{"id":"https://openalex.org/W1746965339","doi":null,"title":"AAV-MEDIATED GENE TRANSFER TO MODELS OF MUSCULAR DYSTROPHY: INSIGHTS INTO ASSEMBLY OF MULTI-SUBUNIT MEMBRANE PROTEINS.","display_name":"AAV-MEDIATED GENE TRANSFER TO MODELS OF MUSCULAR DYSTROPHY: INSIGHTS INTO ASSEMBLY OF MULTI-SUBUNIT MEMBRANE PROTEINS.","publication_year":2002,"publication_date":"2002-04-25","ids":{"openalex":"https://openalex.org/W1746965339","mag":"1746965339"},"language":"en","primary_location":{"id":"pmh:oai:d-scholarship.pitt.edu:7473","doi":null,"is_oa":true,"landing_page_url":"https://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","pdf_url":"http://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","source":{"id":"https://openalex.org/S4306402372","display_name":"D-Scholarship@Pitt (University of Pittsburgh)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I170201317","host_organization_name":"University of Pittsburgh","host_organization_lineage":["https://openalex.org/I170201317"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":null,"raw_type":"PeerReviewed"},"volume":null,"type":"article","is_traditional":true,"indexed_in":[],"open_access":{"is_oa":true,"oa_status":"green","oa_url":"http://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","any_repository_has_fulltext":true},"authorships":[{"author_position":"first","author":{"id":"https://openalex.org/A5040584147","display_name":"Devin C. Dressman","orcid":null,"observed_orcids":[]},"institutions":[],"countries":[],"is_corresponding":true,"raw_author_name":"Devin Dressman","raw_affiliation_strings":[],"raw_orcid":null,"affiliations":[]}],"institutions":[],"countries_distinct_count":0,"institutions_distinct_count":0,"corresponding_author_ids":["https://openalex.org/A5040584147"],"corresponding_institution_ids":[],"apc_list":null,"apc_paid":null,"fwci":0.0,"has_fulltext":true,"cited_by_count":0,"citation_normalized_percentile":{"value":0.1143986,"is_in_top_1_percent":false,"is_in_top_10_percent":false},"cited_by_percentile_year":null,"biblio":{"volume":null,"issue":null,"first_page":null,"last_page":null},"is_retracted":false,"is_paratext":false,"is_xpac":false,"primary_topic":{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.8935999870300293,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},"topics":[{"id":"https://openalex.org/T10613","display_name":"Virus-based gene therapy research","score":0.8935999870300293,"subfield":{"id":"https://openalex.org/subfields/1311","display_name":"Genetics"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T10441","display_name":"Muscle Physiology and Disorders","score":0.07769999653100967,"subfield":{"id":"https://openalex.org/subfields/1312","display_name":"Molecular Biology"},"field":{"id":"https://openalex.org/fields/13","display_name":"Biochemistry, Genetics and Molecular Biology"},"domain":{"id":"https://openalex.org/domains/1","display_name":"Life Sciences"}},{"id":"https://openalex.org/T12400","display_name":"Neurogenetic and Muscular Disorders Research","score":0.01119999960064888,"subfield":{"id":"https://openalex.org/subfields/2716","display_name":"Genetics (clinical)"},"field":{"id":"https://openalex.org/fields/27","display_name":"Medicine"},"domain":{"id":"https://openalex.org/domains/4","display_name":"Health Sciences"}}],"keywords":[{"id":"https://openalex.org/keywords/adeno-associated-virus","display_name":"adeno-associated virus","score":0.7239999771118164},{"id":"https://openalex.org/keywords/gene-therapy","display_name":"gene therapy","score":0.7720000147819519},{"id":"https://openalex.org/keywords/limb-girdle-muscular-dystrophy","display_name":"limb-girdle muscular dystrophy","score":0.8980000019073486},{"id":"https://openalex.org/keywords/sarcoglycanopathies","display_name":"sarcoglycanopathies","score":0.9710000157356262},{"id":"https://openalex.org/keywords/delta-sarcoglycan","display_name":"delta-sarcoglycan","score":0.9480000138282776},{"id":"https://openalex.org/keywords/beta-sarcoglycan","display_name":"beta-sarcoglycan","score":0.9490000009536743},{"id":"https://openalex.org/keywords/alpha-sarcoglycan","display_name":"alpha-sarcoglycan","score":0.9980000257492065},{"id":"https://openalex.org/keywords/dystrophic-muscle","display_name":"dystrophic muscle","score":0.6639999747276306}],"concepts":[{"id":"https://openalex.org/C86803240","wikidata":"https://www.wikidata.org/wiki/Q420","display_name":"Biology","level":0,"score":0.6823395490646362},{"id":"https://openalex.org/C135983454","wikidata":"https://www.wikidata.org/wiki/Q2790163","display_name":"Gene delivery","level":4,"score":0.6591707468032837},{"id":"https://openalex.org/C111599444","wikidata":"https://www.wikidata.org/wiki/Q213901","display_name":"Genetic enhancement","level":3,"score":0.6450648903846741},{"id":"https://openalex.org/C2779030066","wikidata":"https://www.wikidata.org/wiki/Q1137767","display_name":"Muscular dystrophy","level":2,"score":0.6014639139175415},{"id":"https://openalex.org/C8891405","wikidata":"https://www.wikidata.org/wiki/Q1059","display_name":"Immune system","level":2,"score":0.46770569682121277},{"id":"https://openalex.org/C2778649560","wikidata":"https://www.wikidata.org/wiki/Q1531322","display_name":"Limb-girdle muscular dystrophy","level":4,"score":0.4645886719226837},{"id":"https://openalex.org/C77957584","wikidata":"https://www.wikidata.org/wiki/Q1133550","display_name":"Gene knockout","level":3,"score":0.4320603609085083},{"id":"https://openalex.org/C203014093","wikidata":"https://www.wikidata.org/wiki/Q101929","display_name":"Immunology","level":1,"score":0.4200217127799988},{"id":"https://openalex.org/C95444343","wikidata":"https://www.wikidata.org/wiki/Q7141","display_name":"Cell biology","level":1,"score":0.40406370162963867},{"id":"https://openalex.org/C153911025","wikidata":"https://www.wikidata.org/wiki/Q7202","display_name":"Molecular biology","level":1,"score":0.37786391377449036},{"id":"https://openalex.org/C104317684","wikidata":"https://www.wikidata.org/wiki/Q7187","display_name":"Gene","level":2,"score":0.3379107117652893},{"id":"https://openalex.org/C501734568","wikidata":"https://www.wikidata.org/wiki/Q42918","display_name":"Mutation","level":3,"score":0.17659366130828857},{"id":"https://openalex.org/C54355233","wikidata":"https://www.wikidata.org/wiki/Q7162","display_name":"Genetics","level":1,"score":0.15493395924568176}],"mesh":[],"locations_count":2,"locations":[{"id":"pmh:oai:d-scholarship.pitt.edu:7473","doi":null,"is_oa":true,"landing_page_url":"https://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","pdf_url":"http://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","source":{"id":"https://openalex.org/S4306402372","display_name":"D-Scholarship@Pitt (University of Pittsburgh)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I170201317","host_organization_name":"University of Pittsburgh","host_organization_lineage":["https://openalex.org/I170201317"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":null,"raw_type":"PeerReviewed"},{"id":"mag:1746965339","doi":null,"is_oa":false,"landing_page_url":"http://d-scholarship.pitt.edu/7473/","pdf_url":null,"source":null,"license":null,"license_id":null,"version":null,"is_accepted":false,"is_published":false,"raw_source_name":null,"raw_type":null}],"best_oa_location":{"id":"pmh:oai:d-scholarship.pitt.edu:7473","doi":null,"is_oa":true,"landing_page_url":"https://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","pdf_url":"http://d-scholarship.pitt.edu/7473/1/DevinDressmanDissertation200207.pdf","source":{"id":"https://openalex.org/S4306402372","display_name":"D-Scholarship@Pitt (University of Pittsburgh)","issn_l":null,"issn":null,"is_oa":false,"is_in_doaj":false,"is_core":false,"listed_in":[],"host_organization":"https://openalex.org/I170201317","host_organization_name":"University of Pittsburgh","host_organization_lineage":["https://openalex.org/I170201317"],"host_organization_lineage_names":[],"type":"repository"},"license":null,"license_id":null,"version":"acceptedVersion","is_accepted":true,"is_published":false,"raw_source_name":null,"raw_type":"PeerReviewed"},"sustainable_development_goals":[{"id":"https://metadata.un.org/sdg/3","display_name":"Good health and well-being","score":0.98580002784729}],"study_designs":[],"awards":[],"funders":[{"id":"https://openalex.org/F4320306230","display_name":"American Heart Association","ror":"https://ror.org/013kjyp64"},{"id":"https://openalex.org/F4320332161","display_name":"National Institutes of Health","ror":"https://ror.org/01cwqze88"}],"has_content":{"pdf":true,"grobid_xml":true},"content_urls":{"pdf":"https://content.openalex.org/works/W1746965339.pdf","grobid_xml":"https://content.openalex.org/works/W1746965339.grobid-xml"},"referenced_works_count":142,"referenced_works":["https://openalex.org/W1331881","https://openalex.org/W9301733","https://openalex.org/W41403280","https://openalex.org/W72019695","https://openalex.org/W116299411","https://openalex.org/W124592240","https://openalex.org/W186828966","https://openalex.org/W1488519169","https://openalex.org/W1491459594","https://openalex.org/W1494261909","https://openalex.org/W1504372166","https://openalex.org/W1505196125","https://openalex.org/W1519383882","https://openalex.org/W1525849227","https://openalex.org/W1533835843","https://openalex.org/W1572539012","https://openalex.org/W1575992755","https://openalex.org/W1582366477","https://openalex.org/W1593294300","https://openalex.org/W1883425081","https://openalex.org/W1901720315","https://openalex.org/W1942870285","https://openalex.org/W1949194573","https://openalex.org/W1965997845","https://openalex.org/W1966971028","https://openalex.org/W1968370663","https://openalex.org/W1968839934","https://openalex.org/W1970350643","https://openalex.org/W1970699741","https://openalex.org/W1971439416","https://openalex.org/W1972224752","https://openalex.org/W1972725992","https://openalex.org/W1974739948","https://openalex.org/W1974745886","https://openalex.org/W1974877896","https://openalex.org/W1975132105","https://openalex.org/W1975528602","https://openalex.org/W1976919537","https://openalex.org/W1979746830","https://openalex.org/W1980243949","https://openalex.org/W1980606567","https://openalex.org/W1985292788","https://openalex.org/W1985954761","https://openalex.org/W1986088843","https://openalex.org/W1988463041","https://openalex.org/W1992922486","https://openalex.org/W1993655674","https://openalex.org/W1994397207","https://openalex.org/W1995550032","https://openalex.org/W1996010358","https://openalex.org/W1999113624","https://openalex.org/W2003654592","https://openalex.org/W2008980136","https://openalex.org/W2010872781","https://openalex.org/W2011129367","https://openalex.org/W2013534167","https://openalex.org/W2014589399","https://openalex.org/W2016753696","https://openalex.org/W2016761722","https://openalex.org/W2018096072","https://openalex.org/W2018922749","https://openalex.org/W2019612944","https://openalex.org/W2021202201","https://openalex.org/W2023139238","https://openalex.org/W2023820753","https://openalex.org/W2023827769","https://openalex.org/W2027210778","https://openalex.org/W2028134688","https://openalex.org/W2033491178","https://openalex.org/W2035503063","https://openalex.org/W2035600956","https://openalex.org/W2042598793","https://openalex.org/W2044005085","https://openalex.org/W2046556168","https://openalex.org/W2051442157","https://openalex.org/W2055883057","https://openalex.org/W2057200625","https://openalex.org/W2058306260","https://openalex.org/W2062006811","https://openalex.org/W2064836072","https://openalex.org/W2065704959","https://openalex.org/W2066180106","https://openalex.org/W2067492921","https://openalex.org/W2067661389","https://openalex.org/W2068065419","https://openalex.org/W2069791967","https://openalex.org/W2070082633","https://openalex.org/W2070936696","https://openalex.org/W2072288907","https://openalex.org/W2073305844","https://openalex.org/W2073470325","https://openalex.org/W2074226696","https://openalex.org/W2074353166","https://openalex.org/W2082328650","https://openalex.org/W2082829109","https://openalex.org/W2090566743","https://openalex.org/W2091208245","https://openalex.org/W2096309023","https://openalex.org/W2099234787","https://openalex.org/W2100405455","https://openalex.org/W2109801676","https://openalex.org/W2112778291","https://openalex.org/W2118855079","https://openalex.org/W2119951665","https://openalex.org/W2120310826","https://openalex.org/W2123861384","https://openalex.org/W2125683839","https://openalex.org/W2128296855","https://openalex.org/W2128561011","https://openalex.org/W2129278356","https://openalex.org/W2139865704","https://openalex.org/W2143513370","https://openalex.org/W2148223197","https://openalex.org/W2154204467","https://openalex.org/W2154329712","https://openalex.org/W2154729967","https://openalex.org/W2155898643","https://openalex.org/W2159706578","https://openalex.org/W2161789277","https://openalex.org/W2162431196","https://openalex.org/W2163106313","https://openalex.org/W2164418621","https://openalex.org/W2172045470","https://openalex.org/W2174500521","https://openalex.org/W2174994066","https://openalex.org/W2175291125","https://openalex.org/W2209574353","https://openalex.org/W2263291280","https://openalex.org/W2320022870","https://openalex.org/W2320848983","https://openalex.org/W2322780762","https://openalex.org/W2325063647","https://openalex.org/W2329473893","https://openalex.org/W2330930141","https://openalex.org/W2335173351","https://openalex.org/W2340880118","https://openalex.org/W2397236369","https://openalex.org/W2433861420","https://openalex.org/W2437299318","https://openalex.org/W2822822418","https://openalex.org/W2876950260","https://openalex.org/W2900590395"],"related_works":["https://openalex.org/W1997946559","https://openalex.org/W2062006811","https://openalex.org/W2065704959","https://openalex.org/W2048338867","https://openalex.org/W2944008019","https://openalex.org/W7074097248","https://openalex.org/W2134827380","https://openalex.org/W222157178","https://openalex.org/W2174994066","https://openalex.org/W2325063647"],"abstract_inverted_index":{"The":[0,107],"sarcoglycanopathies":[1],"are":[2,157],"a":[3,34,293],"subset":[4],"of":[5,62,76,84,116,126,138,144,175,201,233,241,247,268,276,321,340,346],"the":[6,16,49,172,199,248,251,272],"limb":[7],"girdle":[8],"muscular":[9],"dystrophies":[10],"(LGMD)":[11],"caused":[12],"by":[13,236],"mutations":[14],"in":[15,216],"sarcoglycan":[17,117],"genes":[18,316],"(alpha,":[19],"beta,":[20],"gamma":[21],"and":[22,44,70,86,136,212,218,231,239,265,274,313,342],"delta).":[23],"In":[24],"collaborative":[25],"studies,":[26],"delta-sarcoglycan":[27],"was":[28,91,308],"delivered":[29,196],"to":[30,54,64,78,105,133,195,211,262,310,317,331],"deficient":[31,65,79],"hamsters":[32],"using":[33],"recombinant":[35],"adeno-associated":[36],"virus":[37],"(AAV),":[38],"which":[39,189,307],"rescued":[40],"muscle":[41,147,226,258],"biochemically,":[42],"histologically,":[43],"functionally.":[45],"Murine":[46],"knockouts":[47],"for":[48],"other":[50],"sarcoglycans":[51],"permitted":[52],"us":[53],"pursue":[55],"AAV-mediated":[56,59,73,124,296],"gene":[57,60,74,145,163,202,277,297,332],"delivery.":[58,203,278,333],"delivery":[61,75,125,164,300],"beta-sarcoglycan":[63],"mice":[66,80],"provided":[67],"long-term":[68],"biochemical":[69,85,153,245,284,347],"histological":[71,87],"rescue.":[72],"alpha-sarcoglycan":[77,102],"showed":[81,227],"initial":[82],"rescue":[83,246,285,345],"defects,":[88],"although":[89],"expression":[90],"not":[92],"persistent.":[93],"Severe":[94],"Combined":[95],"Immune-Deficient":[96],"(SCID)":[97],"mouse":[98],"studies":[99,121],"indicated":[100,255],"that":[101,123,161,180,256,266,282],"over-expression":[103],"leads":[104],"cytotoxicity.":[106],"apparent":[108],"cytotoxicity":[109],"can":[110,131,141],"be":[111,187],"interpreted":[112],"with":[113,222],"emerging":[114],"models":[115],"complex":[118],"assembly.":[119],"These":[120],"show":[122],"even":[127],"closely":[128],"related":[129],"proteins":[130],"lead":[132],"different":[134],"outcomes,":[135],"aspects":[137,267],"protein":[139],"biochemistry":[140],"alter":[142],"efficacy":[143,275],"delivery.Inherited":[146],"disorders":[148],"typically":[149],"have":[150],"defined":[151],"primary":[152],"defects.":[154,348],"However,":[155],"there":[156],"likely":[158],"secondary":[159],"responses":[160,264],"mitigate":[162],"success.":[165],"To":[166,204],"dissect":[167],"such":[168],"variables,":[169],"we":[170,207],"studied":[171],"immunostimulatory":[173,288],"properties":[174],"dystrophic":[176,219,225,257],"muscle.":[177,220,322],"We":[178,290,302],"hypothesized":[179],"immune":[181,193,229,252,263],"cell":[182],"infiltrate":[183],"accompanying":[184],"degeneration/regeneration":[185],"could":[186,190],"immunostimulatory,":[188],"elicit":[191],"an":[192,304],"response":[194,210],"transgenes,":[197],"hampering":[198],"success":[200],"study":[205],"this,":[206],"tested":[208],"antibody":[209,237],"persistence":[213,273],"of,":[214],"beta-galactosidase":[215],"normal":[217],"Consistent":[221],"our":[223],"hypothesis,":[224],"increased":[228],"surveillance":[230],"recognition":[232],"beta-galactosidase,":[234],"evidenced":[235],"titers":[238],"clearance":[240],"transduced":[242],"cells.":[243],"Furthermore,":[244],"dystrophy":[249],"quenched":[250],"response.":[253],"This":[254],"is":[259],"more":[260,327],"prone":[261],"tissue":[269],"pathology":[270],"influence":[271],"Our":[279],"results":[280,326],"suggest":[281],"full":[283,344],"will":[286,336],"attenuate":[287],"effects.":[289],"also":[291],"address":[292],"hurdle":[294],"facing":[295],"therapy;":[298],"namely,":[299],"methods.":[301],"developed":[303],"injection":[305],"manifold,":[306],"used":[309],"safely,":[311],"accurately,":[312],"consistently":[314],"deliver":[315],"20":[318],"mm2":[319],"regions":[320],"Taken":[323],"together,":[324],"these":[325],"clearly":[328],"define":[329],"barriers":[330],"Future":[334],"research":[335],"finely":[337],"tune":[338],"regulation":[339],"transgenes":[341],"enable":[343]},"counts_by_year":[],"updated_date":"2026-11-04T10:54:05.112534","created_date":"2016-06-24T00:00:00"}