Molecular Therapy-Nucleic Acids

Papers
(The H4-Index of Molecular Therapy-Nucleic Acids is 42. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2022-08-01 to 2026-08-01.)
ArticleCitations
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres237
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa146
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells135
AAV-vectored base editor trans-splicing delivers dystrophin repair119
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders113
An oversized AAV8 vector to deliver CPS194
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives89
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR285
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells82
Unlocking RNA mysteries: Predicting subcellular localizations with AI78
A novel gene therapy platform for the treatment of type 2 diabetes and obesity78
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis74
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells73
Implications of circular transcripts in DM1 pathomechanism70
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation68
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 168
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration65
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease64
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling63
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease61
Seeking stability for gene addition in inborn errors of metabolism61
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection60
Forced enhancer-promoter rewiring to alter gene expression in animal models59
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine59
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy57
Intranasal Tat-modified PEG-PCL nanomicelles delivering anti-RelA siRNA attenuate ischemia-reperfusion injury in transient MCAO mice56
Comparative immunogenic and structural analysis of virus-like particle and inactivated whole-virion vaccines against enterovirus D6856
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion56
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix56
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies54
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants51
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration51
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations51
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer50
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers50
cncFinder: A graph-attention-network-based interpretable learning model to identify bifunctional long non-coding RNAs47
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides47
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-546
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles45
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome45
Simultaneous Quantitative Detection of Multiple Low-Frequency Variants by High-Dynamic-Range Capillary Electrophoresis44
MASQ: A multiplex qPCR platform for quantitative alternative splicing analysis in clinical specimens42
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