Molecular Therapy-Nucleic Acids

Papers
(The H4-Index of Molecular Therapy-Nucleic Acids is 44. 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 2022-05-01 to 2026-05-01.)
ArticleCitations
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres215
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa119
Forced enhancer-promoter rewiring to alter gene expression in animal models111
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration106
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy101
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells89
AAV-vectored base editor trans-splicing delivers dystrophin repair87
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders86
An oversized AAV8 vector to deliver CPS183
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives82
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR274
cncFinder: A graph-attention-network-based interpretable learning model to identify bifunctional long non-coding RNAs73
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease72
Seeking stability for gene addition in inborn errors of metabolism72
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells69
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease68
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma63
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion63
A novel gene therapy platform for the treatment of type 2 diabetes and obesity61
Unlocking RNA mysteries: Predicting subcellular localizations with AI61
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation59
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling58
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 158
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-558
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration57
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome55
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants55
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations54
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers52
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells51
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides51
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer50
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis49
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine49
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles49
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix49
Implications of circular transcripts in DM1 pathomechanism47
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection47
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies47
Recent advances in CRISPR-Cas9-based genome insertion technologies46
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models46
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations46
Topical application of a CCL22-binding aptamer suppresses contact allergy45
miR-6077 promotes cisplatin/pemetrexed resistance in lung adenocarcinoma via CDKN1A/cell cycle arrest and KEAP1/ferroptosis pathways44
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route44
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia44
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