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




















