Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction

Molecular Genetics and Metabolism

Volumn 117, Issue 2, 2016, Pages 66-83

  Kishnani, Priya S ^a   Dickson, Patricia I ^b   Muldowney, Laurie ^c   Lee, Jessica J ^c   Rosenberg, Amy ^c   Abichandani, Rekha ^d   Bluestone, Jeffrey A ^e   Burton, Barbara K ^f   Dewey, Maureen ^c   Freitas, Alexandra ^g   Gavin, Derek ^g   Griebel, Donna ^c   Hogan, Melissa ^h   Holland, Stephen ⁱ   Tanpaiboon, Pranoot ^j   Turka, Laurence A ^k   Utz, Jeanine J ^l   Wang, Yow Ming ^c   Whitley, Chester B ^l   Kazi, Zoheb B ^a   Pariser, Anne R ^c   more..

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b HARBOR UCLA MEDICAL CENTER   (United States)

^c CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^d SHIRE   (United States)

^e UNIVERSITY OF CALIFORNIA   (United States)

^f NORTHWESTERN UNIVERSITY   (United States)

^g National Organization for Rare Disorders   (United States)

^h A Hunter Syndrome Research Foundation   (United States)

ⁱ National MPS Society   (United States)

^j CHILDREN'S NATIONAL MEDICAL CENTER   (United States)

^k MASSACHUSETTS GENERAL HOSPITAL   (United States)

^l UNIVERSITY OF MINNESOTA   (United States)

more..

Author keywords

Enzyme replacement therapy; Immune tolerance; Inborn errors of metabolism; Lysosomal storage diseases; Neutralizing antibodies; Orphan drugs; Rare diseases

Indexed keywords

AGALSIDASE BETA; ALGLUCERASE; AZATHIOPRINE; BORTEZOMIB; CD19 ANTIGEN; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN; DEXAMETHASONE; DIPHTHERIA PERTUSSIS TETANUS VACCINE; DRUG ANTIBODY; ELOSULFASE ALFA; GALSULFASE; GLUCAN 1,4 ALPHA GLUCOSIDASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; IMMUNOGLOBULIN; LARONIDASE; METHOTREXATE; MYCOPHENOLIC ACID; OFATUMUMAB; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; RITUXIMAB; TALIGLUCERASE ALFA; VELAGLUCERASE ALFA; HYDROLASE; RECOMBINANT PROTEIN;

ANAPHYLAXIS; ANTIBODY RESPONSE; ANTIBODY TITER; AUTOIMMUNITY; CELL THERAPY; DISEASE COURSE; ENZYME REPLACEMENT; FABRY DISEASE; FOOD AND DRUG ADMINISTRATION; GAUCHER DISEASE; GENOTYPE; GLYCOGEN STORAGE DISEASE TYPE 2; HUMAN; HUMORAL IMMUNITY; HURLER SYNDROME; IMMUNE RESPONSE; IMMUNIZATION; IMMUNOGENICITY; IMMUNOLOGICAL TOLERANCE; LONG TERM CARE; LYSOSOME STORAGE DISEASE; MAROTEAUX LAMY SYNDROME; MONOTHERAPY; MORQUIO SYNDROME; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RASH; REGULATORY T LYMPHOCYTE; REVIEW; RISK BENEFIT ANALYSIS; SCHEIE SYNDROME; TREATMENT OUTCOME; URTICARIA; ANIMAL; IMMUNOLOGY; LYSOSOMAL STORAGE DISEASES;

ANIMALS; ENZYME REPLACEMENT THERAPY; HUMANS; HYDROLASES; IMMUNE TOLERANCE; LYSOSOMAL STORAGE DISEASES; RECOMBINANT PROTEINS;

EID: 84957894648     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2015.11.001     Document Type: Review

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