Phosphocholine Headgroup Chemistry Alters the Extrahepatic Accumulation of Lipid Conjugated siRNA dlvr.it/T7Vb20 #MTNA

Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping dlvr.it/T7TQvB #MTNA

A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and viral replication dlvr.it/T7T35Q #MTNA

Decoding the specificity of m6A RNA methylation and its implication in cancer therapy dlvr.it/T7SfWQ #genetherapy #celltherapy

Newly developed oral bioavailable EHMT2 inhibitor as a potential epigenetic therapy for Prader-Willi syndrome dlvr.it/T7SBN6 #genetherapy #celltherapy

Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A dlvr.it/T7R6Nd #genetherapy #celltherapy

Gene therapy for Lafora disease in the Epm2a-/- mouse model dlvr.it/T7QnqV #genetherapy #celltherapy

Evaluation of [18F]fluoroestradiol and ChRERα as a gene expression PET reporter system in rhesus monkey brain dlvr.it/T7QV3d #genetherapy #celltherapy

Neuroglobin overexpression in cerebellar neurons of Harlequin mice improves mitochondrial homeostasis and reduces ataxic behavior dlvr.it/T7Q91R #genetherapy #celltherapy

AAV-mediated gene therapy for Sialidosis dlvr.it/T7PLxh #genetherapy #celltherapy

The novel potential therapeutic target PSMP/MSMP promotes acute kidney injury via CCR2 dlvr.it/T7P2R0 #genetherapy #celltherapy

In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model dlvr.it/T7NkW0 #genetherapy #celltherapy

Empowering virus-free CAR immune cell therapies dlvr.it/T7NNn8 #genetherapy #celltherapy

Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells dlvr.it/T7MTyF #genetherapy #celltherapy

Efficiently targeting neuroblastoma (NB) by the combination of anti-ROR1 CAR NK cells and N-803 in-vitro and in-vivo of NB xenografts dlvr.it/T7M3y9

Deletion of CD38 enhances CD19 chimeric antigen receptor T cell function dlvr.it/T7Ldd5

CDKN2A copy number alteration in bladder cancer: integrative analysis in patient-derived xenografts and cancer patients dlvr.it/T7L7Fx

Targeted rescue of synaptic plasticity improves cognitive decline in sepsis-associated encephalopathy dlvr.it/T7JxyR #genetherapy #celltherapy

Toward the full potential of mRNA therapeutics dlvr.it/T7JVXH #genetherapy #celltherapy

Bispecific CAR-T cells targeting FAP and GPC3 have the potential to treat hepatocellular carcinoma dlvr.it/T7J2tS