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CRISPR/Cas9-mediated ELANE mutation correction in hematopoietic stem and progenitor cells to treat Severe Congenital Neutropenia.
Tran, N.T. and Graf, R. and Wulf-Goldenberg, A. and Stecklum, M. and Strauß, G. and Kühn, R. and Kocks, C. and Rajewsky, K. and Chu, V.T.
Molecular Therapy 28 (12): 2621-2634. 2 December 2020

Targeting CD33 in chemoresistant AML patient-derived xenografts by CAR-CIK cells modified with an improved SB transposon system.
Rotiroti, M.C. and Buracchi, C. and Arcangeli, S. and Galimberti, S. and Valsecchi, M.G. and Perriello, V.M. and Rasko, T. and Alberti, G. and Magnani, C.F. and Cappuzzello, C. and Lundberg, F. and Pande, A. and Dastoli, G. and Introna, M. and Serafini, M. and Biagi, E. and Izsvák, Z. and Biondi, A. and Tettamanti, S.
Molecular Therapy 28 (9): 1974-1986. 2 September 2020

CAR T cells with enhanced sensitivity to B cell maturation antigen for the targeting of B cell non-Hodgkin's lymphoma and multiple myeloma.
Bluhm, J. and Kieback, E. and Marino, S.F. and Oden, F. and Westermann, J. and Chmielewski, M. and Abken, H. and Uckert, W. and Höpken, U.E. and Rehm, A.
Molecular Therapy 26 (8): 1906-1920. 1 August 2018

Efficient non-viral gene delivery into human hematopoietic stem cells by minicircle Sleeping Beauty transposon vectors.
Holstein, M. and Mesa-Nuñez, C. and Miskey, C. and Almarza, E. and Poletti, V. and Schmeer, M. and Grueso, E. and Ordóñez Flores, J.C. and Kobelt, D. and Walther, W. and Aneja, M.K. and Geiger, J, and Bonig, H.B. and Izsvák, Z. and Schleef, M. and Rudolph, C. and Mavilio, F. and Bueren, J.A. and Guenechea, G. and Ivics, Z.
Molecular Therapy 26 (4): 1137-1153. 4 April 2018

Structural determinants of sleeping beauty transposase activity.
Abrusán, G. and Yant, S.R. and Szilágyi, A. and Marsh, J.A. and Mátés, L. and Izsvák, Z. and Barabás, O. and Ivics, Z.
Molecular Therapy 24 (8): 1369-1377. August 2016

Genome-wide profiling reveals remarkable parallels between insertion site selection properties of the MLV retrovirus and the piggyBac transposon in primary human CD4(+) T cells.
Gogol-Döring, A. and Ammar, I. and Gupta, S. and Bunse, M. and Miskey, C. and Chen, W. and Uckert, W. and Schulz, T.F. and Izsvák, Z. and Ivics, Z.
Molecular Therapy 24 (3): 592-606. March 2016

Sleeping Beauty transposon vectors in liver directed gene delivery of LDLR and VLDLR for gene therapy of familial hypercholesterolemia.
Turunen, T.A.K. and Kurkipuro, J. and Heikura, T. and Vuorio, T. and Hytönen, E. and Izsvák, Z. and Ylä-Herttuala, S.
Molecular Therapy 24 (3): 620-635. March 2016

RNAi-mediated TCR knockdown prevents autoimmunity in mice caused by mixed TCR dimers following TCR gene transfer.
Bunse, M. and Bendle, G.M. and Linnemann, C. and Bies, L. and Schulz, S. and Schumacher, T.N. and Uckert, W.
Molecular Therapy 22 (11): 1983-1991. November 2014

Retargeting Sleeping Beauty transposon insertions by engineered zinc finger DNA-binding domains.
Voigt, K. and Gogol-Doering, A. and Miskey, C. and Chen, W. and Cathomen, T. and Izsvak, Z. and Ivics, Z.
Molecular Therapy 20 (10): 1852-1862. October 2012

Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human cells.
Moldt, B. and Miskey, C. and Staunstrup, N.H. and Gogol-Doering, A. and Bak, R.O. and Sharma, N. and Mates, L. and Izsvak, Z. and Chen, W. and Ivics, Z. and Mikkelsen, J.G.
Molecular Therapy 19 (8): 1499-1510. August 2011

Comparative analysis of transposable element vector systems in human cells.
Grabundzija, I. and Irgang, M. and Mates, L. and Belay, E. and Matrai, J. and Gogol-Doering, A. and Kawakami, K. and Chen, W. and Ruiz, P. and Chuah, M.K. and Vandendriessche, T. and Izsvak, Z. and Ivics, Z.
Molecular Therapy 18 (6): 1200-1209. June 2010

Hybrid lentivirus-transposon vectors with a random integration profile in human cells.
Staunstrup, N.H. and Moldt, B. and Mates, L. and Villesen, P. and Jakobsen, M. and Ivics, Z. and Izsvak, Z. and Mikkelsen, J.G.
Molecular Therapy 17 (7): 1205-1214. July 2009

Transcriptional activities of the Sleeping Beauty transposon and shielding its genetic cargo with insulators.
Walisko, O. and Schorn, A. and Rolfs, F. and Devaraj, A. and Miskey, C. and Izsvak, Z. and Ivics, Z.
Molecular Therapy 16 (2): 359-369. February 2008

Complete in vivo reversal of the multidrug resistance phenotype by jet-injection of anti-MDR1 short hairpin RNA-encoding plasmid DNA.
Stein, U. and Walther, W. and Stege, A. and Kaszubiak, A. and Fichtner, I. and Lage, H.
Molecular Therapy 16 (1): 178-186. January 2008

Targeted Sleeping Beauty transposition in human cells.
Ivics, Z. and Katzer, A. and Stuewe, E.E. and Fiedler, D. and Knespel, S. and Izsvak, Z.
Molecular Therapy 15 (6): 1137-1144. June 2007

Nonviral jet-injection gene transfer for efficient in vivo cytosine deaminase suicide gene therapy of colon carcinoma.
Walther, W. and Stein, U. and Fichtner, I. and Kobelt, D. and Aumann, J. and Arlt, F. and Schlag, P.M.
Molecular Therapy 12 (6): 1176-1184. 29 September 2005

Development of hyperactive Sleeping Beauty transposon vectors by mutational analysis.
Zayed, H. and Izsvak, Z. and Walisko, O. and Ivics, Z.
Molecular Therapy 9 (2): 292-304. 1 February 2004

Optimization of nonviral gene transfer of vascular smooth muscle cells in vitro and in vivo.
Armeanu, S. and Pelisek, J. and Krausz, E. and Fuchs, A. and Groth, D. and Curth, R. and Keil, O. and Quilici, J. and Rolland, P.H. and Reszka, R. and Nikol, S.
Molecular Therapy 1 (4): 366-375. 1 April 2000

Therapy of head and neck squamous cell carcinoma with an oncolytic adenovirus expressing HSV-tk.
Morris, J.C. and Wildner, O.
Molecular Therapy 1 : 56-62. 1 January 2000


Sleeping Beauty transposition: biology and applications for molecular therapy.
Izsvak, Z. and Ivics, Z.
Molecular Therapy 9 (2): 147-156. 1 February 2004

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