Bali","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dwight D.","family":"Koeberl","sequence":"additional","affiliation":[{"name":"Division of Medical Genetics, Department of Pediatrics Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2012,9,19]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1023\/B:BOLI.0000031101.12838.c6"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2004.09.017"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.02.006"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/1755-8417-1-6"},{"key":"e_1_2_7_6_1","first-page":"712","article-title":"Carbohydrate\u2010remodeled acid alpha\u2010glucosidase with higher affinity for the cation\u2010independent mannose 6\u2010phosphate receptor demonstrates improved delivery to muscles of Pompe mice","volume":"15","author":"Zhu Y.","year":"2005","journal-title":"Neuromusc. Dis."},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.04.009"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.37"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2010.01.015"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-009-5275-3"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.wnl.0000251268.41188.04"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e31819d0996"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00455-009-9252-x"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.3928\/01913913-20100106-08"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.182412299"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2004.10.004"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2005.04.024"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.195"},{"key":"e_1_2_7_19_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm1358"},{"key":"e_1_2_7_20_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2007.001"},{"key":"e_1_2_7_21_1","doi-asserted-by":"publisher","DOI":"10.1038\/nm1445"},{"key":"e_1_2_7_22_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2008.15"},{"key":"e_1_2_7_23_1","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.16.8861"},{"key":"e_1_2_7_24_1","doi-asserted-by":"publisher","DOI":"10.1089\/hum.2005.16.68"},{"key":"e_1_2_7_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2003.08.022"},{"key":"e_1_2_7_26_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.archoralbio.2006.01.003"},{"key":"e_1_2_7_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1372"},{"key":"e_1_2_7_28_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.660"},{"key":"e_1_2_7_29_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9440(10)63823-0"},{"key":"e_1_2_7_30_1","doi-asserted-by":"publisher","DOI":"10.1369\/jhc.7A7239.2007"},{"key":"e_1_2_7_31_1","first-page":"277","article-title":"Antibody formation and mannose\u20106\u2010phosphate receptor expression impact the efficacy of muscle\u2010specific transgene expression in murine Pompe disease","volume":"25","author":"Sun B.","year":"2010","journal-title":"J. Gene Med."},{"key":"e_1_2_7_32_1","doi-asserted-by":"publisher","DOI":"10.1086\/522236"},{"key":"e_1_2_7_33_1","doi-asserted-by":"publisher","DOI":"10.1210\/en.2006-1646"},{"key":"e_1_2_7_34_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.305"},{"key":"e_1_2_7_35_1","doi-asserted-by":"publisher","DOI":"10.1152\/physrev.00028.2007"},{"key":"e_1_2_7_36_1","doi-asserted-by":"publisher","DOI":"10.1152\/physiol.00041.2007"},{"key":"e_1_2_7_37_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00424-006-0122-1"},{"key":"e_1_2_7_38_1","doi-asserted-by":"publisher","DOI":"10.1042\/bj2640573"},{"key":"e_1_2_7_39_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpendo.2002.282.1.E31"},{"key":"e_1_2_7_40_1","doi-asserted-by":"publisher","DOI":"10.1540\/jsmr.34.139"},{"key":"e_1_2_7_41_1","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2885.1998.00156.x"},{"key":"e_1_2_7_42_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1476-5381.1993.tb13929.x"},{"key":"e_1_2_7_43_1","first-page":"1446","article-title":"[Single and multiple applications and metabolite pattern of clenbuterol in man (author's transl.)]","volume":"26","author":"Zimmer A.","year":"1976","journal-title":"Arzneimittelforschung"},{"key":"e_1_2_7_44_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.57.8.1434"},{"key":"e_1_2_7_45_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000118530.71646.0F"},{"key":"e_1_2_7_46_1","doi-asserted-by":"publisher","DOI":"10.1212\/01.WNL.0000287070.00149.a9"},{"key":"e_1_2_7_47_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.healun.2008.01.013"},{"key":"e_1_2_7_48_1","doi-asserted-by":"crossref","first-page":"246","DOI":"10.1080\/14660820600600558","article-title":"A pilot trial with clenbuterol in amyotrophic lateral sclerosis","volume":"7","author":"Soraru G.","year":"2006","journal-title":"Amyotroph. Lateral. Scler."},{"key":"e_1_2_7_49_1","first-page":"71","article-title":"Adult acid maltase deficiency: an open trial with albuterol and branched\u2010chain aminoacids","volume":"14","author":"Angelini E. P. C.","year":"2004","journal-title":"Basic Appl. Myol."},{"key":"e_1_2_7_50_1","doi-asserted-by":"publisher","DOI":"10.1089\/rej.2009.0920"},{"key":"e_1_2_7_51_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.10002"},{"key":"e_1_2_7_52_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2011.11.005"},{"key":"e_1_2_7_53_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M110.189381"},{"key":"e_1_2_7_54_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00431-007-0635-4"},{"key":"e_1_2_7_55_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pharmthera.2008.06.003"},{"key":"e_1_2_7_56_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpregu.00183.2011"},{"key":"e_1_2_7_57_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2004-0718"}],"container-title":["The FASEB Journal"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1096\/fj.12-207472","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1096\/fj.12-207472","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/faseb.onlinelibrary.wiley.com\/doi\/pdf\/10.1096\/fj.12-207472","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,28]],"date-time":"2025-10-28T19:54:05Z","timestamp":1761681245000},"score":1,"resource":{"primary":{"URL":"https:\/\/faseb.onlinelibrary.wiley.com\/doi\/10.1096\/fj.12-207472"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,9,19]]},"references-count":56,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2013,1]]}},"alternative-id":["10.1096\/fj.12-207472"],"URL":"https:\/\/doi.org\/10.1096\/fj.12-207472","archive":["Portico"],"relation":{},"ISSN":["0892-6638","1530-6860"],"issn-type":[{"value":"0892-6638","type":"print"},{"value":"1530-6860","type":"electronic"}],"subject":[],"published":{"date-parts":[[2012,9,19]]},"assertion":[{"value":"2012-05-05","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2012-09-04","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2012-09-19","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}