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  1. Article ; Online: CD22 CAR T cells demonstrate high response rates and safety in pediatric and adult B-ALL: Phase 1b results.

    Schultz, Liora M / Jeyakumar, Nikeshan / Kramer, Anne Marijn / Sahaf, Bita / Srinagesh, Hrishi / Shiraz, Parveen / Agarwal, Neha / Hamilton, Mark / Erickson, Courtney / Jacobs, Ashley / Moon, Jennifer / Baggott, Christina / Arai, Sally / Bharadwaj, Sushma / Johnston, Laura J / Liedtke, Michaela / Lowsky, Robert / Meyer, Everett / Negrin, Robert /
    Rezvani, Andrew / Shizuru, Judy / Sidana, Surbhi / Egeler, Emily / Mavroukakis, Sharon / Tunuguntla, Ramya / Gkitsas-Long, Nikolaos / Retherford, Aidan / Brown, Annie Kathleen / Gramstrap-Petersen, Anne-Louise / Ibañez, Raquel Martin / Feldman, Steven A / Miklos, David B / Mackall, Crystal L / Davis, Kara L / Frank, Matthew / Ramakrishna, Sneha / Muffly, Lori

    Leukemia

    2024  Volume 38, Issue 5, Page(s) 963–968

    Abstract: Chimeric antigen receptor (CAR) T cells targeting CD22 (CD22-CAR) provide a therapeutic option for patients with ... ...

    Abstract Chimeric antigen receptor (CAR) T cells targeting CD22 (CD22-CAR) provide a therapeutic option for patients with CD22
    MeSH term(s) Humans ; Sialic Acid Binding Ig-like Lectin 2/immunology ; Child ; Adult ; Female ; Male ; Adolescent ; Immunotherapy, Adoptive/methods ; Immunotherapy, Adoptive/adverse effects ; Young Adult ; Receptors, Chimeric Antigen/immunology ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/therapy ; Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/immunology ; Child, Preschool ; Middle Aged ; T-Lymphocytes/immunology ; T-Lymphocytes/metabolism
    Chemical Substances Sialic Acid Binding Ig-like Lectin 2 ; CD22 protein, human ; Receptors, Chimeric Antigen
    Language English
    Publishing date 2024-03-15
    Publishing country England
    Document type Journal Article ; Clinical Trial, Phase I ; Research Support, Non-U.S. Gov't ; Research Support, N.I.H., Extramural
    ZDB-ID 807030-1
    ISSN 1476-5551 ; 0887-6924
    ISSN (online) 1476-5551
    ISSN 0887-6924
    DOI 10.1038/s41375-024-02220-y
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  2. Article ; Online: A trial of plerixafor adjunctive therapy in allogeneic hematopoietic cell transplantation with minimal conditioning for severe combined immunodeficiency.

    Dvorak, Christopher C / Horn, Biljana N / Puck, Jennifer M / Czechowicz, Agnieszka / Shizuru, Judy A / Ko, Rose M / Cowan, Morton J

    Pediatric transplantation

    2014  Volume 18, Issue 6, Page(s) 602–608

    Abstract: For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with G-CSF plus plerixafor given to the host to mobilize ... ...

    Abstract For infants with SCID, the ideal conditioning regimen before allogeneic HCT would omit cytotoxic chemotherapy to minimize short- and long-term complications. We performed a prospective pilot trial with G-CSF plus plerixafor given to the host to mobilize HSC from their niches. We enrolled six patients who received CD34-selected haploidentical cells and one who received T-replete matched unrelated BM. All patients receiving G-CSF and plerixafor had generally poor CD34(+) cell and Lin(-) CD34(+) CD38(-) CD90(+) CD45RA(-) HSC mobilization, and developed donor T cells, but no donor myeloid or B-cell engraftment. Although well tolerated, G-CSF plus plerixafor alone failed to overcome physical barriers to donor engraftment.
    MeSH term(s) Adolescent ; California ; Chemotherapy, Adjuvant ; Child ; Combined Modality Therapy ; Drug Therapy, Combination ; Female ; Granulocyte Colony-Stimulating Factor/therapeutic use ; Hematopoietic Stem Cell Mobilization ; Hematopoietic Stem Cell Transplantation ; Heterocyclic Compounds/therapeutic use ; Histocompatibility Testing ; Humans ; Infant ; Male ; Pilot Projects ; Prospective Studies ; Severe Combined Immunodeficiency/drug therapy ; Severe Combined Immunodeficiency/immunology ; Severe Combined Immunodeficiency/therapy ; Transplantation Conditioning ; Treatment Outcome
    Chemical Substances Heterocyclic Compounds ; Granulocyte Colony-Stimulating Factor (143011-72-7) ; plerixafor (S915P5499N)
    Language English
    Publishing date 2014-06-30
    Publishing country Denmark
    Document type Journal Article ; Research Support, N.I.H., Extramural
    ZDB-ID 1390284-2
    ISSN 1399-3046 ; 1397-3142
    ISSN (online) 1399-3046
    ISSN 1397-3142
    DOI 10.1111/petr.12309
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  3. Article ; Online: Strategic plan for lung vascular research: An NHLBI-ORDR Workshop Report.

    Erzurum, Serpil / Rounds, Sharon I / Stevens, Troy / Aldred, Micheala / Aliotta, Jason / Archer, Stephen L / Asosingh, Kewal / Balaban, Robert / Bauer, Natalie / Bhattacharya, Jahar / Bogaard, Harm / Choudhary, Gaurav / Dorn, Gerald W / Dweik, Raed / Fagan, Karen / Fallon, Michael / Finkel, Toren / Geraci, Mark / Gladwin, Mark T /
    Hassoun, Paul M / Humbert, Marc / Kaminski, Naftali / Kawut, Steven M / Loscalzo, Joseph / McDonald, Donald / McMurtry, Ivan F / Newman, John / Nicolls, Mark / Rabinovitch, Marlene / Shizuru, Judy / Oka, Masahiko / Polgar, Peter / Rodman, David / Schumacker, Paul / Stenmark, Kurt / Tuder, Rubin / Voelkel, Norbert / Sullivan, Eugene / Weinshilboum, Richard / Yoder, Mervin C / Zhao, Yingming / Gail, Dorothy / Moore, Timothy M

    American journal of respiratory and critical care medicine

    2010  Volume 182, Issue 12, Page(s) 1554–1562

    Abstract: The Division of Lung Diseases of the National Heart, Lung, and Blood Institute, with the Office of Rare Diseases Research, held a workshop to identify priority areas and strategic goals to enhance and accelerate research that will result in improved ... ...

    Abstract The Division of Lung Diseases of the National Heart, Lung, and Blood Institute, with the Office of Rare Diseases Research, held a workshop to identify priority areas and strategic goals to enhance and accelerate research that will result in improved understanding of the lung vasculature, translational research needs, and ultimately the care of patients with pulmonary vascular diseases. Multidisciplinary experts with diverse experience in laboratory, translational, and clinical studies identified seven priority areas and discussed limitations in our current knowledge, technologies, and approaches. The focus for future research efforts include the following: (1) better characterizing vascular genotype-phenotype relationships and incorporating systems biology approaches when appropriate; (2) advancing our understanding of pulmonary vascular metabolic regulatory signaling in health and disease; (3) expanding our knowledge of the biologic relationships between the lung circulation and circulating elements, systemic vascular function, and right heart function and disease; (4) improving translational research for identifying disease-modifying therapies for the pulmonary hypertensive diseases; (5) establishing an appropriate and effective platform for advancing translational findings into clinical studies testing; and (6) developing the specific technologies and tools that will be enabling for these goals, such as question-guided imaging techniques and lung vascular investigator training programs. Recommendations from this workshop will be used within the Lung Vascular Biology and Disease Extramural Research Program for planning and strategic implementation purposes.
    MeSH term(s) Biomedical Research/methods ; Guidelines as Topic ; Humans ; Lung/blood supply ; Lung Diseases/physiopathology ; Pulmonary Circulation
    Language English
    Publishing date 2010-09-10
    Publishing country United States
    Document type Journal Article ; Research Support, N.I.H., Extramural ; Review
    ZDB-ID 1180953-x
    ISSN 1535-4970 ; 0003-0805 ; 1073-449X
    ISSN (online) 1535-4970
    ISSN 0003-0805 ; 1073-449X
    DOI 10.1164/rccm.201006-0869WS
    Database MEDical Literature Analysis and Retrieval System OnLINE

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