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

CochleaNet: deep learning-based image analysis for cochlear connectomics and gene therapy dlvr.​it/TW2Ydz #genetherapy #celltherapy

ATMPs in Oncology: A Comprehensive Review of CAR-T and CIK Cell Therapies within the European Regulatory Framework dlvr.​it/TW2Yck

A Scalable, Antibody-Free Lipid Nanoparticle Achieves Preferential mRNA Delivery to Human HSPCs In Vivo Without Conditioning dlvr.​it/TW2Q1n #genetherapy #celltherapy

Closed-loop inhibitory metabotropic glutamate receptor gene therapy for focal refractory epilepsy dlvr.​it/TW1y1Z #genetherapy #celltherapy

A Combinatorial mRNA Therapy for Treating Rheumatoid Arthritis and Osteoarthritis by Inhibiting Inflammation and Preventing Cartilage Degeneration dlvr.​it/TW1rJL #genetherapy #celltherapy

Host factor IRS4 impairs antitumor efficacy of oncolytic alphavirus via proteasomal viral protein degradation dlvr.​it/TW1rDv #genetherapy #celltherapy

Targeting the provirus in HIV-1 latently infected cells with a cell-activation inducible CRISPR-Cas9 system: an in vitro proof of concept dlvr.​it/TW1R7t #MTNA