Molecular Therapy-Nucleic Acids

Papers
(The TQCC of Molecular Therapy-Nucleic Acids is 15. 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 2021-05-01 to 2025-05-01.)
ArticleCitations
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-5153
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy118
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration86
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives77
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling75
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres74
Activating the Cpx response induces tolerance to antisense PNA delivered by an arginine-rich peptide in Escherichia coli74
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation71
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells70
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders69
AAV-vectored base editor trans-splicing delivers dystrophin repair69
RETRACTED: H3K27 demethylase KDM6B aggravates ischemic brain injury through demethylation of IRF4 and Notch2-dependent SOX9 activation69
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma68
HOXA5-miR-574-5p axis promotes adipogenesis and alleviates insulin resistance67
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 165
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations65
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer65
Exosomal miR-590-3p derived from cancer-associated fibroblasts confers radioresistance in colorectal cancer65
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix63
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease63
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells62
Unlocking RNA mysteries: Predicting subcellular localizations with AI62
Emerging roles of circRNAs in the pathological process of myocardial infarction59
Forced enhancer-promoter rewiring to alter gene expression in animal models59
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome58
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles57
Novel insights into the interaction between N6-methyladenosine modification and circular RNA56
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers56
Highly efficient A-to-G base editing by ABE8.17 in rabbits55
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis54
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion52
Right on target: The next class of efficient, safe, and specific RNAi triggers51
Engineering B cells with customized therapeutic responses using a synthetic circuit51
tRF-1001: A potential therapeutic target for ocular neovascular diseases51
RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges49
Structural variants and modifications of hammerhead ribozymes targeting influenza A virus conserved structural motifs49
CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA48
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells48
Nucleic acid delivery for pathology treatment: RNA tissue delivery47
Redefining NSP12 activity in SARS-CoV-2 and its regulation by NSP8 and NSP747
Extracellular viral microRNAs as biomarkers of virus infection in human cells47
miR-6077 promotes cisplatin/pemetrexed resistance in lung adenocarcinoma via CDKN1A/cell cycle arrest and KEAP1/ferroptosis pathways46
miR-204 suppresses cancer stemness and enhances osimertinib sensitivity in non-small cell lung cancer by targeting CD4445
HnRNP-L-regulated circCSPP1/miR-520h/EGR1 axis modulates autophagy and promotes progression in prostate cancer44
Cancer immunomodulation using bispecific aptamers44
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations44
Double-stranded RNA induction asa potential dynamic biomarkerfor DNA-demethylating agents44
Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss44
Inhibition of microRNA-30a alleviates vascular remodeling in pulmonary arterial hypertension44
Towards SINEUP-based therapeutics: Design of an in vitro synthesized SINEUP RNA43
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models43
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD43
RETRACTED: LncRNA PVT1 promotes tumorigenesis of glioblastoma by recruiting COPS5 to deubiquitinate and stabilize TRIM2443
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition43
circPTEN suppresses colorectal cancer progression through regulating PTEN/AKT pathway42
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration42
Topical application of a CCL22-binding aptamer suppresses contact allergy42
Heterogeneity of human corneal endothelium implicates lncRNA NEAT1 in Fuchs endothelial corneal dystrophy41
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency41
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling41
Antitumor effects of chemically modified miR-143 lipoplexes in a mouse model of pelvic colorectal cancer via myristoylated alanine-rich C kinase substrate downregulation41
Chemical modification of uridine modulates mRNA-mediated proinflammatory and antiviral response in primary human macrophages41
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route41
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery40
Recent advances in CRISPR-Cas9-based genome insertion technologies40
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia40
Rapid molecular imaging of active thrombi in vivo using aptamer-antidote probes40
Development of a novel PROTAC using the nucleic acid aptamer as a targeting ligand for tumor selective degradation of nucleolin40
Dual effects of N6-methyladenosine on cancer progression and immunotherapy40
Recent research progress on circular RNAs: Biogenesis, properties, functions, and therapeutic potential39
Targeting triple-negative breast cancer cells with a β1-integrin binding aptamer39
Naringin improves sepsis-induced intestinal injury by modulating macrophage polarization via PPARγ/miR-21 axis39
Biological roles and therapeutic potential of circular RNAs in osteoarthritis39
PPM1H is down-regulated by ATF6 and dephosphorylates p-RPS6KB1 to inhibit progression of hepatocellular carcinoma39
Epigenetic regulation of MIR145 core promoter controls miR-143/145 cluster in bladder cancer progression and treatment outcome38
miR-140 inhibits osteosarcoma progression by impairing USP22-mediated LSD1 stabilization and promoting p21 expression38
Nimbolide-based nanomedicine inhibits breast cancer stem-like cells by epigenetic reprogramming of DNMTs-SFRP1-Wnt/β-catenin signaling axis37
Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs37
Combining independent protein and cellular SELEX with bioinformatic analysis may allow high affinity aptamer hit discovery37
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective36
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways36
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin36
Global RNA editing identification and characterization during human pluripotent-to-cardiomyocyte differentiation36
TREX2 enables efficient genome disruption mediated by paired CRISPR-Cas9 nickases that generate 3′-overhanging ends36
The roles of DNA methylation and hydroxymethylation at short interspersed nuclear elements in the hypothalamic arcuate nucleus during puberty35
AhR/miR-23a-3p/PKCα axis contributes to memory deficits in ovariectomized and normal aging female mice35
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT35
ERα determines the chemo-resistant function of mutant p53 involving the switch between lincRNA-p21 and DDB2 expressions35
CircRNA Chordc1 protects mice from abdominal aortic aneurysm by contributing to the phenotype and growth of vascular smooth muscle cells34
Ubiquitinated ligation protein NEDD4L participates in MiR-30a-5p attenuated atherosclerosis by regulating macrophage polarization and lipid metabolism34
DNA electroporation in a vacuum: A “shocking” innovation for vaccines33
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture33
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles33
CircSTK40 contributes to recurrent implantation failure via modulating the HSP90/AKT/FOXO1 axis33
CABE-RY: A PAM-flexible dual-mutation base editor for reliable modeling of multi-nucleotide variants33
Expanding DdCBE-mediated targeting scope to aC motif preference in rat33
miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC32
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM132
Retraction Notice to: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma32
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response32
Retraction Notice to: Upregulation of OIP5-AS1 Predicts Poor Prognosis and Contributes to Thyroid Cancer Cell Proliferation and Migration32
Expanding RNA editing toolkit using an IDR-based strategy32
Hepatocyte-targeting and microenvironmentally responsive glycolipid-like polymer micelles for gene therapy of hepatitis B31
TGFB3-AS1 promotes Hcy-induced inflammation of macrophages via inhibiting the maturity of miR-144 and upregulating Rap1a31
miR-363 Alleviates Detrusor Fibrosis via the TGF-β1/Smad Signaling Pathway by Targeting Col1a2 in Rat Models of STZ-Induced T2DM31
Targeting microRNA-145-mediated progressive phenotypes of early bladder cancer in a molecularly defined in vivo model31
Lung transcriptome of nonhuman primates exposed to total- and partial-body irradiation31
Biologically stable threose nucleic acid-based probes for real-time microRNA detection and imaging in living cells31
miR-21 mimic blocks obesity in mice: A novel therapeutic option30
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders30
Antisense transcription from lentiviral gene targeting linked to an integrated stress response in colorectal cancer cells30
Palmitoyl transferases act as potential regulators of tumor-infiltrating immune cells and glioma progression30
Gene therapy to enhance angiogenesis in chronic wounds30
Testis electroporation coupled with autophagy inhibitor to treat non-obstructive azoospermia30
Nanoparticle-Formulated mRNA Encoding Engineered Multivalent SIRPα-Fc Fusion Proteins Shows Robust anti-Cancer Activity in Preclinical Models30
LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model30
MicroRNA Profiling in Paired Left and Right Eyes, Lungs, and Testes of Normal Mice30
m6A-induced repression of SIAH1 facilitates alternative splicing of androgen receptor variant 7 by regulating CPSF129
Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy29
miR-99a regulates CD4+ T cell differentiation and attenuates experimental autoimmune encephalomyelitis by mTOR-mediated glycolysis29
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control29
Hsa_circ_0024093 accelerates VSMC proliferation via miR-4677-3p/miR-889-3p/USP9X/YAP1 axis in in vitro model of lower extremity ASO29
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors29
Base editing corrects the common Salla disease SLC17A5 c.115C>T variant29
Delivery of therapeutic small interfering RNA: The current patent-based landscape29
PCAT-1 facilitates breast cancer progression via binding to RACK1 and enhancing oxygen-independent stability of HIF-1α29
LRRK2 Antisense Oligonucleotides Ameliorate α-Synuclein Inclusion Formation in a Parkinson’s Disease Mouse Model29
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease29
An important resource and analytic platform for human and mouse cardiovascular-related cis-regulatory elements29
Self-assembling short immunostimulatory duplex RNAs with broad-spectrum antiviral activity29
Hepatocyte-derived exosomal miR-27a activates hepatic stellate cells through the inhibition of PINK1-mediated mitophagy in MAFLD28
Enhancing natural killer cells proliferation and cytotoxicity using imidazole-based lipid nanoparticles encapsulating interleukin-2 mRNA28
Engineering miniature CRISPR-Cas Un1Cas12f1 for efficient base editing28
Local intraluminal delivery of a smooth muscle-targeted RNA ligand inhibits neointima growth in a porcine model of peripheral vascular disease28
Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies28
Combining an optimized mRNA template with a double purification process allows strong expression of in vitro transcribed mRNA28
Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets28
CRISPR-Cas9-mediated homology-directed repair for precise gene editing27
Aberrant HSF1 signaling activation underlies metformin amelioration of myocardial infarction in mice27
A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases27
Retraction Notice to: GLP2 Promotes Directed Differentiation from Osteosarcoma Cells to Osteoblasts and Inhibits Growth of Osteosarcoma Cells27
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice27
Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing27
SINEUPs to boost translation27
Retraction Notice to: Schizandrin A Protects Human Retinal Pigment Epithelial Cell Line ARPE-19 against HG-Induced Cell Injury by Regulation of miR-14527
Gene network landscape of mouse splenocytes reveals integrin complex as the A151 ODN-responsive hub molecule in the immune transcriptome26
Plug-and-play nucleic acid-mediated multimerization of biparatopic nanobodies for molecular imaging26
Intratumoral electroporation of a self-amplifying RNA expressing IL-12 induces antitumor effects in mouse models of cancer26
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina26
A model system for antiviral siRNA therapeutics using exosome-based delivery26
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV26
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells25
Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy25
siRNA goes after diseases of the bone25
Retraction Notice to: TNFAIP8 Promotes Cisplatin Chemoresistance in Triple-Negative Breast Cancer by Repressing p53-Mediated miR-205-5p Expression25
Unveiling the role of PUS7-mediated pseudouridylation in host protein interactions specific for the SARS-CoV-2 RNA genome25
Axl deficiency promotes preeclampsia and vascular malformations in mice25
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 125
An instructive attempt on developing aptamer-constructed PROTAC for breast cancer treatment24
Modulation of miR-181 influences dopaminergic neuronal degeneration in a mouse model of Parkinson’s disease24
Promising strategies employing nucleic acids as antimicrobial drugs24
Dynamic regulatory networks of T cell trajectory dissect transcriptional control of T cell state transition24
MicroRNA-378 contributes to osteoarthritis by regulating chondrocyte autophagy and bone marrow mesenchymal stem cell chondrogenesis24
Selection of DNA aptamers that prevent the fibrillization of α-synuclein protein in cellular and mouse models24
Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations24
Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity24
Pharmacoepitranscriptomic landscape revealing m6A modification could be a drug-effect biomarker for cancer treatment24
Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications24
Aptamer-based factor IXa inhibition preserves hemostasis and prevents thrombosis in a piglet model of ECMO24
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects24
ncRNAs-mediated high expression of SEMA3F correlates with poor prognosis and tumor immune infiltration of hepatocellular carcinoma24
A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy23
A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication23
Blood pressure reduction through brain delivery of nanoparticles loaded with plasmid DNA encoding angiotensin receptor shRNA23
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity23
Recent advances in crosstalk between N6-methyladenosine (m6A) modification and circular RNAs in cancer23
Long noncoding RNA LUCAT1 enhances the survival and therapeutic effects of mesenchymal stromal cells post-myocardial infarction23
MiR-142a-3p: A novel ACh receptor transcriptional regulator in association with peripheral nerve injury23
Delivering therapeutic RNA into the brain using extracellular vesicles23
A potential therapeutic approach for tauopathies23
Inducible miR-1224 silences cerebrovascular Serpine1 and restores blood flow to the stroke-affected site of the brain23
Aptamer-mediated transcriptional gene silencing of Foxp3 inhibits regulatory T cells and potentiates antitumor response23
Base editing in humanized dystrophic mice23
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy23
Tubule-specific deletion of LincRNA-p21 ameliorates lipotoxic kidney injury22
Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site22
Deciphering a TB-related DNA methylation biomarker and constructing a TB diagnostic classifier22
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties22
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus22
Self-attention enabled deep learning of dihydrouridine (D) modification on mRNAs unveiled a distinct sequence signature from tRNAs22
Retraction Notice to: MTTL3 upregulates microRNA-1246 to promote occurrence and progression of NSCLC via targeting paternally expressed gene 322
A more efficient CRISPR-Cas12a variant derived from Lachnospiraceae bacterium MA202022
Retraction Notice to: Overexpression of microRNA-203 Suppresses Proliferation, Invasion, and Migration while Accelerating Apoptosis of CSCC Cell Line SCL-122
Emerging roles of circular RNAs in systemic lupus erythematosus22
Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope22
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells22
Adenovirus expressing nc886, an anti-interferon and anti-apoptotic non-coding RNA, is an improved gene delivery vector22
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes22
Live-cell imaging of microRNA expression with post-transcriptional feedback control22
Overexpression of lncRNA EPB41L4A-AS1 Induces Metabolic Reprogramming in Trophoblast Cells and Placenta Tissue of Miscarriage21
Role of long non-coding RNAs in cancer: From subcellular localization to nanoparticle-mediated targeted regulation21
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis-regulation in human and mouse21
Retraction Notice to: MicroRNA-140 Inhibits the Epithelial-Mesenchymal Transition and Metastasis in Colorectal Cancer21
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model21
Immunogenicity of stabilized HIV-1 Env trimers delivered by self-amplifying mRNA21
RETRACTED: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma21
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF21
Broadly neutralizing aptamers to SARS-CoV-2: A diverse panel of modified DNA antiviral agents21
Retraction Notice to: lncRNA MALAT1 Accelerates Wound Healing of Diabetic Mice Transfused with Modified Autologous Blood via the HIF-1α Signaling Pathway21
RETRACTED: MTTL3 upregulates microRNA-1246 to promote occurrence and progression of NSCLC via targeting paternally expressed gene 321
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice21
Retraction Notice to: Exosome miR-155 Derived from Gastric Carcinoma Promotes Angiogenesis by Targeting the c-MYB/VEGF Axis of Endothelial Cells21
Royal jelly extracellular vesicles promote wound healing by modulating underlying cellular responses21
Minicircle DNA vectors: A breakthrough in non-viral delivery of CRISPR base editors?21
Retraction Notice to: Circular RNA Profiling Reveals Exosomal circ_0006156 as a Novel Biomarker in Papillary Thyroid Cancer21
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection21
Unveiling the emerging functions of nuclear actin in gene function regulation20
Toward learning the rules that predict siRNA efficacy20
HIF-1α or HOTTIP/CTCF Promotes Head and Neck Squamous Cell Carcinoma Progression and Drug Resistance by Targeting HOXA920
miR-429 RNA therapy as generic strategy to protect against photoreceptor loss20
Harnessing defective interfering particles and lipid nanoparticles for effective delivery of an anti-dengue virus RNA therapy20
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA420
Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs20
EPRIM: An approach of identifying cancer immune-related epigenetic regulators20
Gal-3 activates Tyro3 to ameliorate ferroptosis of hippocampal neurons after traumatic brain injury20
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide20
A cystic fibrosis gene editing approach that is on target20
Novel Fas-TNFR chimeras that prevent Fas ligand-mediated kill and signal synergistically to enhance CAR T cell efficacy20
Cell-type-specific alternative polyadenylation as a therapeutic biomarker in lung cancer progression20
Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome20
Translatomics to explore dynamic differences in immunocytes in the tumor microenvironment19
Analysis and identification of m6A RNA methylation regulators in metastatic osteosarcoma19
miR-940 is a new biomarker with tumor diagnostic and prognostic value19
Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform19
First-in-class transactivator-free, doxycycline-inducible IL-18-engineered CAR-T cells for relapsed/refractory B cell lymphomas19
Chimeric RNA-binding protein-based killing switch targeting hepatocellular carcinoma cells19
mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks19
Advancing peptide siRNA-carrier designs through L/D-amino acid stereochemical modifications to enhance gene silencing19
MicroRNA-451a inhibits gemcitabine-refractory biliary tract cancer progression by suppressing the MIF-mediated PI3K/AKT pathway19
cGAMP-adjuvanted multivalent influenza mRNA vaccines induce broadly protective immunity through cutaneous vaccination in mice19
Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS19
Lipid nanoparticle-encapsulated, chemically modified anti-adenoviral siRNAs inhibit hepatic adenovirus infection in immunosuppressed Syrian hamsters19
Engineering an adenine base editor in human embryonic stem cells with minimal DNA and RNA off-target activities19
Predicting transcription factor binding sites using DNA shape features based on shared hybrid deep learning architecture19
Endoplasmic reticulum stress promotes sorafenib resistance via miR-188-5p/hnRNPA2B1-mediated upregulation of PKM2 in hepatocellular carcinoma19
Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs19
Enhancing arginase 2 expression using target site blockers as a strategy to modulate macrophage phenotype19
Development of a novel PTD-mediated IVT-mRNA delivery platform for potential protein replacement therapy of metabolic/genetic disorders19
lncRNA PRR34-AS1 promotes HCC development via modulating Wnt/β-catenin pathway by absorbing miR-296-5p and upregulating E2F2 and SOX1218
Targeted exon skipping of NF1 exon 17 as a therapeutic for neurofibromatosis type I18
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