Molecular Therapy Family of Journals (@MolTherapy) header
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The leading family of journals in cell and gene therapy. Journals include Molecular Therapy, Advances, Nucleic Acids, and Oncology.

Joined February 2014
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Aptamers in Neurodegenerative Disease: Systematic Review and Computational Analysis of Aptamer–Protein Interactions dlvr.​it/TW6rk8 #MTNA
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AAV-mediated Modulation of Dorsal Raphe Serotonergic Neurons Recapitulates Fenfluramine Efficacy in a Rat Model of Dravet Syndrome dlvr.​it/TW66Zq #genetherapy #celltherapy
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329
Absence of Hepatobiliary Toxicity in Large Animal Species Dosed with AAV8 Gene Therapy for X-linked Myotubular Myopathy dlvr.​it/TW66SV #genetherapy #celltherapy
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The RNA-binding protein RPS3 accelerates cell cycling via the Wnt/β-catenin signaling pathway in pancreatic cancer dlvr.​it/TW66JW
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Deciphering Mechanisms of Feeder Cell-Induced NK Cell Activation to Inform Development of Feeder-Free Expansion Systems dlvr.​it/TW5dc3 #MTMCD
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Evaluation of HexM and its high-level mannose 6-phosphorylated form, PhosHexM, as treatments of models of GM2 gangliosidoses dlvr.​it/TW5dX5 #MTMCD
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Reproductive Safety and Germline Transmission Risk of Onasemnogene Abeparvovec Following IV or ICV Administration in Mice dlvr.​it/TW5dRL #MTMCD
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Sharpening therapeutic tools toward blunting pathological nuclear lamina with advanced in vitro disease modeling dlvr.​it/TW4TFx #genetherapy #celltherapy
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Allele-selective and pan-allelic huntingtin lowering differentially affect neuronal homeostasis and BDNF transport dlvr.​it/TW4Ptv #MTNA
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Multi-level systems analysis identifies XPO1 as a druggable target linked to NFκB activation, tumor clonality and long-term survival in Adult T-cell Leukemia dlvr.​it/TW4Ppz
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Continuous in situ production of CAR T-cells from in vivo-transduced HSCs is safe and mediates delay in growth of mouse HER2high tumors. dlvr.​it/TW4Pjk
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Single-dose, dual-antigen VSV-based vaccines protect against Crimean-Congo hemorrhagic fever in two lethal mouse models dlvr.​it/TW48Wf #MTMCD
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NIS reporter gene PET imaging reveals expansion kinetics and localization of anti-CD20 CAR-T in mice and nonhuman primates dlvr.​it/TW3vcP #MTMCD
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VP2-TALE fusions increase AAV2 capsid filling with payload bearing cognate TALE target sequences as determined by qPCR/ELISA-based estimation dlvr.​it/TW3hhz #MTMCD
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Baculovirus helper functions during recombinant adeno-associated virus vector production in insect cells for gene therapy applications dlvr.​it/TW3hdQ #MTMCD
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249
Subcutaneous administration of adipose-tropic leptin gene therapy for congenital generalized lipodystrophy dlvr.​it/TW3hZ1 #MTMCD
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The Circular RNA circSKA3 Binds Integrin β1 to Induce Invadopodium Formation Enhancing Breast Cancer Invasion dlvr.​it/TW2sjD #genetherapy #celltherapy
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Molecular Therapy Family Highlights dlvr.​it/TW2sdd #genetherapy #celltherapy
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Least but not last: Durable C5 silencing for IgA nephropathy dlvr.​it/TW2sb7 #genetherapy #celltherapy
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From worms to therapeutics: Helminth-derived immunomodulatory proteins and peptides as potential therapeutics for inflammatory diseases dlvr.​it/TW2jLM #genetherapy #celltherapy
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