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Molecular Therapy cover

Molecular Therapy

IF12
Papers9970
Citations29788
Journal Papers 25433
Publication Date
Planning advanced therapy trials for Huntington’s Disease: ethical considerations
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deleteTowns Cindy; Ballantyne Angela; Konstatinova Pavlina; Drew Cheney; Rosser Anne
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Retraction Notice to: Gas5 Exerts Tumor-suppressive Functions in Human Glioma Cells by Targeting miR-222
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deleteXihe Zhao; Ping Wang; Jing Liu; Jian Zheng; Yunhui Liu; Jiajia Chen; Yixue Xue
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Targeting Telomerase with an HLA Class II-Restricted TCR for Cancer Immunotherapy
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deletePierre Dillard; Hakan Köksal; Solrun Melkorka Maggadottir; Anna Winge-Main; Sylvie Pollmann; Mathilde Menard; Marit Renée Myhre; Gunhild M. Mælandsmo; Vivi Ann Flørenes; Gustav Gaudernack; Gunnar Kvalheim; Sébastien Wälchli; Else Marit Inderberg
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Novel HIV fusion-inhibitory lipopeptides exhibit dramatically improved activity against resistant mutants
delete2026-09-26
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deleteYuanmei Zhu; Ting Chen; Nian Liu; Xiuzhu Geng; Na Li; Jiahan Lu; Fei Wang; Jiasen Yang; Zhe Cong; Bo Qin; Huihui Chong; Sheng Cui; Jing Xue; Yuxian He
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BTCE-secreting CAR T cells fight solid tumors
delete2026-09-25
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deleteClaudia Arndt; Alexandra von Jutrzenka-Trzebiatowski
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A new capless self-amplifying RNA
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deletePatrick B. Arbuthnot; Kristie M. Bloom; Abdullah Ely
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Repurposing triamterene to target CLIC1 in glioblastoma stem cells: A new opportunity for an old drug?
delete2026-09-25
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deleteShubha Gururaja Rao; Aviana R. Smith; Harpreet Singh
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Broad targeting of the cell adhesion machinery - cancer cell detachment triggered by the alpha1-oleate complex
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deleteArunima Chaudhuri; Farhan Haq; Ines Ambite; Atefeh Nazari; Samudra Sabari; Michele Tavecchio; Antonio Pedro NBM Carneiro; Olof Stenström; Shahram Ahmadi; Hien Tran; Antonin Brisuda; Jaromir Hacek; Marek Babjuk; Catharina Svanborg
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Base editing-mediated induction of stop codons in HPV18 E6/E7 for cervical cancer therapy
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deleteYue Kang; Yifan Wu; Bingting Bao; Xueqian Wang; Zhiqiang Yu; Chen Cao; Zhi Wang; Zhaojie Wang; Gang Chen; Ding Ma; Tao Yi; Zheng Hu; Xun Tian
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Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy
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deleteJane L. Kinsella; Simon A. Jones; Karen F. Buckland; Rebecca J. Holley; Helena Lee; Wendy Ogden; Jane Potter; Heather J. Church; Kathryn L. Brammeier; Karen L. Tylee; Stuart Ellison; Rachel Searle; Claire Booth; Farzin Farzeneh; Louise Sherwin; Adrian J. Thrasher; Stewart Rust; Brian W. Bigger; Robert F. Wynn
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LNP-formulated dbDNA™ vaccines elicit durable humoral and cellular immunity with distinct kinetics to mRNA in non-human primates
delete2026-09-22
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deleteHelen Horton; Robert F. Cunliffe; Sungwon Kim; Madina Wane; Lisa J. Caproni; John S. Tregoning; Alexander M. de Bruin
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Pre-clinical validation of AAV-mediated gene therapy for KCNV2 retinopathy improves visual function and expression in mouse and patient models
delete2026-09-22
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deleteRabab Rashwan; Paula I. Fuller-Carter; Xin Ru Lim; Alicia A. Brunet; Annie L. Miller; Yashvi Bhatt; Rebekah James; Denise Anderson; Valentina Voigt; Joao A. Paulo; Mehdi Mirzaei; Melissa M. Mangala; Emilie O. Wong; Robyn V. Jamieson; Anai Gonzalez-Cordero; David M. Hunt; Livia S. Carvalho
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Catestatin peptide ameliorates tauopathy and amyloidogenesis via adrenergic inhibition
delete2026-09-22
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deleteSuborno Jati; Satadeepa Kal; Daniel Munoz-Mayorga; Kechun Tang; Debashis Sahoo; Xu Chen; Sushil K. Mahata
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Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction
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deleteMark DeWitt; Beatriz Campo-Fernandez; Sohini Roy; Alissa L.W. Kleinhenz; Alejandra Davila; Mallory Lennon; Jonnathan T. Vu; Netravathi Krishnappa; Suzanne Said; Chin-hong Tseng; So Hyun Park; Gang Bao; Wendy Magis; David I.K. Martin; Dario Boffelli; Stacia K. Wyman; Donald B. Kohn; Mark C. Walters; Zulema Romero
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Targeting white matter astrocytes with a Gfap-disruptive Cas9 nuclease benefits a murine model of Alexander disease
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deleteAnne E.J. Hillen; Irene Capuano; Barbara Gega; Rossana Norata; Margaux Mombled; Ilaria Laface; Giada Zambonini; Andrea Lomagno; Marine Rouillon; Serena G. Giannelli; Fabrizio Benedicenti; Francesco Gazzo; Sergio Arévalo; Giulio Spinozzi; Francesca Sanvito; Mario Amendola; Vania Broccoli; Eugenio Montini; Angela Gritti; Vasco Meneghini
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D-Amino Acid Prodrug DLMEH Activates mTORC1 via Sestrin2 to Restore Muscle Protein Synthesis in Sarcopenia
delete2026-09-11
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deleteJae Ho Shim; Ji Yeon Lee; Byung Kook Ahn; Sang Woo Woo; Jihye Han; Hyeon Soo Kim
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Combination Of Bispecific Antibodies Enable Targeted TNFRSF Agonism and Effective Antitumor Activity
delete2026-09-10
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deleteLucie Diby; Pauline Malinge; Valery Moine; Lise Nouveau; Laurence Chatel; Krzysztof Masternak; Limin Shang; Walter Ferlin; Nicolas Fischer; Mark S. Cragg; Mikael Pittet; Vanessa Buatois; Eric Hatterer
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Multiplexed genome editing by CRISPR-Un1Cas12f1 restores dystrophin expression in a mouse model of Duchenne muscular dystrophy
delete2026-09-10
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deleteJin-Seok Gil; Eunyoung Lee; Hyunsun Na; Gyubin Oh; Eunyoung Choi; Yejin Park; Hyeonjeong Park; Taeyoung Koo
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