Improving AAV Gene Therapy Safety Analysis: Multiplex LAM-PCR Provide New Insights Into AAV Vector Integration

被引:0
|
作者
Gil-Farina, Irene [1 ]
Kaeppel, Christine [1 ]
Wilkening, Stefan [1 ]
Lopez-Franco, Esperanza [2 ]
Paneda, Maria Astrid [3 ]
Prieto, Jesus [4 ]
Spronck, Lisa [5 ]
von Kalle, Christof [1 ]
Petry, Harald [5 ]
Gonzalez-Aseguinolaza, Gloria [2 ]
Schmidt, Manfred [1 ]
机构
[1] NCT DKFZ, Translat Oncol, Heidelberg, Germany
[2] CIMA, Gene Therapy & Hepatol, Pamplona, Spain
[3] DIGNA Biotech SL, Madrid, Spain
[4] Univ Hosp Navarra, Pamplona, Spain
[5] uniQure, Amsterdam, Netherlands
关键词
D O I
10.1016/S1525-0016(16)34273-3
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
664
引用
收藏
页码:S264 / S264
页数:1
相关论文
共 41 条
  • [1] New insights into AAV vectors integration profile: internal vector regions
    Gil-Farina, I.
    Kaeppel, C.
    Wilkening, S.
    Rodriguez, M. A. Paneda
    Lopez-Franco, E.
    Fontanellas, A.
    Prieto, J.
    Spronck, L.
    von Kalle, C.
    Petry, H.
    Gonzalez-Aseguinolaza, G.
    Schmidt, M.
    HUMAN GENE THERAPY, 2015, 26 (10) : A3 - A3
  • [2] Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B
    Schmidt, Manfred
    Foster, Graham R.
    Coppens, Michiel
    Thomsen, Hauke
    Dolmetsch, Ricardo
    Heijink, Liesbeth
    Monahan, Paul E.
    Pipe, Steven W.
    BLOOD ADVANCES, 2023, 7 (17) : 4966 - 4969
  • [3] ANALYSIS OF RETROVIRAL INTEGRATION SITES USING LAM-PCR TO SUPPORT THE INITIATION OF A PHASE I CANCER GENE THERAPY TRIAL
    Hyman, Jessica
    Kramer, Belinda A.
    Ginn, Samantha L.
    Alexander, Ian E.
    JOURNAL OF GENE MEDICINE, 2009, 11 (09): : 850 - 850
  • [4] Preclinical Safety Studies for AAV2-MerTK Gene Therapy Vector for Retinitis Pigmentosa
    Conlon, Thomas J.
    Deng, Wen-Tao
    Ryals, Renee C.
    Erger, Kirsten E.
    Cossette, Travis L.
    Boye, Shannon E.
    McDoom, Isaam
    Clemente, Nathalie
    Cleaver, Brian
    Potter, Mark
    Abernathy, Corinne
    Boye, Sanford L.
    Hauswirth, William W.
    MOLECULAR THERAPY, 2013, 21 : S23 - S23
  • [5] A novel AAV design improving efficiency and safety in a gene therapy approach for Rett syndrome in a mouse model
    Giannelli, S. G.
    Luoni, M.
    Morabito, G.
    Bido, S.
    Indrigo, M.
    Niro, A.
    Ricci, R.
    Valtorta, M.
    Ordazzo, G.
    Rossi, G.
    Broccoli, V.
    HUMAN GENE THERAPY, 2018, 29 (12) : A79 - A80
  • [6] Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia. Dog after AAV Gene Therapy
    Van Gorder, Lucas
    Doshi, Bhavya S.
    Willis, Elinor
    Lanza, Matthew
    Arruda, Valder R.
    Callan, Mary Beth
    Samelson-Jones, Ben J.
    BLOOD, 2022, 140 : 4919 - 4920
  • [7] The effect of exercise on improving strength and function in a Pompe mouse model treated with AAV vector mediated gene therapy
    Case, Laura
    Prater, Sean
    Rodriguiz, Ramona
    Pastva, Amy
    Taylor, Andrea
    Kishnani, Priya
    Koeberl, Dwight
    MOLECULAR GENETICS AND METABOLISM, 2011, 102 (02) : S10 - S10
  • [8] Safety and Toxicology of Ocular Gene Therapy with Recombinant AAV Vector rAAV.hCNGA3 in Nonhuman Primates
    Tobias, Peters
    Philipp, Seitz Immanuel
    Stylianos, Michalakis
    Martin, Biel
    Barbara, Wilhelm
    Felix, Reichel
    Alexander, Ochakovski Guy
    Eberhart, Zrenner
    Marius, Ueffing
    Birgit, Korbmacher
    Sven, Korte
    Ulrich, Bartz-Schmidt Karl
    Dominik, Fischer Manuel
    Bartz-Schmidt, Karl Ulrich
    Bolz, Sylvia
    Fischer, Dominik
    Kohl, Susanne
    Kuehlewein, Laura
    Muehlfriedel, Regine
    Neubauer, Jonas
    Ochakovski, Alex
    Paquet-Durand, Francois
    Seeliger, Mathias
    Sothilingam, Vithiyanjali
    Ueffing, Marius
    Weisschuh, Nicole
    Wissinger, Bernd
    Zhour, Ahmad
    Zobor, Ditta
    Zrenner, Eberhart
    Biel, Martin
    Michalakis, Stylianos
    Schoen, Christian
    Kahle, Nadine
    Peters, Tobias
    Wilhelm, Barbara
    Tsang, Steven
    Gloeckner, Christian Johannes
    HUMAN GENE THERAPY CLINICAL DEVELOPMENT, 2019, 30 (02) : 50 - 56
  • [9] Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog
    Van Gorder, Lucas
    Doshi, Bhavya S.
    Willis, Elinor
    Nichols, Timothy C.
    Cook, Emma
    Everett, John K.
    Merricks, Elizabeth P.
    Arruda, Valder R.
    Bushman, Frederic D.
    Callan, Mary Beth
    Samelson-Jones, Benjamin J.
    MOLECULAR THERAPY METHODS & CLINICAL DEVELOPMENT, 2023, 31
  • [10] ENHANCING EV-AAV INCORPORATION: INSIGHTS INTO NATURAL LOADING AND LOADING STRATEGIES FOR IMPROVED GENE THERAPY VECTOR DELIVERY
    Vorobieva, I.
    Friedrichson, H.
    Ahlskog, N.
    Staton, C.
    Wood, M.
    Conceicao, M.
    CYTOTHERAPY, 2024, 26 (06) : S88 - S89