Molecular Therapy-Nucleic Acids

Article	Citations
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres	237
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa	146
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells	135
AAV-vectored base editor trans-splicing delivers dystrophin repair	119
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders	113
An oversized AAV8 vector to deliver CPS1	94
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives	89
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR2	85
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells	82
Unlocking RNA mysteries: Predicting subcellular localizations with AI	78
A novel gene therapy platform for the treatment of type 2 diabetes and obesity	78
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis	74
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells	73
Implications of circular transcripts in DM1 pathomechanism	70
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation	68
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1	68
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration	65
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease	64
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling	63
Seeking stability for gene addition in inborn errors of metabolism	61
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease	61
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection	60
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine	59
Forced enhancer-promoter rewiring to alter gene expression in animal models	59
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy	57

Comparative immunogenic and structural analysis of virus-like particle and inactivated whole-virion vaccines against enterovirus D68	56
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion	56
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix	56
Intranasal Tat-modified PEG-PCL nanomicelles delivering anti-RelA siRNA attenuate ischemia-reperfusion injury in transient MCAO mice	56
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies	54
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration	51
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations	51
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants	51
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer	50
Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers	50
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides	47
cncFinder: A graph-attention-network-based interpretable learning model to identify bifunctional long non-coding RNAs	47
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-5	46
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome	45
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles	45
Simultaneous Quantitative Detection of Multiple Low-Frequency Variants by High-Dynamic-Range Capillary Electrophoresis	44
MASQ: A multiplex qPCR platform for quantitative alternative splicing analysis in clinical specimens	42
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling	41
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models	41
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD	41
Extracellular viral microRNAs as biomarkers of virus infection in human cells	40
Effect of degeneration stage on non-viral tissue transfection of rd10 retina ex vivo	40
tRF-1001: A potential therapeutic target for ocular neovascular diseases	40
Engineering B cells with customized therapeutic responses using a synthetic circuit	40
Antitumor effects of chemically modified miR-143 lipoplexes in a mouse model of pelvic colorectal cancer via myristoylated alanine-rich C kinase substrate downregulation	39
Nucleic acid delivery for pathology treatment: RNA tissue delivery	39
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition	39
Rapid molecular imaging of active thrombi in vivo using aptamer-antidote probes	39
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route	38
Topical application of a CCL22-binding aptamer suppresses contact allergy	38
Redefining NSP12 activity in SARS-CoV-2 and its regulation by NSP8 and NSP7	38
PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent chronic myeloid leukemia by suppressing leukemia stem cell regeneration	38
Double-stranded RNA induction asa potential dynamic biomarkerfor DNA-demethylating agents	37
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells	37
Structural variants and modifications of hammerhead ribozymes targeting influenza A virus conserved structural motifs	37
A genome-wide CRISPR screen unveils the endosomal maturation protein WDR91 as a promoter of productive ASO activity in melanoma	36
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia	36
Targeting triple-negative breast cancer cells with a β1-integrin binding aptamer	35
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery	35
Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss	34
Development of a novel PROTAC using the nucleic acid aptamer as a targeting ligand for tumor selective degradation of nucleolin	34
Improved cross-protection and immunity against influenza A virus in mice using a novel mRNA vaccine with optimized design of RNA sequence	34
RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges	34
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations	34
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges	33
Recent advances in CRISPR-Cas9-based genome insertion technologies	33
CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA	33
Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis	33
miR-204 suppresses cancer stemness and enhances osimertinib sensitivity in non-small cell lung cancer by targeting CD44	33
Multi-omics integrative analysis reveals novel genetic loci and candidate genes for ischemic stroke	32

miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC	32
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles	32
miR-363 Alleviates Detrusor Fibrosis via the TGF-β1/Smad Signaling Pathway by Targeting Col1a2 in Rat Models of STZ-Induced T2DM	32
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders	31
Lung transcriptome of nonhuman primates exposed to total- and partial-body irradiation	31
DNA electroporation in a vacuum: A “shocking” innovation for vaccines	31
Molecular treatment options for patients carrying KIAA0586/TALPID3 variants	31
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin	31
A proximity-labeling-based approach to directly detect mRNA delivery to specific subcellular locations	31
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response	31
Targeting microRNA-145-mediated progressive phenotypes of early bladder cancer in a molecularly defined in vivo model	31
MicroRNA-300 Regulates the Ubiquitination of PTEN through the CRL4BDCAF13 E3 Ligase in Osteosarcoma Cells	30
Turning premature stop codons into therapeutic opportunities	30
Expanding RNA editing toolkit using an IDR-based strategy	30
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective	30
Hepatic-stellate-cell-targeted delivery of PU.1 decoy ODN by Apt-Tan attenuates liver fibrosis in mice	30
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture	30
Retraction Notice to: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma	30
Nimbolide-based nanomedicine inhibits breast cancer stem-like cells by epigenetic reprogramming of DNMTs-SFRP1-Wnt/β-catenin signaling axis	30
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1	30
Dynamic regulation of NeuroD1 expression level by a novel viral construct during astrocyte-to-neuron reprogramming	30
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways	29
TREX2 enables efficient genome disruption mediated by paired CRISPR-Cas9 nickases that generate 3′-overhanging ends	29
Expanding DdCBE-mediated targeting scope to aC motif preference in rat	29
Gene therapy to enhance angiogenesis in chronic wounds	29
Epigenetic regulation of MIR145 core promoter controls miR-143/145 cluster in bladder cancer progression and treatment outcome	29
Combining independent protein and cellular SELEX with bioinformatic analysis may allow high affinity aptamer hit discovery	28
In vivo precision base editing to rescue mouse models of disease	28
Nanoparticle-formulated mRNA encoding engineered multivalent SIRPα-Fc fusion proteins shows robust anti-cancer activity in preclinical models	28
Single-cell transcriptomics of multi-site cell therapy in osteoarthritis: Tissue-specific treatment correlations	27
CD39 mRNA therapy attenuates localized acute inflammation: A novel anti-inflammatory strategy using cationic nanoliposomes	27
Retraction Notice to: PIWIL1/piRNA-DQ593109 Regulates the Permeability of the Blood-Tumor Barrier via the MEG3/miR-330-5p/RUNX3 Axis	27
Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs	27
TIPE2 gene knockdown in mice attenuates experimental colitis by diminishing inflammatory cell infiltration	27
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT	26
PPM1H is down-regulated by ATF6 and dephosphorylates p-RPS6KB1 to inhibit progression of hepatocellular carcinoma	26
SHMT2 is essential for mammalian preimplantation embryonic development through de novo biosynthesis of nucleotide metabolites	26
Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy	26
mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment	26
Retraction Notice to: LncRNA LINC00963 Promotes Tumorigenesis and Radioresistance in Breast Cancer by Sponging miR-324-3p and Inducing ACK1 Expression	25
An important resource and analytic platform for human and mouse cardiovascular-related cis-regulatory elements	25
Self-assembling short immunostimulatory duplex RNAs with broad-spectrum antiviral activity	25
mRNA-encoded ACE2 decoy lipid nanoparticles for neutralizing SARS-CoV-2 variants	25
Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets	25
Lipid self-assembling nanoparticles as a novel platform for mRNA-based vaccination	25
Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies	25
MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target	25
Delivery of therapeutic small interfering RNA: The current patent-based landscape	25
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease	25
Local intraluminal delivery of a smooth muscle-targeted RNA ligand inhibits neointima growth in a porcine model of peripheral vascular disease	25
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control	24
Testis electroporation coupled with autophagy inhibitor to treat non-obstructive azoospermia	24
Unlocking translational resilience for mRNA vaccines by viral UTRs	24
Engineering miniature CRISPR-Cas Un1Cas12f1 for efficient base editing	24
Unlocking mRNA-driven CRISPR-Cas9 gene therapy via optimizing mRNA and the delivery vectors	24
Retraction Notice to: circ-SHKBP1 Regulates the Angiogenesis of U87 Glioma-Exposed Endothelial Cells through miR-544a/FOXP1 and miR-379/FOXP2 Pathways	24
Non-cytolytic re-engineering of a viral vaccine vector enables durable effector-memory T cell immunity by reinforcing type I IFN induction	24
Comparative analysis of expression, immunogenicity, and safety profiles between linear and circular RNA vaccine platforms	24
Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing	24
Enhancing natural killer cells proliferation and cytotoxicity using imidazole-based lipid nanoparticles encapsulating interleukin-2 mRNA	23
CRISPR-Cas9-mediated homology-directed repair for precise gene editing	23
A model system for antiviral siRNA therapeutics using exosome-based delivery	23
Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency	23
Base editing corrects the common Salla disease SLC17A5 c.115C>T variant	23
siRNA goes after diseases of the bone	23
Retraction Notice to: GLP2 Promotes Directed Differentiation from Osteosarcoma Cells to Osteoblasts and Inhibits Growth of Osteosarcoma Cells	23
Rethinking CRISPR delivery for liver-targeted gene editing: The case for spatially fractionated intra-arterial approaches	23
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors	23
The long non-coding RNA, CyKILRb, acts as a competing endogenous RNA for tumor suppressive microRNAs to augment oncogenic phenotypes via induction of the PIK3R2/PI3K/AKT axis	23
MVA-HBVac—A novel vaccine vector that allows pan-genotypic targeting of hepatitis B virus by therapeutic vaccination	23
Retraction Notice to: TNFAIP8 Promotes Cisplatin Chemoresistance in Triple-Negative Breast Cancer by Repressing p53-Mediated miR-205-5p Expression	23
An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment	23
SINEUPs to boost translation	22
Selection of DNA aptamers that prevent the fibrillization of α-synuclein protein in cellular and mouse models	22
Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice	22
Gene network landscape of mouse splenocytes reveals integrin complex as the A151 ODN-responsive hub molecule in the immune transcriptome	22
Manipulating the delivery and immunogenicity of DNA vaccines through the addition of CB[8] to cationic polymers	22
Axl deficiency promotes preeclampsia and vascular malformations in mice	22
FUBP3: A new player in HIV-1 transcriptional activation and immune regulation	22
Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations	22

Human opsin restoration by histone methylation using methyltransferase fusion protein SETD7-dCas9	22
Opposing impacts of DNA polyplex crosslinking on delivery efficiency and vaccine responses	22
A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases	22
An instructive attempt on developing aptamer-constructed PROTAC for breast cancer treatment	22
Dendritic siRNA conjugate riding albumin for targeted delivery to solid tumors	22
Intratumoral electroporation of a self-amplifying RNA expressing IL-12 induces antitumor effects in mouse models of cancer	21
Unveiling the role of PUS7-mediated pseudouridylation in host protein interactions specific for the SARS-CoV-2 RNA genome	21
Directed evolution of novel AAV capsids for enhanced delivery to mouse and human Schwann cells	21
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV	21
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina	21
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects	21
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells	21
Biodistribution of mRNA vaccines in rats: Enrichment in injection site and lymph tissues and rapid clearance without tissue persistence	21
Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections	21
Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications	21
Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse	21
Lipid nanoparticle-encapsulated microRNA-192: An anti-inflammatory adjuvant that enhances vaccine efficacy in aged mice	21
Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity	21
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1	20
Replication not required: mRNA vaccines take on non-enveloped viruses	20
Plug-and-play nucleic acid-mediated multimerization of biparatopic nanobodies for molecular imaging	20
Enhancing mRNA translation efficiency by introducing sequence optimized AU-rich elements in 3′ UTR via HuR anchorage	20
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties	20
Multi-layer molecular profiling defines an immune-active colorectal cancer subtype with therapeutic relevance	20
Promising strategies employing nucleic acids as antimicrobial drugs	20
Adenovirus expressing nc886, an anti-interferon and anti-apoptotic non-coding RNA, is an improved gene delivery vector	20
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy	20
Aberrant HSF1 signaling activation underlies metformin amelioration of myocardial infarction in mice	20
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity	19
A potential therapeutic approach for tauopathies	19
Inducible miR-1224 silences cerebrovascular Serpine1 and restores blood flow to the stroke-affected site of the brain	19
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus	19
A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication	19
Retraction Notice to: MTTL3 upregulates microRNA-1246 to promote occurrence and progression of NSCLC via targeting paternally expressed gene 3	19
MiR-142a-3p: A novel ACh receptor transcriptional regulator in association with peripheral nerve injury	19
Self-attention enabled deep learning of dihydrouridine (D) modification on mRNAs unveiled a distinct sequence signature from tRNAs	19
mRNA vaccine expressing enterovirus D68 virus-like particles induces potent neutralizing antibodies and protects against infection	19
RNA activation of CEBPA improves leukemia treatment	19
A fleeting glimpse of functional benefit of the complete DMD gene in a large animal model of Duchenne muscular dystrophy	19
Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells	19
Restoration of myogenesis in ALS-myocytes through miR-26a-5p-mediated Smad4 inhibition and its impact on motor neuron development	19
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells	19
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis-regulation in human and mouse	18
miR-429 RNA therapy as generic strategy to protect against photoreceptor loss	18
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice	18
A cystic fibrosis gene editing approach that is on target	18
Retraction Notice to: MicroRNA-140 Inhibits the Epithelial-Mesenchymal Transition and Metastasis in Colorectal Cancer	18
Role of long non-coding RNAs in cancer: From subcellular localization to nanoparticle-mediated targeted regulation	18
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection	18
Blood pressure reduction through brain delivery of nanoparticles loaded with plasmid DNA encoding angiotensin receptor shRNA	18
Base editing in humanized dystrophic mice	18
The potential and challenges of circular RNA in the development of vaccines and drugs for emerging infectious diseases	18
Conjugated STING agonists	18
Unveiling the emerging functions of nuclear actin in gene function regulation	18
Harnessing defective interfering particles and lipid nanoparticles for effective delivery of an anti-dengue virus RNA therapy	18
Delivering therapeutic RNA into the brain using extracellular vesicles	18
Broadly neutralizing aptamers to SARS-CoV-2: A diverse panel of modified DNA antiviral agents	18
Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site	18
Bile acid accumulation induced by miR-122 deficiency in liver parenchyma promotes cancer cell growth in hepatocellular carcinoma	18
Royal jelly extracellular vesicles promote wound healing by modulating underlying cellular responses	18
N1MΨU-modified mRNA vaccines break the mold in fish by enhancing innate immune activation	18
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes	18
Recent applications, future perspectives, and limitations of the CRISPR-Cas system	18
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF	18
Overexpression of lncRNA EPB41L4A-AS1 Induces Metabolic Reprogramming in Trophoblast Cells and Placenta Tissue of Miscarriage	18
Analytical approach for identification and mechanistic insights into mRNA-lipid adduct formation	18
Toward learning the rules that predict siRNA efficacy	17
Minicircle DNA vectors: A breakthrough in non-viral delivery of CRISPR base editors?	17
Translatomics to explore dynamic differences in immunocytes in the tumor microenvironment	17
Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform	17
Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs	17
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4	17
Combined cancer immunotherapy with lipid nanoparticle delivery of oligo-based cGAS-agonistic adjuvant and peptide or mRNA vaccines	17
NADPH oxidase 3 inhibition preserves hearing in mice after stereotactic radiosurgery	17
Cell-penetrating antibody enhances nuclear delivery of triplex-forming oligonucleotides targeting HER2-positive cancers	17
Rescue of the endogenous FVIII expression in hemophilia A mice using CRISPR-Cas9 mRNA LNPs	17
EPRIM: An approach of identifying cancer immune-related epigenetic regulators	17
Cell-type-specific alternative polyadenylation as a therapeutic biomarker in lung cancer progression	17
cGAMP-adjuvanted multivalent influenza mRNA vaccines induce broadly protective immunity through cutaneous vaccination in mice	17
Gal-3 activates Tyro3 to ameliorate ferroptosis of hippocampal neurons after traumatic brain injury	17
Modulating G-quadruplexes for therapeutic intervention: Structural diversity, stability, and emerging nucleic-acid-based strategies	17
ACE-2 blockade and TMPRSS2 inhibition mitigate SARS-CoV-2 severity following cigarette smoke exposure in airway epithelial cells in vitro	17
Engineered miR-122 inhibitors preserve endothelial mitochondrial function and prevent vascular dysfunction in obesity-associated prediabetes	17
Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome	17
Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping	17
MicroRNA-451a inhibits gemcitabine-refractory biliary tract cancer progression by suppressing the MIF-mediated PI3K/AKT pathway	17
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model	17
Antisense oligonucleotide therapy for patients with Friedreich’s ataxia carrying the c.165+5G>C splicing mutation	17
Exosome-like nanovesicles from acerola for CRISPR-Cas9 ribonucleoprotein delivery to the central nervous system	17
Good news for the mdx mouse community: Improved dystrophin restoration after skipping mouse dystrophin exon 23	16
A shift of paradigm, emerging role of miRNAs as therapeutic targets for complex disease	16
Retraction Notice to: Inflammatory-Related P62 Triggers Malignant Transformation of Mesenchymal Stem Cells through the Cascade of CUDR-CTCF-IGFII-RAS Signaling	16
Deficiency of miRNA-149-3p shaped gut microbiota and enhanced dextran sulfate sodium-induced colitis	16
Retraction Notice to: LINC01234/MicroRNA-31-5p/MAGEA3 Axis Mediates the Proliferation and Chemoresistance of Hepatocellular Carcinoma Cells	16
Lipid nanoparticle-encapsulated, chemically modified anti-adenoviral siRNAs inhibit hepatic adenovirus infection in immunosuppressed Syrian hamsters	16