Molecular Therapy-Nucleic Acids

Papers
(The TQCC of Molecular Therapy-Nucleic Acids is 14. 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 2021-12-01 to 2025-12-01.)
ArticleCitations
RETRACTED: HOXA5-miR-574-5p axis promotes adipogenesis and alleviates insulin resistance165
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-5103
Novel insights into the interaction between N6-methyladenosine modification and circular RNA91
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells90
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy89
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion86
Emerging roles of circRNAs in the pathological process of myocardial infarction81
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres80
Highly efficient A-to-G base editing by ABE8.17 in rabbits80
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease79
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis79
AAV-vectored base editor trans-splicing delivers dystrophin repair79
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders75
Unlocking RNA mysteries: Predicting subcellular localizations with AI72
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells71
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives71
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies71
An oversized AAV8 vector to deliver CPS170
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR265
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration64
Implications of circular transcripts in DM1 pathomechanism64
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells63
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma61
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 160
Forced enhancer-promoter rewiring to alter gene expression in animal models60
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers60
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles59
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling59
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation58
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration54
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine54
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa53
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants53
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations52
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer52
A novel gene therapy platform for the treatment of type 2 diabetes and obesity52
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection51
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix51
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome51
Antitumor effects of chemically modified miR-143 lipoplexes in a mouse model of pelvic colorectal cancer via myristoylated alanine-rich C kinase substrate downregulation50
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition50
Structural variants and modifications of hammerhead ribozymes targeting influenza A virus conserved structural motifs50
Towards SINEUP-based therapeutics: Design of an in vitro synthesized SINEUP RNA50
Right on target: The next class of efficient, safe, and specific RNAi triggers50
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery49
Rapid molecular imaging of active thrombi in vivo using aptamer-antidote probes49
Nucleic acid delivery for pathology treatment: RNA tissue delivery49
Extracellular viral microRNAs as biomarkers of virus infection in human cells49
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling49
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models48
Effect of degeneration stage on non-viral tissue transfection of rd10 retina ex vivo48
miR-204 suppresses cancer stemness and enhances osimertinib sensitivity in non-small cell lung cancer by targeting CD4448
Engineering B cells with customized therapeutic responses using a synthetic circuit47
tRF-1001: A potential therapeutic target for ocular neovascular diseases47
Redefining NSP12 activity in SARS-CoV-2 and its regulation by NSP8 and NSP746
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD46
Chemical modification of uridine modulates mRNA-mediated proinflammatory and antiviral response in primary human macrophages46
HnRNP-L-regulated circCSPP1/miR-520h/EGR1 axis modulates autophagy and promotes progression in prostate cancer45
Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss45
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency45
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration45
Topical application of a CCL22-binding aptamer suppresses contact allergy45
RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges44
Development of a novel PROTAC using the nucleic acid aptamer as a targeting ligand for tumor selective degradation of nucleolin44
miR-6077 promotes cisplatin/pemetrexed resistance in lung adenocarcinoma via CDKN1A/cell cycle arrest and KEAP1/ferroptosis pathways44
RETRACTED: LncRNA PVT1 promotes tumorigenesis of glioblastoma by recruiting COPS5 to deubiquitinate and stabilize TRIM2444
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations44
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells44
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges43
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia43
circPTEN suppresses colorectal cancer progression through regulating PTEN/AKT pathway43
Targeting triple-negative breast cancer cells with a β1-integrin binding aptamer43
A genome-wide CRISPR screen unveils the endosomal maturation protein WDR91 as a promoter of productive ASO activity in melanoma42
Cancer immunomodulation using bispecific aptamers42
CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA42
Double-stranded RNA induction asa potential dynamic biomarkerfor DNA-demethylating agents41
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route41
Inhibition of microRNA-30a alleviates vascular remodeling in pulmonary arterial hypertension40
Heterogeneity of human corneal endothelium implicates lncRNA NEAT1 in Fuchs endothelial corneal dystrophy40
Recent advances in CRISPR-Cas9-based genome insertion technologies40
Molecular treatment options for patients carrying KIAA0586/TALPID3 variants39
miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC39
Lung transcriptome of nonhuman primates exposed to total- and partial-body irradiation39
Retraction Notice to: Upregulation of OIP5-AS1 Predicts Poor Prognosis and Contributes to Thyroid Cancer Cell Proliferation and Migration38
A proximity-labeling-based approach to directly detect mRNA delivery to specific subcellular locations37
Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis37
Hepatic-stellate-cell-targeted delivery of PU.1 decoy ODN by Apt-Tan attenuates liver fibrosis in mice36
DNA electroporation in a vacuum: A “shocking” innovation for vaccines36
Targeting microRNA-145-mediated progressive phenotypes of early bladder cancer in a molecularly defined in vivo model35
TGFB3-AS1 promotes Hcy-induced inflammation of macrophages via inhibiting the maturity of miR-144 and upregulating Rap1a35
The roles of DNA methylation and hydroxymethylation at short interspersed nuclear elements in the hypothalamic arcuate nucleus during puberty35
Retraction Notice to: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma35
CircSTK40 contributes to recurrent implantation failure via modulating the HSP90/AKT/FOXO1 axis35
Epigenetic regulation of MIR145 core promoter controls miR-143/145 cluster in bladder cancer progression and treatment outcome35
Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs34
Global RNA editing identification and characterization during human pluripotent-to-cardiomyocyte differentiation34
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin34
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture34
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective34
miR-363 Alleviates Detrusor Fibrosis via the TGF-β1/Smad Signaling Pathway by Targeting Col1a2 in Rat Models of STZ-Induced T2DM34
SHMT2 is essential for mammalian preimplantation embryonic development through de novo biosynthesis of nucleotide metabolites33
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM133
Nanoparticle-formulated mRNA encoding engineered multivalent SIRPα-Fc fusion proteins shows robust anti-cancer activity in preclinical models33
Expanding RNA editing toolkit using an IDR-based strategy33
TREX2 enables efficient genome disruption mediated by paired CRISPR-Cas9 nickases that generate 3′-overhanging ends33
CABE-RY: A PAM-flexible dual-mutation base editor for reliable modeling of multi-nucleotide variants32
Multi-omics integrative analysis reveals novel genetic loci and candidate genes for ischemic stroke32
In vivo precision base editing to rescue mouse models of disease32
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT32
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles32
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders32
Palmitoyl transferases act as potential regulators of tumor-infiltrating immune cells and glioma progression32
PPM1H is down-regulated by ATF6 and dephosphorylates p-RPS6KB1 to inhibit progression of hepatocellular carcinoma31
Nimbolide-based nanomedicine inhibits breast cancer stem-like cells by epigenetic reprogramming of DNMTs-SFRP1-Wnt/β-catenin signaling axis31
Combining independent protein and cellular SELEX with bioinformatic analysis may allow high affinity aptamer hit discovery31
Expanding DdCBE-mediated targeting scope to aC motif preference in rat31
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response31
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors30
miR-21 mimic blocks obesity in mice: A novel therapeutic option30
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways30
Gene therapy to enhance angiogenesis in chronic wounds30
CircRNA Chordc1 protects mice from abdominal aortic aneurysm by contributing to the phenotype and growth of vascular smooth muscle cells30
Biologically stable threose nucleic acid-based probes for real-time microRNA detection and imaging in living cells30
Ubiquitinated ligation protein NEDD4L participates in MiR-30a-5p attenuated atherosclerosis by regulating macrophage polarization and lipid metabolism30
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease30
Dynamic regulation of NeuroD1 expression level by a novel viral construct during astrocyte-to-neuron reprogramming30
mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment30
Hsa_circ_0024093 accelerates VSMC proliferation via miR-4677-3p/miR-889-3p/USP9X/YAP1 axis in in vitro model of lower extremity ASO29
Antisense transcription from lentiviral gene targeting linked to an integrated stress response in colorectal cancer cells29
Testis electroporation coupled with autophagy inhibitor to treat non-obstructive azoospermia29
Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy29
Local intraluminal delivery of a smooth muscle-targeted RNA ligand inhibits neointima growth in a porcine model of peripheral vascular disease29
m6A-induced repression of SIAH1 facilitates alternative splicing of androgen receptor variant 7 by regulating CPSF129
Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets29
miR-99a regulates CD4+ T cell differentiation and attenuates experimental autoimmune encephalomyelitis by mTOR-mediated glycolysis29
MicroRNA Profiling in Paired Left and Right Eyes, Lungs, and Testes of Normal Mice29
An important resource and analytic platform for human and mouse cardiovascular-related cis-regulatory elements29
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control28
MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target28
Base editing corrects the common Salla disease SLC17A5 c.115C>T variant28
MVA-HBVac—A novel vaccine vector that allows pan-genotypic targeting of hepatitis B virus by therapeutic vaccination28
Self-assembling short immunostimulatory duplex RNAs with broad-spectrum antiviral activity28
mRNA-encoded ACE2 decoy lipid nanoparticles for neutralizing SARS-CoV-2 variants28
Retraction Notice to: LncRNA LINC00963 Promotes Tumorigenesis and Radioresistance in Breast Cancer by Sponging miR-324-3p and Inducing ACK1 Expression28
Unlocking mRNA-driven CRISPR-Cas9 gene therapy via optimizing mRNA and the delivery vectors27
Combining an optimized mRNA template with a double purification process allows strong expression of in vitro transcribed mRNA27
Hepatocyte-derived exosomal miR-27a activates hepatic stellate cells through the inhibition of PINK1-mediated mitophagy in MAFLD27
Enhancing natural killer cells proliferation and cytotoxicity using imidazole-based lipid nanoparticles encapsulating interleukin-2 mRNA27
Engineering miniature CRISPR-Cas Un1Cas12f1 for efficient base editing27
CRISPR-Cas9-mediated homology-directed repair for precise gene editing27
Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing27
Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies27
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice27
Delivery of therapeutic small interfering RNA: The current patent-based landscape27
Selection of DNA aptamers that prevent the fibrillization of α-synuclein protein in cellular and mouse models26
Recent advances in crosstalk between N6-methyladenosine (m6A) modification and circular RNAs in cancer26
Gene network landscape of mouse splenocytes reveals integrin complex as the A151 ODN-responsive hub molecule in the immune transcriptome26
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells26
Intratumoral electroporation of a self-amplifying RNA expressing IL-12 induces antitumor effects in mouse models of cancer26
Dynamic regulatory networks of T cell trajectory dissect transcriptional control of T cell state transition26
Retraction Notice to: Schizandrin A Protects Human Retinal Pigment Epithelial Cell Line ARPE-19 against HG-Induced Cell Injury by Regulation of miR-14526
An instructive attempt on developing aptamer-constructed PROTAC for breast cancer treatment26
siRNA goes after diseases of the bone26
Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations26
FUBP3: A new player in HIV-1 transcriptional activation and immune regulation26
A model system for antiviral siRNA therapeutics using exosome-based delivery26
SINEUPs to boost translation25
Aptamer-based factor IXa inhibition preserves hemostasis and prevents thrombosis in a piglet model of ECMO25
Dendritic siRNA conjugate riding albumin for targeted delivery to solid tumors25
Retraction Notice to: GLP2 Promotes Directed Differentiation from Osteosarcoma Cells to Osteoblasts and Inhibits Growth of Osteosarcoma Cells25
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 125
Opposing impacts of DNA polyplex crosslinking on delivery efficiency and vaccine responses25
Retraction Notice to: TNFAIP8 Promotes Cisplatin Chemoresistance in Triple-Negative Breast Cancer by Repressing p53-Mediated miR-205-5p Expression25
Axl deficiency promotes preeclampsia and vascular malformations in mice25
A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases24
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina24
Human opsin restoration by histone methylation using methyltransferase fusion protein SETD7-dCas924
Pharmacoepitranscriptomic landscape revealing m6A modification could be a drug-effect biomarker for cancer treatment24
Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity24
Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy24
Modulation of miR-181 influences dopaminergic neuronal degeneration in a mouse model of Parkinson’s disease24
Manipulating the delivery and immunogenicity of DNA vaccines through the addition of CB[8] to cationic polymers24
Promising strategies employing nucleic acids as antimicrobial drugs24
MicroRNA-378 contributes to osteoarthritis by regulating chondrocyte autophagy and bone marrow mesenchymal stem cell chondrogenesis24
Aberrant HSF1 signaling activation underlies metformin amelioration of myocardial infarction in mice24
Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse24
Plug-and-play nucleic acid-mediated multimerization of biparatopic nanobodies for molecular imaging23
Enhancing mRNA translation efficiency by introducing sequence optimized AU-rich elements in 3′ UTR via HuR anchorage23
Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications23
Lipid nanoparticle-encapsulated microRNA-192: an anti-inflammatory adjuvant that enhances vaccine efficacy in aged mice23
Unveiling the role of PUS7-mediated pseudouridylation in host protein interactions specific for the SARS-CoV-2 RNA genome23
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects23
Base editing in humanized dystrophic mice23
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV23
Rethinking CRISPR delivery for liver-targeted gene editing: The case for spatially fractionated intra-arterial approaches23
Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency23
Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections23
Blood pressure reduction through brain delivery of nanoparticles loaded with plasmid DNA encoding angiotensin receptor shRNA22
Retraction Notice to: MTTL3 upregulates microRNA-1246 to promote occurrence and progression of NSCLC via targeting paternally expressed gene 322
Long noncoding RNA LUCAT1 enhances the survival and therapeutic effects of mesenchymal stromal cells post-myocardial infarction22
Analytical approach for identification and mechanistic insights into mRNA-lipid adduct formation22
Tubule-specific deletion of LincRNA-p21 ameliorates lipotoxic kidney injury22
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells22
Self-attention enabled deep learning of dihydrouridine (D) modification on mRNAs unveiled a distinct sequence signature from tRNAs22
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF22
A potential therapeutic approach for tauopathies22
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity22
Broadly neutralizing aptamers to SARS-CoV-2: A diverse panel of modified DNA antiviral agents22
MiR-142a-3p: A novel ACh receptor transcriptional regulator in association with peripheral nerve injury22
A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy22
Retraction Notice to: Overexpression of microRNA-203 Suppresses Proliferation, Invasion, and Migration while Accelerating Apoptosis of CSCC Cell Line SCL-122
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis-regulation in human and mouse22
A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication22
Deciphering a TB-related DNA methylation biomarker and constructing a TB diagnostic classifier21
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes21
Overexpression of lncRNA EPB41L4A-AS1 Induces Metabolic Reprogramming in Trophoblast Cells and Placenta Tissue of Miscarriage21
Recent applications, future perspectives, and limitations of the CRISPR-Cas system21
mRNA vaccine expressing enterovirus D68 virus-like particles induces potent neutralizing antibodies and protects against infection21
Restoration of myogenesis in ALS-myocytes through miR-26a-5p-mediated Smad4 inhibition and its impact on motor neuron development21
Conjugated STING agonists21
Adenovirus expressing nc886, an anti-interferon and anti-apoptotic non-coding RNA, is an improved gene delivery vector21
RNA activation of CEBPA improves leukemia treatment21
Role of long non-coding RNAs in cancer: From subcellular localization to nanoparticle-mediated targeted regulation21
Live-cell imaging of microRNA expression with post-transcriptional feedback control21
Royal jelly extracellular vesicles promote wound healing by modulating underlying cellular responses21
Bile acid accumulation induced by miR-122 deficiency in liver parenchyma promotes cancer cell growth in hepatocellular carcinoma21
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties21
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus21
Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope21
Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site20
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells20
Retraction Notice to: Exosome miR-155 Derived from Gastric Carcinoma Promotes Angiogenesis by Targeting the c-MYB/VEGF Axis of Endothelial Cells20
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection20
Inducible miR-1224 silences cerebrovascular Serpine1 and restores blood flow to the stroke-affected site of the brain20
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy20
A cystic fibrosis gene editing approach that is on target20
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice20
Replication not required: mRNA vaccines take on non-enveloped viruses20
Delivering therapeutic RNA into the brain using extracellular vesicles20
Unveiling the emerging functions of nuclear actin in gene function regulation20
Toward learning the rules that predict siRNA efficacy19
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model19
Retraction Notice to: lncRNA MALAT1 Accelerates Wound Healing of Diabetic Mice Transfused with Modified Autologous Blood via the HIF-1α Signaling Pathway19
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide19
Binding of G-quadruplex DNA and serum albumins by synthetic non-proteinogenic amino acids: Implications for c-Myc-related anticancer activity and drug delivery19
Retraction Notice to: MicroRNA-140 Inhibits the Epithelial-Mesenchymal Transition and Metastasis in Colorectal Cancer19
HIF-1α or HOTTIP/CTCF Promotes Head and Neck Squamous Cell Carcinoma Progression and Drug Resistance by Targeting HOXA919
Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome19
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease19
Gal-3 activates Tyro3 to ameliorate ferroptosis of hippocampal neurons after traumatic brain injury19
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