作者：Priya S. Kishnani^a ; ^{priya.kishnani@duke.edu" class="auth_mail" title="E-mail the corresponding author} ; Patricia I. Dickson^b ; ^{pdickson@labiomed.org" class="auth_mail" title="E-mail the corresponding author} ; Laurie Muldowney^c ; ^{laurie.muldowney@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Jessica J. Lee^c ; ^{jessica.j.lee@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Amy Rosenberg^d ; ^{amy.rosenberg@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Rekha Abichandani^e ; ^{rabichan@shire.com" class="auth_mail" title="E-mail the corresponding author} ; Jeffrey A. Bluestone^f ; ^{jeff.bluestone@ucsf.edu" class="auth_mail" title="E-mail the corresponding author} ; Barbara K. Burton^g ; ^{bburton@luriechildrens.org" class="auth_mail" title="E-mail the corresponding author} ; Maureen Dewey^c ; ^{maureen.dewey@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Alexandra Freitas^h ; ^{afreitas@rarediseases.org" class="auth_mail" title="E-mail the corresponding author} ; Derek Gavin^h ; ^{dgavin@rarediseases.org" class="auth_mail" title="E-mail the corresponding author} ; Donna Griebel^c ; ^{donna.griebel@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Melissa Hoganⁱ ; ^{melissa@savingcase.com" class="auth_mail" title="E-mail the corresponding author} ; Stephen Holland^j ; ^{steve.holland@thomsonreuters.com" class="auth_mail" title="E-mail the corresponding author} ; Pranoot Tanpaiboon^k ; ^{ptanpaib@childrensnational.org" class="auth_mail" title="E-mail the corresponding author} ; Laurence A. Turka^l ; ^{lturka@partners.org" class="auth_mail" title="E-mail the corresponding author} ; Jeanine J. Utz^m ; ^{utz002@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Yow-Ming Wangⁿ ; ^{yowming.wang@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author} ; Chester B. Whitley^m ; ^{whitley@umn.edu" class="auth_mail" title="E-mail the corresponding author} ; Zoheb B. Kazi^a ; ^{zoheb.kazi@dm.duke.edu" class="auth_mail" title="E-mail the corresponding author} ; Anne R. Pariser^o ; ^{anne.pariser@fda.hhs.gov" class="auth_mail" title="E-mail the corresponding author}

关键词：Ab ; antibody ; ADAs ; anti-drug antibodies ; Ag ; antigen ; BiPAP ; bilevel positive airway pressure ; CDER ; Center for Drug Evaluation and Research ; CNS ; central nervous system ; CRIM ; cross-reactive immunologic material ; DGIEP ; Division of Gastroenterology and Inborn Errors of Metabolism ; ERT ; enzyme replacement therapy ; FcR ; Fc receptor ; FDA ; Food and Drug Administration ; GAA ; acid alpha glucosidase ; GAG ; glycosaminoglycans ; HLA ; human leukocyte antigen ; HSAT ; high and sustained anti-rhGAA IgG antibo

刊名：Molecular Genetics and Metabolism

出版年：2016

作者：Ankit K. Desai ; Zoheb B. Kazi ; Priya S. Kishnani

刊名：Molecular Genetics and Metabolism

出版年：2016

作者：Takashi Matsuoka^a ; Yoshiyuki Miwa^a ; Makiko Tajika^a ; Madoka Sawada^a ; Koichiro Fujimaki^a ; Takashi Soga^a ; Hideshi Tomita^b ; Shigeru Uemura^b ; Ichizo Nishino^c ; Tokiko Fukuda^d ; Hideo Sugie^e ; Motomichi Kosuga^f ; ^{kosuga-mo@ncchd.go.jp} ; Torayuki Okuyama^g ; Yoh Umeda^a

刊名：Molecular Genetics and Metabolism Reports

出版年：2016

作者：Zhaohui Wang ; Patricia Okamoto ; Joan Keutzer

关键词：IOPD ; infantile-onset Pompe disease ; GAA ; acid 伪-glucosidase ; ERT ; enzyme replacement therapy ; rhGAA ; recombinant human GAA ; CRIM ; cross-reactive immunologic material ; ITI ; immune tolerance induction ; PBMC ; peripheral blood mononuclear cell ; PBS ; phosphate-buffered saline ; ACD ; citrate dextrose-A ; EDTA ; ethylenediaminetetraacetic acid ; BSA ; bovine serum albumin ; OD ; optical density ; LDS ; lithium dodecyl sulfate ; DTT ; dithiothreitol ; SDS ; sodium dodecyl sulfate ; PVDF ; polyvinylidene difluoride

刊名：Molecular Genetics and Metabolism

出版年：February, 2014

作者：Sari H. Pakkanen ; Jussi M. Kantele ; Christian Herzog ; Anu Kantele

关键词：ALS ; antibodies in lymphocyte supernatants ; ASC ; antibody-secreting cell ; CLA ; cutaneous lymphocyte antigen ; HR ; homing receptor ; iNTS ; invasive non-typhoid Salmonella ; NTS ; non-typhoid Salmonella ; PBMC ; peripheral blood mononuclear cells ; PBS ; phosphate buffered saline

刊名：Vaccine

出版年：23 January, 2014

作者：M.I. Nilsson ; M.A. Kroos ; A.J. Reuser ; E. Hatcher ; M. Akhtar ; M.E. McCready ; M.A. Tarnopolsky

关键词：4-MU ; 4-methylumbelliferone ; A ; adenosine ; AO ; adult onset ; Asp ; aspartic acid ; Arg ; arginine ; C ; cytidine ; cDNA ; DNA complementary to RNA ; CON ; control(s) ; CK ; creatine kinase ; CRIM ; cross-reactive immunologic material ; Cys ; cysteine ; ERT ; enzyme replacement therapy ; FEV ; forced expiratory volume ; FVC ; forced vital capacity ; G ; guanosine ; GAA ; acid 伪-glucosidase (gene) ; GAA ; acid 伪-glucosidase (protein) ; Gly ; glycine ; GSD II ; glycogen storage disorder type II ; HET ; heterozygous ; HRP ; horserad

刊名：Gene

出版年：1 March, 2014

作者：Brian R. Vuillemenot^a ; ^{bvuillemenot@bmrn.com" class="auth_mail} ; Derek Kennedy^a ; Randall P. Reed^b ; Robert B. Boyd^b ; Mark T. Butt^c ; Donald G. Musson^a ; Steve Keve^a ; Rhea Cahayag^a ; Laurie S. Tsuruda^a ; Charles A. O'Neill^a

关键词：aCSF ; artificial cerebrospinal fluid ; AUC ; area under the curve ; CHO ; Chinese hamster ovary ; CLN2 ; ceroid lipofuscinosis neuronal type 2 ; Cmax ; maximal concentration ; CRIM ; cross-reactive immunological material ; D ; deep tissue ; ERT ; enzyme replacement therapy ; EU ; endotoxin unit ; H&E ; hematoxylin and eosin ; GFAP ; glial fibrillary acidic protein ; ICV ; intracerebroventricular ; IT ; intrathecal ; IT-L ; intrathecal-lumbar ; LLOQ ; lower limit of quantification ; NBF ; neutral buffered formalin ; ND ; not de

刊名：Toxicology and Applied Pharmacology

出版年：15 May, 2014

作者：Kathryn B. Sheets ; Priya S. Kishnani ; Zoheb Kazi ; Stephanie M. DeArmey ; Deeksha S. Bali ; Jian Dai ; Catherine W. Rehder

刊名：Molecular Genetics and Metabolism

出版年：February, 2014

作者：Melissa E. Elder ; Sushrusha Nayak ; Shelley W. Collins ; Lee Ann Lawson ; Jeffry S. Kelley ; Rol ; W. Herzog ; Renee F. Modica ; Judy Lew ; Robert M. Lawrence ; Barry J. Byrne

关键词：Ab ; Antibody ; CRIM ; Cross-reactive immunologic material ; ERT ; Enzyme replacement therapy ; GAA ; Acid alpha-glucosidase ; IV ; Intravenous ; IVIG ; Intravenous immunoglobulin ; LVMI ; Left ventricular mass index

刊名：The Journal of Pediatrics

出版年：2013

作者：Priya S. Kishnani ; Paula C. Goldenberg ; Stephanie L. DeArmey ; James Heller ; Danny Benjamin ; Sarah Young ; Deeksha Bali ; Sue Ann Smith ; Jennifer S. Li ; Hanna M ; el ; Dwight Koeberl ; Amy Rosenberg ; Y-T Che

关键词：Pompe disease ; Glycogen storage disease ; Enzyme replacement therapy ; Lysosomal storage disease ; Acid alpha glucosidase ; GAA ; Cross-reactive immunologic material ; CRIM ; Antibody

刊名：Molecular Genetics and Metabolism

出版年：2010