Molecular Therapy-Nucleic Acids

Article	Citations
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy	151
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix	99
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling	90
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer	88
RETRACTED: HOXA5-miR-574-5p axis promotes adipogenesis and alleviates insulin resistance	85
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion	84
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations	79
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-5	79
Forced enhancer-promoter rewiring to alter gene expression in animal models	78
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation	78
Emerging roles of circRNAs in the pathological process of myocardial infarction	78
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells	77
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma	70
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome	70
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles	69
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres	68
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR2	66
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives	66
Novel insights into the interaction between N6-methyladenosine modification and circular RNA	64
Highly efficient A-to-G base editing by ABE8.17 in rabbits	63
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis	62
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells	62
AAV-vectored base editor trans-splicing delivers dystrophin repair	60
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders	58
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease	57

An oversized AAV8 vector to deliver CPS1	56
Unlocking RNA mysteries: Predicting subcellular localizations with AI	56
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells	55
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies	52
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection	52
Implications of circular transcripts in DM1 pathomechanism	52
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1	51
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia	51
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers	51
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration	51
RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges	51
Antitumor effects of chemically modified miR-143 lipoplexes in a mouse model of pelvic colorectal cancer via myristoylated alanine-rich C kinase substrate downregulation	50
RETRACTED: LncRNA PVT1 promotes tumorigenesis of glioblastoma by recruiting COPS5 to deubiquitinate and stabilize TRIM24	50
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition	50
Topical application of a CCL22-binding aptamer suppresses contact allergy	49
Extracellular viral microRNAs as biomarkers of virus infection in human cells	49
Towards SINEUP-based therapeutics: Design of an in vitro synthesized SINEUP RNA	49
Redefining NSP12 activity in SARS-CoV-2 and its regulation by NSP8 and NSP7	49
miR-204 suppresses cancer stemness and enhances osimertinib sensitivity in non-small cell lung cancer by targeting CD44	48
Heterogeneity of human corneal endothelium implicates lncRNA NEAT1 in Fuchs endothelial corneal dystrophy	48
CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA	48
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery	48
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling	47
A genome-wide CRISPR screen unveils the endosomal maturation protein WDR91 as a promoter of productive ASO activity in melanoma	47
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells	47
Effect of degeneration stage on non-viral tissue transfection of rd10 retina ex vivo	46
Recent advances in CRISPR-Cas9-based genome insertion technologies	46
Nucleic acid delivery for pathology treatment: RNA tissue delivery	46
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges	46
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration	46
miR-6077 promotes cisplatin/pemetrexed resistance in lung adenocarcinoma via CDKN1A/cell cycle arrest and KEAP1/ferroptosis pathways	45
Double-stranded RNA induction asa potential dynamic biomarkerfor DNA-demethylating agents	44
Right on target: The next class of efficient, safe, and specific RNAi triggers	44
Inhibition of microRNA-30a alleviates vascular remodeling in pulmonary arterial hypertension	44
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models	44
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations	44
Rapid molecular imaging of active thrombi in vivo using aptamer-antidote probes	44
Chemical modification of uridine modulates mRNA-mediated proinflammatory and antiviral response in primary human macrophages	44
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency	43
Targeting triple-negative breast cancer cells with a β1-integrin binding aptamer	43
HnRNP-L-regulated circCSPP1/miR-520h/EGR1 axis modulates autophagy and promotes progression in prostate cancer	43
Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss	42
circPTEN suppresses colorectal cancer progression through regulating PTEN/AKT pathway	42
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD	42
Structural variants and modifications of hammerhead ribozymes targeting influenza A virus conserved structural motifs	42
Engineering B cells with customized therapeutic responses using a synthetic circuit	41
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route	41
Development of a novel PROTAC using the nucleic acid aptamer as a targeting ligand for tumor selective degradation of nucleolin	41
tRF-1001: A potential therapeutic target for ocular neovascular diseases	41
Nanoparticle-formulated mRNA encoding engineered multivalent SIRPα-Fc fusion proteins shows robust anti-cancer activity in preclinical models	40

Cancer immunomodulation using bispecific aptamers	40
Retraction Notice to: Upregulation of OIP5-AS1 Predicts Poor Prognosis and Contributes to Thyroid Cancer Cell Proliferation and Migration	39
Expanding RNA editing toolkit using an IDR-based strategy	39
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective	38
Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis	38
Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs	38
SHMT2 is essential for mammalian preimplantation embryonic development through de novo biosynthesis of nucleotide metabolites	38
Hepatic-stellate-cell-targeted delivery of PU.1 decoy ODN by Apt-Tan attenuates liver fibrosis in mice	36
A proximity-labeling-based approach to directly detect mRNA delivery to specific subcellular locations	36
Molecular treatment options for patients carrying KIAA0586/TALPID3 variants	36
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin	35
miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC	35
In vivo precision base editing to rescue mouse models of disease	35
DNA electroporation in a vacuum: A “shocking” innovation for vaccines	35
Expanding DdCBE-mediated targeting scope to aC motif preference in rat	35
TREX2 enables efficient genome disruption mediated by paired CRISPR-Cas9 nickases that generate 3′-overhanging ends	35
Nimbolide-based nanomedicine inhibits breast cancer stem-like cells by epigenetic reprogramming of DNMTs-SFRP1-Wnt/β-catenin signaling axis	35
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1	34
Multi-omics integrative analysis reveals novel genetic loci and candidate genes for ischemic stroke	34
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways	33
Gene therapy to enhance angiogenesis in chronic wounds	33
miR-21 mimic blocks obesity in mice: A novel therapeutic option	33
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders	33
TGFB3-AS1 promotes Hcy-induced inflammation of macrophages via inhibiting the maturity of miR-144 and upregulating Rap1a	33
Palmitoyl transferases act as potential regulators of tumor-infiltrating immune cells and glioma progression	33
Targeting microRNA-145-mediated progressive phenotypes of early bladder cancer in a molecularly defined in vivo model	33
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles	33
miR-363 Alleviates Detrusor Fibrosis via the TGF-β1/Smad Signaling Pathway by Targeting Col1a2 in Rat Models of STZ-Induced T2DM	32
Lung transcriptome of nonhuman primates exposed to total- and partial-body irradiation	32
Ubiquitinated ligation protein NEDD4L participates in MiR-30a-5p attenuated atherosclerosis by regulating macrophage polarization and lipid metabolism	32
Combining independent protein and cellular SELEX with bioinformatic analysis may allow high affinity aptamer hit discovery	32
Global RNA editing identification and characterization during human pluripotent-to-cardiomyocyte differentiation	32
Epigenetic regulation of MIR145 core promoter controls miR-143/145 cluster in bladder cancer progression and treatment outcome	32
Retraction Notice to: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma	31
The roles of DNA methylation and hydroxymethylation at short interspersed nuclear elements in the hypothalamic arcuate nucleus during puberty	31
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture	30
CircSTK40 contributes to recurrent implantation failure via modulating the HSP90/AKT/FOXO1 axis	30
PPM1H is down-regulated by ATF6 and dephosphorylates p-RPS6KB1 to inhibit progression of hepatocellular carcinoma	30
Biologically stable threose nucleic acid-based probes for real-time microRNA detection and imaging in living cells	30
CABE-RY: A PAM-flexible dual-mutation base editor for reliable modeling of multi-nucleotide variants	30
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response	30
CircRNA Chordc1 protects mice from abdominal aortic aneurysm by contributing to the phenotype and growth of vascular smooth muscle cells	30
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT	30
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control	29
Base editing corrects the common Salla disease SLC17A5 c.115C>T variant	29
CRISPR-Cas9-mediated homology-directed repair for precise gene editing	29
MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target	29
mRNA-encoded ACE2 decoy lipid nanoparticles for neutralizing SARS-CoV-2 variants	29
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors	29
Self-assembling short immunostimulatory duplex RNAs with broad-spectrum antiviral activity	29
Retraction Notice to: LncRNA LINC00963 Promotes Tumorigenesis and Radioresistance in Breast Cancer by Sponging miR-324-3p and Inducing ACK1 Expression	29
An important resource and analytic platform for human and mouse cardiovascular-related cis-regulatory elements	29
Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets	29
MVA-HBVac—A novel vaccine vector that allows pan-genotypic targeting of hepatitis B virus by therapeutic vaccination	29
Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy	29
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease	29
Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies	28
Hsa_circ_0024093 accelerates VSMC proliferation via miR-4677-3p/miR-889-3p/USP9X/YAP1 axis in in vitro model of lower extremity ASO	28
MicroRNA Profiling in Paired Left and Right Eyes, Lungs, and Testes of Normal Mice	28
Combining an optimized mRNA template with a double purification process allows strong expression of in vitro transcribed mRNA	28
Delivery of therapeutic small interfering RNA: The current patent-based landscape	28
miR-99a regulates CD4+ T cell differentiation and attenuates experimental autoimmune encephalomyelitis by mTOR-mediated glycolysis	28
Local intraluminal delivery of a smooth muscle-targeted RNA ligand inhibits neointima growth in a porcine model of peripheral vascular disease	28
Engineering miniature CRISPR-Cas Un1Cas12f1 for efficient base editing	27
m6A-induced repression of SIAH1 facilitates alternative splicing of androgen receptor variant 7 by regulating CPSF1	27
Antisense transcription from lentiviral gene targeting linked to an integrated stress response in colorectal cancer cells	27
Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing	27
Hepatocyte-derived exosomal miR-27a activates hepatic stellate cells through the inhibition of PINK1-mediated mitophagy in MAFLD	27
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice	27
Enhancing natural killer cells proliferation and cytotoxicity using imidazole-based lipid nanoparticles encapsulating interleukin-2 mRNA	27
Testis electroporation coupled with autophagy inhibitor to treat non-obstructive azoospermia	27
Unveiling the role of PUS7-mediated pseudouridylation in host protein interactions specific for the SARS-CoV-2 RNA genome	26
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina	26
siRNA goes after diseases of the bone	26
Selection of DNA aptamers that prevent the fibrillization of α-synuclein protein in cellular and mouse models	26
Aptamer-based factor IXa inhibition preserves hemostasis and prevents thrombosis in a piglet model of ECMO	26
Aberrant HSF1 signaling activation underlies metformin amelioration of myocardial infarction in mice	25
Gene network landscape of mouse splenocytes reveals integrin complex as the A151 ODN-responsive hub molecule in the immune transcriptome	25
A model system for antiviral siRNA therapeutics using exosome-based delivery	25
FUBP3: A new player in HIV-1 transcriptional activation and immune regulation	25

MicroRNA-378 contributes to osteoarthritis by regulating chondrocyte autophagy and bone marrow mesenchymal stem cell chondrogenesis	25
Pharmacoepitranscriptomic landscape revealing m6A modification could be a drug-effect biomarker for cancer treatment	25
Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy	25
Axl deficiency promotes preeclampsia and vascular malformations in mice	25
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects	25
An instructive attempt on developing aptamer-constructed PROTAC for breast cancer treatment	25
Retraction Notice to: Schizandrin A Protects Human Retinal Pigment Epithelial Cell Line ARPE-19 against HG-Induced Cell Injury by Regulation of miR-145	25
Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity	25
SINEUPs to boost translation	24
Retraction Notice to: GLP2 Promotes Directed Differentiation from Osteosarcoma Cells to Osteoblasts and Inhibits Growth of Osteosarcoma Cells	24
Intratumoral electroporation of a self-amplifying RNA expressing IL-12 induces antitumor effects in mouse models of cancer	24
Enhancing mRNA translation efficiency by introducing sequence optimized AU-rich elements in 3′ UTR via HuR anchorage	24
Opposing impacts of DNA polyplex crosslinking on delivery efficiency and vaccine responses	24
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1	24
Plug-and-play nucleic acid-mediated multimerization of biparatopic nanobodies for molecular imaging	24
Dynamic regulatory networks of T cell trajectory dissect transcriptional control of T cell state transition	24
Modulation of miR-181 influences dopaminergic neuronal degeneration in a mouse model of Parkinson’s disease	24
Promising strategies employing nucleic acids as antimicrobial drugs	24
Human opsin restoration by histone methylation using methyltransferase fusion protein SETD7-dCas9	24
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells	24
Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations	24
Retraction Notice to: TNFAIP8 Promotes Cisplatin Chemoresistance in Triple-Negative Breast Cancer by Repressing p53-Mediated miR-205-5p Expression	24
Dendritic siRNA conjugate riding albumin for targeted delivery to solid tumors	24
Manipulating the delivery and immunogenicity of DNA vaccines through the addition of CB[8] to cationic polymers	24
Recent advances in crosstalk between N6-methyladenosine (m6A) modification and circular RNAs in cancer	23
Repeated intracisternal magna delivery of antisense oligonucleotides improves behavioural outcome in the dystrophic mdx23 mouse	23
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV	23
Base editing in humanized dystrophic mice	23
Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse	23
A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases	23
Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications	23
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy	22
Delivering therapeutic RNA into the brain using extracellular vesicles	22
Antisense Oligonucleotide Targeting the Ubiquitin Ligase RFFL Potentiates CFTR Modulator Efficacy in CF Primary Bronchial Epithelial Cells	22
Retraction Notice to: MTTL3 upregulates microRNA-1246 to promote occurrence and progression of NSCLC via targeting paternally expressed gene 3	22
Tubule-specific deletion of LincRNA-p21 ameliorates lipotoxic kidney injury	22
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes	22
Blood pressure reduction through brain delivery of nanoparticles loaded with plasmid DNA encoding angiotensin receptor shRNA	22
Self-attention enabled deep learning of dihydrouridine (D) modification on mRNAs unveiled a distinct sequence signature from tRNAs	22
MiR-142a-3p: A novel ACh receptor transcriptional regulator in association with peripheral nerve injury	22
Broadly neutralizing aptamers to SARS-CoV-2: A diverse panel of modified DNA antiviral agents	22
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis-regulation in human and mouse	22
Retraction Notice to: Overexpression of microRNA-203 Suppresses Proliferation, Invasion, and Migration while Accelerating Apoptosis of CSCC Cell Line SCL-1	22
Bile acid accumulation induced by miR-122 deficiency in liver parenchyma promotes cancer cell growth in hepatocellular carcinoma	22
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties	22
Deciphering a TB-related DNA methylation biomarker and constructing a TB diagnostic classifier	22
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF	22
A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy	22
Adenovirus expressing nc886, an anti-interferon and anti-apoptotic non-coding RNA, is an improved gene delivery vector	21
Recent applications, future perspectives, and limitations of the CRISPR-Cas system	21
Inducible miR-1224 silences cerebrovascular Serpine1 and restores blood flow to the stroke-affected site of the brain	21
Retraction Notice to: Exosome miR-155 Derived from Gastric Carcinoma Promotes Angiogenesis by Targeting the c-MYB/VEGF Axis of Endothelial Cells	21
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells	21
A potential therapeutic approach for tauopathies	21
Role of long non-coding RNAs in cancer: From subcellular localization to nanoparticle-mediated targeted regulation	21
Royal jelly extracellular vesicles promote wound healing by modulating underlying cellular responses	21
Live-cell imaging of microRNA expression with post-transcriptional feedback control	21
RNA activation of CEBPA improves leukemia treatment	21
Conjugated STING agonists	21
Long noncoding RNA LUCAT1 enhances the survival and therapeutic effects of mesenchymal stromal cells post-myocardial infarction	21
Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope	21
Restoration of myogenesis in ALS-myocytes through miR-26a-5p-mediated Smad4 inhibition and its impact on motor neuron development	20
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity	20
A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication	20
A cystic fibrosis gene editing approach that is on target	20
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice	20
Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site	20
Splicing correction by peptide-conjugated morpholinos as a novel treatment for late-onset Pompe disease	20
Analytical approach for identification and mechanistic insights into mRNA-lipid adduct formation	20
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus	20
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection	20
Overexpression of lncRNA EPB41L4A-AS1 Induces Metabolic Reprogramming in Trophoblast Cells and Placenta Tissue of Miscarriage	20
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model	19
Gal-3 activates Tyro3 to ameliorate ferroptosis of hippocampal neurons after traumatic brain injury	19
Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs	19
Minicircle DNA vectors: A breakthrough in non-viral delivery of CRISPR base editors?	19
Combined cancer immunotherapy with lipid nanoparticle delivery of oligo-based cGAS-agonistic adjuvant and peptide or mRNA vaccines	19
Cell-type-specific alternative polyadenylation as a therapeutic biomarker in lung cancer progression	19
Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome	19
Unveiling the emerging functions of nuclear actin in gene function regulation	19
Novel Fas-TNFR chimeras that prevent Fas ligand-mediated kill and signal synergistically to enhance CAR T cell efficacy	19
Harnessing defective interfering particles and lipid nanoparticles for effective delivery of an anti-dengue virus RNA therapy	19
miR-429 RNA therapy as generic strategy to protect against photoreceptor loss	19
MicroRNA-451a inhibits gemcitabine-refractory biliary tract cancer progression by suppressing the MIF-mediated PI3K/AKT pathway	19
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4	19
Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform	19
Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping	18
EPRIM: An approach of identifying cancer immune-related epigenetic regulators	18
A bovine adenoviral-vector-based universal influenza vaccine confers protection against influenza A and B viruses in mice and ferrets	18
Toward learning the rules that predict siRNA efficacy	18
Antisense oligonucleotide therapy for patients with Friedreich’s ataxia carrying the c.165+5G>C splicing mutation	18
Progress and applications of single-cell RNA sequencing and spatial transcriptome technology in acute kidney injury research	18
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide	18
ACE-2 blockade and TMPRSS2 inhibition mitigate SARS-CoV-2 severity following cigarette smoke exposure in airway epithelial cells in vitro	18
Binding of G-quadruplex DNA and serum albumins by synthetic non-proteinogenic amino acids: Implications for c-Myc-related anticancer activity and drug delivery	18