Molecular Therapy-Nucleic Acids

Papers
(The H4-Index of Molecular Therapy-Nucleic Acids is 46. 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 2022-06-01 to 2026-06-01.)
ArticleCitations
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres222
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa122
Forced enhancer-promoter rewiring to alter gene expression in animal models116
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells108
AAV-vectored base editor trans-splicing delivers dystrophin repair106
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders91
An oversized AAV8 vector to deliver CPS189
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives87
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR286
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells84
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma76
Unlocking RNA mysteries: Predicting subcellular localizations with AI75
A novel gene therapy platform for the treatment of type 2 diabetes and obesity74
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis73
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells72
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants72
Implications of circular transcripts in DM1 pathomechanism66
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection65
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation64
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 161
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-560
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration59
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies59
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration59
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles59
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers57
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion56
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease56
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy54
MASQ: A multiplex qPCR platform for quantitative alternative splicing analysis in clinical specimens52
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations52
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling52
Comparative immunogenic and structural characterization of virus-like particle and inactivated whole-virion vaccines against enterovirus D6851
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine51
cncFinder: A graph-attention-network-based interpretable learning model to identify bifunctional long non-coding RNAs50
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome50
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer49
Seeking stability for gene addition in inborn errors of metabolism49
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix49
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease49
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides49
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models48
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling47
Topical application of a CCL22-binding aptamer suppresses contact allergy47
Right on target: The next class of efficient, safe, and specific RNAi triggers46
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration46
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD46
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