Phase I/II gene therapy study for Chronic Granulomatous Disease: Results, lessons and perspectives

被引:1
|
作者
Ott, Marion G. [1 ]
Stein, Stefan [2 ]
Schultze-Strasser, Stephan [2 ]
Jauch, Anna [3 ]
Burwinkel, Barbara [4 ]
Schlegelberger, Brigitte [5 ]
Goehring, Gudrun [5 ]
Preiss, Carolin [2 ]
Kramer, Rolf [2 ]
Schwarzwaelder, Kerstin [6 ]
Koehl, Ulrike [7 ]
Kunkel, Hanna [2 ]
Kuehlcke, Klaus [8 ]
Schilz, Andrea [8 ]
Schmidt, Manfred [6 ]
von Kalle, Christof [6 ]
Trasher, Adrian [9 ]
Hoelzer, Dieter [1 ]
Seger, Reinhard [10 ]
Grez, Manuel [2 ]
机构
[1] Univ Frankfurt, Sch Med, Dept Hematol, Frankfurt, Germany
[2] Inst Biomed Res, Frankfurt, Germany
[3] Univ Heidelberg, Inst Human Genet, D-6900 Heidelberg, Germany
[4] German Canc Res Ctr, Div Mol Epidemiol, Heidelberg, Germany
[5] Ctr Mol Pathol, Hannover Med Sch, Hannover, Germany
[6] Univ Heidelberg, Natl Ctr Tumor Dis, D-6900 Heidelberg, Germany
[7] Univ Frankfurt, Sch Med, Dept Pediat Hematol, Frankfurt, Germany
[8] EUFETS AG, Idar Oberstein, Germany
[9] UCL, Inst Child Hlth, London WC1E 6BT, England
[10] Childrens Hosp Zurich, Div Hematol Immunol, Zurich, Switzerland
关键词
D O I
10.1182/blood.V110.11.503.503
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
503
引用
收藏
页码:155A / 155A
页数:1
相关论文
共 50 条
  • [1] Phase I/II gene therapy study for X-CGD: Results, lessons and perspectives
    Grez, Manuel
    Ott, Marion G.
    Stein, Stefan
    Schultze-Strasser, Stephan
    Preiss, Carolin
    Kramer, Rolf
    Schmidt, Manfred
    Kunkel, Hana
    von Kalle, Christof
    Thrasher, Adrian
    Hoelzer, Dieter
    Seger, Reinhard
    BLOOD CELLS MOLECULES AND DISEASES, 2008, 40 (02) : 268 - 269
  • [2] Gene Therapy of Chronic Granulomatous Disease: Lessons Learned and Future Perspectives
    Grez, Manuel
    HUMAN GENE THERAPY, 2014, 25 (11) : A24 - A24
  • [3] Retroviral Gene Therapy for X-Linked Chronic Granulomatous Disease: Results from Phase I/II Trial
    Kim, Joong Gon
    Ahn, Hyo Seop
    Kang, Hyoung Jin
    Kim, Sujeong
    Hong, Youngtae
    Joo, Chang-Wan
    Yoon, Nam-Kyung
    Kim, Sunyoung
    BLOOD, 2008, 112 (11) : 818 - 818
  • [4] Retroviral Gene Therapy for X-linked Chronic Granulomatous Disease: Results From Phase I/II Trial
    Kang, Hyoung Jin
    Bartholomae, Cynthia C.
    Paruzynski, Anna
    Arens, Anne
    Kim, Sujeong
    Yu, Seung Shin
    Hong, Youngtae
    Joo, Chang-Wan
    Yoon, Nam-Kyung
    Rhim, Jung-Woo
    Kim, Joong Gon
    Von Kalle, Christof
    Schmidt, Manfred
    Kim, Sunyoung
    Ahn, Hyo Seop
    MOLECULAR THERAPY, 2011, 19 (11) : 2092 - 2101
  • [5] RETROVIRAL GENE THERAPY FOR CHRONIC GRANULOMATOUS DISEASE: PRELIMINARY DATA FROM PHASE I/II TRIAL
    Kim, Joong Gon
    Ahn, Hyo Seop
    Kang, Hyoung Jin
    Kim, Sujeong
    Hong, Youngtae
    Joo, Chang-Wan
    Yoon, Nam-Kyung
    Kim, Sunyoung
    JOURNAL OF GENE MEDICINE, 2009, 11 (12): : 1144 - 1144
  • [6] Phase I/II gene therapy trial for the correction of chronic granulomatous disease: Results from 3 years follow-up
    Grez, M.
    Stein, S.
    Ott, M. G.
    Preiss, C.
    Schultze-Strasser, S.
    Schmidt, M.
    Kunkel, H.
    von Kalle, C.
    Hoelzer, D.
    Seger, R.
    EXPERIMENTAL HEMATOLOGY, 2007, 35 (09) : 79 - 79
  • [7] Development of a phase I clinical trial of gene therapy for chronic granulomatous disease
    Malech, HL
    Sekhsaria, S
    WhitingTheobald, N
    Linton, GF
    Vowells, SJ
    Li, F
    Miller, JA
    Holland, SM
    Leitman, SF
    Carter, CS
    Read, EJ
    Butz, R
    Wannebo, C
    Fleisher, TA
    Deans, RJ
    Spratt, SK
    Maack, CA
    Rokovich, JA
    Cohen, LK
    Maples, PB
    Gallin, JI
    BLOOD, 1995, 86 (10) : 1167 - 1167
  • [8] Gene therapy of chronic granulomatous disease: Results from pre-clinical and phase I clinical studies
    Ott, MG
    Koehl, U
    Sadat, MA
    Merget-Millitzer, H
    Stein, S
    Saulnier, S
    Hossle, JP
    Dinauer, MC
    Seger, R
    Hoelzer, D
    Grez, M
    MOLECULAR THERAPY, 2003, 7 (05) : S408 - S408
  • [9] Results from a Phase I/II Clinical Trial for X Linked Chronic Granulomatous Disease (CGD): Possible Impact of Inflammation on Gene Therapy Efficacy
    Magnani, Alessandra
    Sobrino, Steicy
    Cousin, Chloe
    Martignetti, Loredana
    Semeraro, Michaela
    Magrin, Elisa
    Soheili, Tayebeh
    Gabrion, Aurelie
    Roudaut, Cecile
    Lortholary, Olivier
    Barnabei, Laura
    Tuchmann-Durand, Caroline
    Lefrere, Francois
    Andre, Isabelle
    Rieux-Laucat, Frederic
    Thrasher, Adrian J.
    Suarez, Felipe
    Marcais, Ambroise
    Galy, Anne
    Rausell, Antonio
    Blanche, Stephane
    Six, Emmanuelle
    Cavazzana, Marina
    MOLECULAR THERAPY, 2020, 28 (04) : 58 - 58
  • [10] Gene Therapy for Chronic Granulomatous Disease: Current Status and Future Perspectives
    Kaufmann, Kerstin B.
    Chiriaco, Maria
    Siler, Ulrich
    Finocchi, Andrea
    Reichenbach, Janine
    Stein, Stefan
    Grez, Manuel
    CURRENT GENE THERAPY, 2014, 14 (06) : 447 - 460