Article

Bispecific BAFF-R/BCMA CAR T cells control growth of heterogeneous plasma cells in multiple myeloma.
Fiori, Agnese ORCID: https://orcid.org/0000-0003-0440-4857, Zimmermann, Karin, Li, Anna ORCID: https://orcid.org/0000-0002-4702-7783, Westermann, Jörg, Anagnostopoulos, Ioannis, Menzel, Lutz ORCID: https://orcid.org/0000-0002-3816-6255, Bunse, Mario ORCID: https://orcid.org/0000-0002-7554-2520, Erdlei, Henry, Jayarajan, Jeyan ORCID: https://orcid.org/0009-0009-6205-143X, Grünschläger, Florian, Ortiz-Aguirre, Juan Pablo ORCID: https://orcid.org/0000-0002-6557-0069, Haas, Simon ORCID: https://orcid.org/0000-0001-9227-2051, Teßmann, Uwe-Jens, Freitag, Jens, Rosenwald, Andreas, Kwak, Larry, Wang, Xiuli, Dong, Zhenyuan, Cha, Soungchul, Reiser, John, Peralta, Eigen, Valamehr, Bahram, Krönke, Jan, Höpken, Uta E. ORCID: https://orcid.org/0000-0002-0776-4893 and Rehm, Armin ORCID: https://orcid.org/0000-0001-7490-1950
Molecular Therapy 34 (3): 1399-1420. 4 March 2026

In vivo base editing reduces liver cysts in autosomal dominant polycystic kidney disease.
Ibel, Antonia ORCID: https://orcid.org/0000-0002-3880-6558, Bhardwaj, Rishi, Yilmaz, Duygu Elif, Kong, Shuhan ORCID: https://orcid.org/0009-0008-0164-3346, Wendlinger, Sarah ORCID: https://orcid.org/0000-0001-6771-4794, Cordero, Carlos ORCID: https://orcid.org/0000-0001-5357-9413, Papaioannou, Dimitra ORCID: https://orcid.org/0009-0000-6194-8434, Papazian, Maria, Schönauer, Ria, Meng, Qiyao, Eckardt, Kai-Uwe, Hassan, Fatima, Volpe, Isabel, Klämbt, Verena, Halbritter, Jan, Fedeles, Sorin, Krappitz, Matteus and Kaminski, Michael M. ORCID: https://orcid.org/0000-0003-0429-7027
Molecular Therapy 33 (11): 5373-5382. 5 November 2025

EBAG9-silencing exerts an immune checkpoint function without aggravating adverse effects.
Wirges, A. ORCID: https://orcid.org/0000-0001-7038-4503, Bunse, M. ORCID: https://orcid.org/0000-0002-7554-2520, Joedicke, J.J. ORCID: https://orcid.org/0000-0002-4708-3216, Blanc, E. ORCID: https://orcid.org/0000-0002-4369-0254, Gudipati, V., Moles, M.W., Shiku, H., Beule, D. ORCID: https://orcid.org/0000-0002-3284-0632, Huppa, J.B., Höpken, U.E. ORCID: https://orcid.org/0000-0002-0776-4893 and Rehm, A. ORCID: https://orcid.org/0000-0001-7490-1950
Molecular Therapy 30 (11): 3358-3378. 2 November 2022

Key benefits of dexamethasone and antibody treatment in COVID-19 hamster models revealed by single cell transcriptomics.
Wyler, E. ORCID: https://orcid.org/0000-0002-9884-1806, Adler, J.M., Eschke, K., Teixeira Alves, G. ORCID: https://orcid.org/0000-0002-5822-4168, Peidli, S. ORCID: https://orcid.org/0000-0002-4257-8690, Pott, F., Kazmierski, J., Michalick, L., Kershaw, O., Bushe, J., Andreotti, S., Pennitz, P., Abdelgawad, A., Postmus, D., Goffinet, C., Kreye, J., Reincke, S.M. ORCID: https://orcid.org/0000-0002-8132-3527, Prüss, H., Blüthgen, N. ORCID: https://orcid.org/0000-0002-0171-7447, Gruber, A.D., Kuebler, W.M., Witzenrath, M., Landthaler, M. ORCID: https://orcid.org/0000-0002-1075-8734, Nouailles, G. and Trimpert, J. ORCID: https://orcid.org/0000-0003-1616-0810
Molecular Therapy 30 (5): 1952-1965. 4 May 2022

CRISPR/Cas9-mediated ELANE mutation correction in hematopoietic stem and progenitor cells to treat severe congenital neutropenia.
Tran, N.T. ORCID: https://orcid.org/0000-0003-0896-8641, Graf, R. ORCID: https://orcid.org/0000-0002-5400-9938, Wulf-Goldenberg, A., Stecklum, M., Strauß, G., Kühn, R. ORCID: https://orcid.org/0000-0003-1694-9803, Kocks, C. ORCID: https://orcid.org/0000-0003-1749-3334, Rajewsky, K. ORCID: https://orcid.org/0000-0002-6633-6370 and Chu, V.T. ORCID: https://orcid.org/0000-0003-1327-8696
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. ORCID: https://orcid.org/0000-0002-3214-1540, Buracchi, C., Arcangeli, S., Galimberti, S., Valsecchi, M.G., Perriello, V.M., Rasko, T., Alberti, G., Magnani, C.F., Cappuzzello, C., Lundberg, F., Pande, A. ORCID: https://orcid.org/0009-0008-0826-973X, Dastoli, G., Introna, M., Serafini, M., Biagi, E., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384, 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., Kieback, E. ORCID: https://orcid.org/0000-0003-3831-2846, Marino, S.F. ORCID: https://orcid.org/0000-0001-5696-9679, Oden, F., Westermann, J., Chmielewski, M., Abken, H., Uckert, W., Höpken, U.E. ORCID: https://orcid.org/0000-0002-0776-4893 and Rehm, A. ORCID: https://orcid.org/0000-0001-7490-1950
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., Mesa-Nuñez, C., Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Almarza, E., Poletti, V., Schmeer, M., Grueso, E., Ordóñez Flores, J.C., Kobelt, D. ORCID: https://orcid.org/0000-0002-9373-0648, Walther, W. ORCID: https://orcid.org/0000-0003-2884-0231, Aneja, M.K., Geiger, J,, Bonig, H.B., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Schleef, M., Rudolph, C., Mavilio, F., Bueren, J.A., Guenechea, G. and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
Molecular Therapy 26 (4): 1137-1153. 4 April 2018

Structural determinants of sleeping beauty transposase activity.
Abrusán, G., Yant, S.R., Szilágyi, A., Marsh, J.A., Mátés, L., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Barabás, O. and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
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. ORCID: https://orcid.org/0009-0001-7697-0551, Ammar, I., Gupta, S., Bunse, M. ORCID: https://orcid.org/0000-0002-7554-2520, Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Chen, W. ORCID: https://orcid.org/0000-0003-3263-1627, Uckert, W., Schulz, T.F., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384 and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
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., Kurkipuro, J., Heikura, T., Vuorio, T., Hytönen, E., Izsvák, Z. ORCID: https://orcid.org/0000-0002-2053-2384 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. ORCID: https://orcid.org/0000-0002-7554-2520, Bendle, G.M., Linnemann, C., Bies, L., Schulz, S., 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., Gogol-Doering, A. ORCID: https://orcid.org/0009-0001-7697-0551, Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Chen, W. ORCID: https://orcid.org/0000-0003-3263-1627, Cathomen, T., Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384 and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
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., Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Staunstrup, N.H., Gogol-Doering, A. ORCID: https://orcid.org/0009-0001-7697-0551, Bak, R.O., Sharma, N., Mates, L. ORCID: https://orcid.org/0000-0002-0058-470X, Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Chen, W. ORCID: https://orcid.org/0000-0003-3263-1627, Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658 and Mikkelsen, J.G.
Molecular Therapy 19 (8): 1499-1510. August 2011

Comparative analysis of transposable element vector systems in human cells.
Grabundzija, I., Irgang, M., Mates, L. ORCID: https://orcid.org/0000-0002-0058-470X, Belay, E., Matrai, J., Gogol-Doering, A. ORCID: https://orcid.org/0009-0001-7697-0551, Kawakami, K., Chen, W. ORCID: https://orcid.org/0000-0003-3263-1627, Ruiz, P., Chuah, M.K., Vandendriessche, T., Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384 and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
Molecular Therapy 18 (6): 1200-1209. June 2010

Hybrid lentivirus-transposon vectors with a random integration profile in human cells.
Staunstrup, N.H., Moldt, B., Mates, L. ORCID: https://orcid.org/0000-0002-0058-470X, Villesen, P., Jakobsen, M., Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658, Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384 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., Schorn, A., Rolfs, F., Devaraj, A. ORCID: https://orcid.org/0000-0002-8685-6728, Miskey, C. ORCID: https://orcid.org/0000-0002-7566-9556, Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384 and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
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. ORCID: https://orcid.org/0000-0001-7006-282X, Walther, W. ORCID: https://orcid.org/0000-0003-2884-0231, Stege, A., Kaszubiak, A., Fichtner, I. and Lage, H.
Molecular Therapy 16 (1): 178-186. January 2008

Targeted Sleeping Beauty transposition in human cells.
Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658, Katzer, A., Stuewe, E.E., Fiedler, D., Knespel, S. and Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384
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. ORCID: https://orcid.org/0000-0003-2884-0231, Stein, U. ORCID: https://orcid.org/0000-0001-7006-282X, Fichtner, I., Kobelt, D. ORCID: https://orcid.org/0000-0002-9373-0648, Aumann, J., 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. ORCID: https://orcid.org/0000-0001-8838-6638, Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384, Walisko, O. and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
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., Pelisek, J., Krausz, E., Fuchs, A., Groth, D., Curth, R., Keil, O., Quilici, J., Rolland, P.H., 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

Review

Targeting RNA adenosine editing and modification enzymes for RNA therapeutics.
Vlachogiannis, Nikolas I. ORCID: https://orcid.org/0000-0001-7878-8078, Polycarpou-Schwarz, Maria, Avdi, Aikaterini-Paraskevi, Tual-Chalot, Simon and Stellos, Konstantinos ORCID: https://orcid.org/0000-0002-0194-0825
Molecular Therapy 33 (9): 4044-4090. 3 September 2025

Sleeping Beauty transposition: biology and applications for molecular therapy.
Izsvak, Z. ORCID: https://orcid.org/0000-0002-2053-2384 and Ivics, Z. ORCID: https://orcid.org/0000-0002-7803-6658
Molecular Therapy 9 (2): 147-156. 1 February 2004

Editorial

Pluripotent stem cell-based therapy toward the clinic for muscular dystrophy.
Escobar, Helena ORCID: https://orcid.org/0000-0002-8128-7845
Molecular Therapy 33 (10): 4670-4672. 1 October 2025

Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells.
Novikova, Nataliia and Huppa, Johannes B. ORCID: https://orcid.org/0000-0003-2634-8198
Molecular Therapy 33 (9): 4029-4031. 3 September 2025

Comment

Cytosine base editing achieves robust lipoprotein(a) reduction while preserving genomic integrity.
Klämbt, Verena and Kaminski, Michael M. ORCID: https://orcid.org/0000-0003-0429-7027
Molecular Therapy 34 (6): 3152-3154. 3 June 2026