Toxicology studies in support of the UK CF Gene Therapy Consortium's Multi-dose Clinical Trial

被引:0
|
作者
Griesenbach, Uta [1 ]
McLachlan, Gerry [2 ,3 ]
Collie, David D. [2 ,3 ]
Innes, J. Alastair [4 ]
Higgins, Tracy E. [1 ]
Cheng, Seng H. [5 ]
Scheule, Ronald K. [5 ]
Alton, Eric W. F. W. [1 ]
Davies, Jane C. [1 ]
Hyde, Steve C. [6 ]
Gill, Deborah R. [6 ]
Porteous, David J. [4 ]
Boyd, A. Christopher [4 ]
机构
[1] Univ London Imperial Coll Sci Technol & Med, Dept Gene Therapy, UK CF Gene Therapy Consortium, Natl Heart & Lung Inst, London SW3 6LR, England
[2] Univ Edinburgh, Roslin Inst, Easter Bush EH25 9RG, Midlothian, Scotland
[3] Univ Edinburgh, Royal Dick Sch Vet Med, Easter Bush EH25 9RG, Midlothian, Scotland
[4] Univ Edinburgh, Royal Hosp Sick Children, Dept Resp & Sleep Med, Edinburgh EH9 1LF, Midlothian, Scotland
[5] Genzyme Corp, Framingham, MA 01701 USA
[6] Univ Oxford, John Radcliffe Hosp, Nuffield Dept Clin Lab Sci, Gene Med Grp, Oxford OX3 9DU, England
关键词
D O I
暂无
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
引用
收藏
页码:A12 / A12
页数:1
相关论文
共 50 条
  • [31] HSV multi-suicide gene therapy for malignant glioblastoma (GBM): Efficacy, toxicology and bio-distribution studies
    Niranjan, A
    Wolfe, DP
    Tamura, M
    Soares, MK
    Krisky, DM
    Lunsford, LD
    Kondziolka, D
    DeLuca, NA
    Goins, WF
    Cohen, JB
    Glorioso, JC
    CANCER GENE THERAPY, 2004, 11 (12) : 844 - 844
  • [32] Preclinical Studies of HG004 Gene Replacement Therapy in Support of Clinical Trial for RPE65-Associated Inherited Retinal Dystrophy
    Wu, Weiwei
    He, Sheng
    Yang, Qi
    Yang, Chenhua
    Zhu, Mengke
    Wang, Shaoran
    Yao, Xuan
    Yang, Hui
    Luk, Alvin
    Shi, Linyu
    MOLECULAR THERAPY, 2024, 32 (04) : 758 - 759
  • [33] Adeno Associated Virus (AAV) Mediated Follistatin Gene Transfer Toxicology Studies in Preparation of Phase I/II Clinical Trial
    Kota, Janaiah
    Shilling, Christopher
    Montgomery, Chrystal
    Lewis, Sarah
    Bevan, Adam
    Shontz, Kim
    Kaminoh, Yuuki
    Heller, Kristin
    Rosales, Xiomara
    Viollet, Laurence
    Flanigan, Kevin
    Clark, K. Reed
    Kaspar, Brian
    Sahenk, Zarife
    Mendell, Jerry
    MOLECULAR THERAPY, 2012, 20 : S72 - S72
  • [34] Rare disease clinical research network's urea cycle consortium delivers a successful clinical trial to improve alternate pathway therapy
    Mistry, Pramod K.
    HEPATOLOGY, 2013, 57 (06) : 2100 - 2102
  • [35] Preclinical efficacy and safety of lentiviral (LV)-based gene therapy in IMO support clinical trial initiation
    Moscatelli, Ilana
    Loefvall, Henrik
    Kohn, Donald
    Ricks, David
    Rothe, Michael
    Almarza, Elena
    Herzog, Chris
    Gutierrez, Sonia
    Law, Ken
    Nicoletti, Eileen
    Schambach, Axel
    Schwartz, Jonathan
    Richter, Johan
    JOURNAL OF BONE AND MINERAL RESEARCH, 2022, 37 : 196 - 196
  • [36] Preclinical efficacy and safety of lentiviral (LV)-based gene therapy in IMO support clinical trial initiation
    Moscatelli, I.
    Kohn, D. B.
    Ricks, D.
    Rothe, M.
    Almarza, E.
    Herzog, C.
    Gutierrez, S.
    Law, K. M.
    Nicoletti, E.
    Schambach, A.
    Schwartz, J.
    Richter, J.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A81 - A82
  • [37] Early results from a clinical trial of AADC gene therapy for Parkinson's disease
    Eberling, JL
    Bankiewicz, KS
    McGuire, D
    Aminoff, M
    Christine, C
    Starr, P
    Larson, P
    Jagust, WJ
    NEUROLOGY, 2006, 66 (05) : 276 - 277
  • [38] Gene Therapy in Parkinson Disease: From Preclinical Studies in Primates to a Phase I Clinical Trial
    Jarraya, B.
    Lepetit, H.
    Ralph, S.
    Miskin, J.
    Gurruchaga, J-M
    Fenelon, G.
    Boulet, S.
    Jan, C.
    Bonvento, G.
    Azzouz, M.
    Brugiere, P.
    Kingsman, S.
    Naylor, S.
    Hantraye, P.
    Remy, P.
    Mazarakis, N. D.
    Mitrophanous, K.
    Palfi, S.
    HUMAN GENE THERAPY, 2010, 21 (06) : 764 - 765
  • [39] Efficacy and Safety of ZS-9 in Patients with Hyperkalemia: Results from the HyperkAlemia RandoMized interventiON multI-dose ZS-9 maintEnance (HARMONIZE) Clinical Trial
    Kosiborod, Mikhail
    Rasmussen, Henrik S.
    Lavin, Philip
    Qunibi, Wajeh Y.
    Spinowitz, Bruce
    Packham, David
    Roger, Simon
    Yang, Alex
    Lerma, Edgar
    Singh, Bhupinder
    CIRCULATION, 2014, 130 (23) : 2115 - 2116
  • [40] Targeting Protein Disulfide Isomerase with the Oral Flavonoid Isoquercetin Prevents Venous Thromboembolism in Advanced Cancer: Results of a Multi-Dose, Multi-Center, Phase II Clinical Trial (CATIQ Study)
    Zwicker, Jeffrey I.
    Schlechter, Benjamin L.
    Stopa, Jack Davis
    Liebman, Howard A.
    Aggarwal, Anita
    Caughey, Thomas
    Bauer, Ken A.
    Kuemmerle, Nancy
    Wong, Ellice Y.
    Wun, Ted
    Mclaughlin, Marilyn
    Puligandla, Maneka
    Neuberg, Donna S.
    Furie, Bruce
    Flaumenhaft, Robert C.
    BLOOD, 2018, 132