Outcomes in Two Japanese Adenosine Deaminase-Deficiency Patients Treated by Stem Cell Gene Therapy with No Cytoreductive Conditioning

Journal of Clinical Immunology

Volumn 35, Issue 4, 2015, Pages 384-398

  Otsu, Makoto ^a,b   Yamada, Masafumi ^b   Nakajima, Satoru ^b   Kida, Miyuki ^b   Maeyama, Yoshihiro ^b   Hatano, Norikazu ^b   Toita, Nariaki ^b   Takezaki, Shunichiro ^b   Okura, Yuka ^b   Kobayashi, Ryoji ^b   Matsumoto, Yoshinori ^b   Tatsuzawa, Osamu ^c   Tsuchida, Fumiko ^d   Kato, Shunichi ^d   Kitagawa, Masanari ^e   Mineno, Junichi ^e   Hershfield, Michael S ^f   Bali, Pawan ^f   Candotti, Fabio ^g   Onodera, Masafumi ^c   Kawamura, Nobuaki ^b   Sakiyama, Yukio ^b   Ariga, Tadashi ^b   more..

^a UNIVERSITY OF TOKYO   (Japan)

^b HOKKAIDO UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^c NATIONAL CENTER FOR CHILD HEALTH AND DEVELOPMENT   (Japan)

^d TOKAI UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^e Takara Bio   (Japan)

^f DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^g NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

Author keywords

adenosine deaminase (ADA); Gene therapy; hematopoietic stem cell(s); primary immunodeficiency (PID); retroviral vector(s); severe combined immunodeficiency (SCID)

Indexed keywords

ADENOSINE DEAMINASE; ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CELL THERAPY AGENT; COMPLEMENTARY DNA; RETROVIRUS VECTOR; PEGADEMASE;

ADENOSINE DEAMINASE DEFICIENCY; ADOLESCENT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; BONE MARROW CELL; CASE REPORT; CD3+ T LYMPHOCYTE; CHILD; DETOXIFICATION; DNA VECTOR; DRUG DETOXIFICATION; DRUG WITHDRAWAL; ENGRAFTMENT; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; FLOW CYTOMETRY; GASTROINTESTINAL SYMPTOM; GENETIC TRANSDUCTION; HEMATOPOIESIS; HEMATOPOIETIC CELL; HUMAN; LYMPHOCYTE COUNT; LYMPHOCYTE SUBPOPULATION; MALE; NATURAL KILLER CELL; OUTCOME ASSESSMENT; PERIPHERAL BLOOD MONONUCLEAR CELL; PERIPHERAL LYMPHOCYTE; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN EXPRESSION; STEM CELL GENE THERAPY; THIN LAYER CHROMATOGRAPHY; THYMIDINE INCORPORATION ASSAY; VIRAL GENE DELIVERY SYSTEM; AGAMMAGLOBULINEMIA; CELL DIFFERENTIATION; CYTOLOGY; DEFICIENCY; ENZYME ACTIVATION; GAMMARETROVIRUS; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNITY; IMMUNOLOGY; IMMUNOPHENOTYPING; INFANT; JAPAN; METABOLISM; MUTATION; NEWBORN; ONSET AGE; SEVERE COMBINED IMMUNODEFICIENCY; TRANSGENE; TREATMENT OUTCOME;

ADENOSINE DEAMINASE; ADOLESCENT; AGAMMAGLOBULINEMIA; AGE OF ONSET; CELL DIFFERENTIATION; CHILD, PRESCHOOL; ENZYME ACTIVATION; ENZYME REPLACEMENT THERAPY; GAMMARETROVIRUS; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IMMUNITY; IMMUNOPHENOTYPING; INFANT; INFANT, NEWBORN; JAPAN; LYMPHOCYTE SUBSETS; MUTATION; SEVERE COMBINED IMMUNODEFICIENCY; TRANSDUCTION, GENETIC; TRANSGENES; TREATMENT OUTCOME;

EID: 84929841679     PISSN: 02719142     EISSN: 15732592     Source Type: Journal    
DOI: 10.1007/s10875-015-0157-1     Document Type: Article

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