Clinical efficacy of gene-modified stem cells in adenosine deaminase-deficient immunodeficiency

Journal of Clinical Investigation

Volumn 127, Issue 5, 2017, Pages 1689-1699

  Shaw, Kit L ^a   Garabedian, Elizabeth ^b   Mishra, Suparna ^a,n   Barman, Provaboti ^a   Davila, Alejandra ^a   Carbonaro, Denise ^a,o   Shupien, Sally ^a   Silvin, Christopher ^b,p   Geiger, Sabine ^a,q   Nowicki, Barbara ^c   Smogorzewska, E Monika ^d   Brown, Berkley ^a   Wang, Xiaoyan ^a   De Oliveira, Satiro ^a   Choi, Yeong ^a,r   Ikeda, Alan ^e   Terrazas, Dayna ^a   Fu, Pei Yu ^a   Yu, Allen ^a   Fernandez, Beatriz Campo ^a   Cooper, Aaron R ^a   Engel, Barbara ^d,s   Podsakoff, Greg ^d,s   Balamurugan, Arumugam ^a   Anderson, Stacie ^b   Muul, Linda ^b   Jagadeesh, G Jayashree ^b   Kapoor, Neena ^d   Tse, John ^c   Moore, Theodore B ^a   Purdy, Ken ^f   Rishi, Radha ^g   Mohan, Kathey ^h   Skoda Smith, Suzanne ^h   Buchbinder, David ⁱ   Abraham, Roshini S ^j   Scharenberg, Andrew ^k   Yang, Otto O ^a   Cornetta, Kenneth ^l   Gjertson, David ^a   Hershfield, Michael ^m   Sokolic, Rob ^b,t   Candotti, Fabio ^b,u   Kohn, Donald B ^a   more..

^a UNIVERSITY OF CALIFORNIA   (United States)

^b NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^c UCLA MEDICAL CENTER   (United States)

^d CHILDREN S HOSPITAL LOS ANGELES   (United States)

^e Childrens Specialty Centers of Nevada   (United States)

^f KAISER PERMANENTE   (United States)

^g Arizona Allergy Assoc   (United States)

^h SEATTLE CHILDREN S HOSPITAL   (United States)

ⁱ CHILDREN S HOSPITAL OF ORANGE COUNTY   (United States)

^j MAYO CLINIC   (United States)

^k UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^l INDIANA UNIVERSITY   (United States)

^m DUKE UNIVERSITY SCHOOL OF MEDICINE   (United States)

ⁿ Calimmune Australia Inc   (United States)

^o Orchard Therapeutics   (United Kingdom)

^p NATIONAL INSTITUTE ON DEAFNESS AND OTHER COMMUNICATION DISORDERS   (United States)

^q Apceth GmbH and Co KG   (Germany)

^r ROSWELL PARK CANCER INSTITUTE   (United States)

^s CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^t Federal Drug Administration   (United States)

^u LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ADENINE NUCLEOTIDE; B CELL ACTIVATING FACTOR; BUSULFAN; CD34 ANTIGEN; IMMUNOGLOBULIN G; INTERLEUKIN 7; ADA PROTEIN, HUMAN; ADENOSINE DEAMINASE;

ADENOSINE DEAMINASE DEFICIENCY; ADOLESCENT; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CD3+ T LYMPHOCYTE; CD4+ T LYMPHOCYTE; CHILD; CLINICAL ARTICLE; CONTROLLED STUDY; ENZYME ACTIVITY; ENZYME REPLACEMENT; FAILURE TO THRIVE; FOLLOW UP; GENE EXPRESSION; GRANULOCYTE; HUMAN; INFANT; INFECTIOUS COMPLICATION; LYMPHOCYTE COUNT; MYELOPOIESIS; NEUTROPENIA; PATIENT SAFETY; PERIPHERAL BLOOD MONONUCLEAR CELL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; REDUCED INTENSITY CONDITIONING; SIBLING; T LYMPHOCYTE ACTIVATION; AGAMMAGLOBULINEMIA; AUTOGRAFT; BIOSYNTHESIS; CLINICAL TRIAL; DEFICIENCY; ENZYMOLOGY; FEMALE; GENE EXPRESSION REGULATION; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MALE; PRESCHOOL CHILD; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY;

ADENOSINE DEAMINASE; ADOLESCENT; AGAMMAGLOBULINEMIA; AUTOGRAFTS; CHILD; CHILD, PRESCHOOL; FEMALE; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; RETROVIRIDAE; SEVERE COMBINED IMMUNODEFICIENCY; TRANSDUCTION, GENETIC;

EID: 85018744295     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI90367     Document Type: Article

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