Molecular Therapy-Nucleic Acids

Article	Citations
Effective intravitreal gene delivery to retinal pigment epithelium with hyaluronic acid nanospheres	222
Novel eRF3a degrader enhances gentamicin-induced premature termination codon readthrough in epidermolysis bullosa	122
Forced enhancer-promoter rewiring to alter gene expression in animal models	116
Antisense oligonucleotide-mediated exon 27 skipping restores dysferlin function in dysferlinopathy patient-derived muscle cells	108
AAV-vectored base editor trans-splicing delivers dystrophin repair	106
LinQURE: A novel AAV gene silencing platform that supports multi-transcript targeting for complex disorders	91
An oversized AAV8 vector to deliver CPS1	89
Insights into the regulatory role of epigenetics in moyamoya disease: Current advances and future prospectives	87
SPRINGing forward: Advancing RNA editing efficiency and precision with engineered ADAR2	86
Cas-CLOVER is a novel high-fidelity nuclease for safe and robust generation of TSCM-enriched allogeneic CAR-T cells	84
Retraction Notice to: Hypoxic tumor-derived exosomal circular RNA SETDB1 promotes invasive growth and EMT via the miR-7/Sp1 axis in lung adenocarcinoma	76
Unlocking RNA mysteries: Predicting subcellular localizations with AI	75
A novel gene therapy platform for the treatment of type 2 diabetes and obesity	74
Single-cell transcriptome profiling implicates the psychological stress-induced disruption of spermatogenesis	73
Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells	72
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants	72
Implications of circular transcripts in DM1 pathomechanism	66
A potent GalNAc-siRNA drug, RBD1016, leads to sustained HBsAg reduction and seroconversion in mouse models of HBV infection	65
Gene-repressing epigenetic reader EED unexpectedly enhances cyclinD1 gene activation	64
Combining nonsense mutation suppression therapy with nonsense-mediated decay inhibition in neurofibromatosis type 1	61
MicroRNA-466 and microRNA-200 increase endothelial permeability in hyperglycemia by targeting Claudin-5	60
Ferroptosis as a therapeutic target in glioblastoma: Mechanisms and emerging strategies	59
Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration	59
Alleviation of ischemia-reperfusion induced renal injury by chemically modified SOD2 mRNA delivered via lipid nanoparticles	59
Inefficacy of anti-VEGF therapy reflected in VEGF-mediated photoreceptor degeneration	59

Enhanced molecular dynamic simulation studies unravel long-range effects caused by sequence variations and partner binding in RNA aptamers	57
Implications of miR-148a-3p/p35/PTEN signaling in tau hyperphosphorylation and autoregulatory feedforward of Akt/CREB in Alzheimer’s disease	56
Efficient transfected liposomes co-loaded with pNrf2 and pirfenidone improves safe delivery for enhanced pulmonary fibrosis reversion	56
All roads lead to cure: Diversity of oligonucleotides in DM1 therapy	54
Single-swap editing for the correction of common Duchenne muscular dystrophy mutations	52
Endothelial derived, secreted long non-coding RNAs Gadlor1 and Gadlor2 aggravate cardiac remodeling	52
MASQ: A multiplex qPCR platform for quantitative alternative splicing analysis in clinical specimens	52
Progress and prospect of minicircle as a minimized non-viral DNA vector in gene therapy and regenerative medicine	51
Comparative immunogenic and structural characterization of virus-like particle and inactivated whole-virion vaccines against enterovirus D68	51
Mitochondrial double-stranded RNAs as a pivotal mediator in the pathogenesis of Sjӧgren’s syndrome	50
cncFinder: A graph-attention-network-based interpretable learning model to identify bifunctional long non-coding RNAs	50
Seeking stability for gene addition in inborn errors of metabolism	49
Modulation of miR-29 influences myocardial compliance likely through coordinated regulation of calcium handling and extracellular matrix	49
Using RNA-targeting CRISPR-Cas13 and engineered U1 systems to target ABCA4 splice variants in Stargardt disease	49
DUX4 reduction and muscle function improvement by subcutaneous delivery of gapmer antisense oligonucleotides	49
A bimolecular modification strategy for developing long-lasting bone anabolic aptamer	49
Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models	48
Deficiency of miR-409-3p improves myocardial neovascularization and function through modulation of DNAJB9/p38 MAPK signaling	47
Topical application of a CCL22-binding aptamer suppresses contact allergy	47
Right on target: The next class of efficient, safe, and specific RNAi triggers	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
Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD	46
Double-stranded RNA induction asa potential dynamic biomarkerfor DNA-demethylating agents	45
tRF-1001: A potential therapeutic target for ocular neovascular diseases	43
A genome-wide CRISPR screen unveils the endosomal maturation protein WDR91 as a promoter of productive ASO activity in melanoma	43
Extracellular viral microRNAs as biomarkers of virus infection in human cells	42
Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency	41
Engineering B cells with customized therapeutic responses using a synthetic circuit	41
Recent advances in CRISPR-Cas9-based genome insertion technologies	40
Joint masking and self-supervised strategies for inferring small molecule-miRNA associations	40
Effect of degeneration stage on non-viral tissue transfection of rd10 retina ex vivo	39
DNA-PKcs inhibition improves sequential gene insertion of the full-length CFTR cDNA in airway stem cells	39
Targeting triple-negative breast cancer cells with a β1-integrin binding aptamer	38
CleanCap M6 inhibits decapping of exogenously delivered IVT mRNA	38
Nucleic acid delivery for pathology treatment: RNA tissue delivery	38
Rapid molecular imaging of active thrombi in vivo using aptamer-antidote probes	38
Antitumor effects of chemically modified miR-143 lipoplexes in a mouse model of pelvic colorectal cancer via myristoylated alanine-rich C kinase substrate downregulation	38
RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges	38
Unlocking the therapeutic potential of locked nucleic acids through lipid nanoparticle delivery	38
A conditional RNA Pol II mono-promoter drives HIV-inducible, CRISPR-mediated cyclin T1 suppression and HIV inhibition	38
Structural variants and modifications of hammerhead ribozymes targeting influenza A virus conserved structural motifs	37
miR-204 suppresses cancer stemness and enhances osimertinib sensitivity in non-small cell lung cancer by targeting CD44	37
Change of intracellular calcium level causes acute neurotoxicity by antisense oligonucleotides via CSF route	37
Discovery of peptides for ligand-mediated delivery of mRNA lipid nanoparticles to cystic fibrosis lung epithelia	36
Off-target effects in CRISPR-Cas genome editing for human therapeutics: Progress and challenges	36
Improved cross-protection and immunity against influenza A virus in mice using a novel mRNA vaccine with optimized design of RNA sequence	36
Precise detection of CRISPR-Cas9 editing in hair cells in the treatment of autosomal dominant hearing loss	36
Redefining NSP12 activity in SARS-CoV-2 and its regulation by NSP8 and NSP7	35
Development of a novel PROTAC using the nucleic acid aptamer as a targeting ligand for tumor selective degradation of nucleolin	35
Extracellular vesicle microRNA and protein cargo profiling in three clinical-grade stem cell products reveals key functional pathways	34

Molecular treatment options for patients carrying KIAA0586/TALPID3 variants	34
miR-6077 promotes cisplatin/pemetrexed resistance in lung adenocarcinoma via CDKN1A/cell cycle arrest and KEAP1/ferroptosis pathways	34
Multi-omics integrative analysis reveals novel genetic loci and candidate genes for ischemic stroke	34
Hepatic-stellate-cell-targeted delivery of PU.1 decoy ODN by Apt-Tan attenuates liver fibrosis in mice	33
miR-342-5p downstream to Notch enhances arterialization of endothelial cells in response to shear stress by repressing MYC	33
Targeting microRNA-145-mediated progressive phenotypes of early bladder cancer in a molecularly defined in vivo model	33
Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis	33
Combining independent protein and cellular SELEX with bioinformatic analysis may allow high affinity aptamer hit discovery	33
A proximity-labeling-based approach to directly detect mRNA delivery to specific subcellular locations	33
DNA electroporation in a vacuum: A “shocking” innovation for vaccines	33
Block or degrade? Balancing on- and off-target effects of antisense strategies against transcripts with expanded triplet repeats in DM1	33
Targeting oncogenic KRAS in non-small cell lung cancer with EGFR aptamer-conjugated multifunctional RNA nanoparticles	32
Palmitoyl transferases act as potential regulators of tumor-infiltrating immune cells and glioma progression	32
Nanoparticle-formulated mRNA encoding engineered multivalent SIRPα-Fc fusion proteins shows robust anti-cancer activity in preclinical models	32
Multispecies-targeting siRNAs for the modulation of JAK1 in the skin	32
TREX2 enables efficient genome disruption mediated by paired CRISPR-Cas9 nickases that generate 3′-overhanging ends	32
Lung transcriptome of nonhuman primates exposed to total- and partial-body irradiation	31
The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders	31
Dynamic regulation of NeuroD1 expression level by a novel viral construct during astrocyte-to-neuron reprogramming	31
Targeting ZC3H11A elicits immunogenic cancer cell death through augmentation of antigen presentation and interferon response	31
Expanding DdCBE-mediated targeting scope to aC motif preference in rat	31
PPM1H is down-regulated by ATF6 and dephosphorylates p-RPS6KB1 to inhibit progression of hepatocellular carcinoma	31
Retraction Notice to: Oncogenic miR-27a delivered by exosomes binds to SFRP1 and promotes angiogenesis in renal clear cell carcinoma	31
Expanding RNA editing toolkit using an IDR-based strategy	31
Gymnotic uptake of AntimiRs alter microRNA-34a levels in 2D and 3D epithelial cell culture	30
Retraction Notice to: PIWIL1/piRNA-DQ593109 Regulates the Permeability of the Blood-Tumor Barrier via the MEG3/miR-330-5p/RUNX3 Axis	30
Epigenetic regulation of MIR145 core promoter controls miR-143/145 cluster in bladder cancer progression and treatment outcome	30
TIPE2 gene knockdown in mice attenuates experimental colitis by diminishing inflammatory cell infiltration	30
Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective	30
miR-363 Alleviates Detrusor Fibrosis via the TGF-β1/Smad Signaling Pathway by Targeting Col1a2 in Rat Models of STZ-Induced T2DM	30
Enhanced hammerhead ribozyme turnover rates: Reevaluating therapeutic space for small catalytic RNAs	30
In vivo precision base editing to rescue mouse models of disease	29
Gene therapy to enhance angiogenesis in chronic wounds	29
CD39 mRNA Therapy Attenuates Localized Acute Inflammation: A Novel Anti-Inflammatory Strategy using Cationic Nanoliposomes	29
mRNA therapeutics: Transforming medicine through innovation in design, delivery, and disease treatment	29
SHMT2 is essential for mammalian preimplantation embryonic development through de novo biosynthesis of nucleotide metabolites	29
Nimbolide-based nanomedicine inhibits breast cancer stem-like cells by epigenetic reprogramming of DNMTs-SFRP1-Wnt/β-catenin signaling axis	28
Optimized allele-specific silencing of the dominant-negative COL6A1 G293R substitution causing collagen VI-related dystrophy	28
MicroRNA-300 Regulates the Ubiquitination of PTEN through the CRL4BDCAF13 E3 Ligase in Osteosarcoma Cells	28
Single-cell transcriptomics of multi-site cell therapy in osteoarthritis: Tissue-specific treatment correlations	28
Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT	28
Turning premature stop codons into therapeutic opportunities	28
MicroRNA Profiling in Paired Left and Right Eyes, Lungs, and Testes of Normal Mice	28
Retraction Notice to: LncRNA LINC00963 Promotes Tumorigenesis and Radioresistance in Breast Cancer by Sponging miR-324-3p and Inducing ACK1 Expression	27
Antisense transcription from lentiviral gene targeting linked to an integrated stress response in colorectal cancer cells	27
mRNA-encoded ACE2 decoy lipid nanoparticles for neutralizing SARS-CoV-2 variants	27
MiR-34 at the crossroads of SMA pathogenesis and therapy: Emerging biomarker and therapeutic target	27
Testis electroporation coupled with autophagy inhibitor to treat non-obstructive azoospermia	27
An important resource and analytic platform for human and mouse cardiovascular-related cis-regulatory elements	27
CRISPR-Cas9 in hiPSCs: A new era in personalized treatment for Stargardt disease	26
Polymeric nanoparticle-based mRNA vaccine is protective against influenza virus infection in ferrets	26
Base editing corrects the common Salla disease SLC17A5 c.115C>T variant	26
Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing	25
Self-assembling short immunostimulatory duplex RNAs with broad-spectrum antiviral activity	25
Engineering miniature CRISPR-Cas Un1Cas12f1 for efficient base editing	25
Delivery of therapeutic small interfering RNA: The current patent-based landscape	25
MVA-HBVac—A novel vaccine vector that allows pan-genotypic targeting of hepatitis B virus by therapeutic vaccination	25
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control	25
m6A-induced repression of SIAH1 facilitates alternative splicing of androgen receptor variant 7 by regulating CPSF1	25
Enhancing natural killer cells proliferation and cytotoxicity using imidazole-based lipid nanoparticles encapsulating interleukin-2 mRNA	25
Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies	25
Retraction Notice to: circ-SHKBP1 Regulates the Angiogenesis of U87 Glioma-Exposed Endothelial Cells through miR-544a/FOXP1 and miR-379/FOXP2 Pathways	25
Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors	24
Comparative analysis of expression, immunogenicity, and safety profiles between linear and circular RNA vaccine platforms	24
Unlocking mRNA-driven CRISPR-Cas9 gene therapy via optimizing mRNA and the delivery vectors	24
Retraction Notice to: GLP2 Promotes Directed Differentiation from Osteosarcoma Cells to Osteoblasts and Inhibits Growth of Osteosarcoma Cells	24
Local intraluminal delivery of a smooth muscle-targeted RNA ligand inhibits neointima growth in a porcine model of peripheral vascular disease	24
Lipid self-assembling nanoparticles as a novel platform for mRNA-based vaccination	24
siRNA goes after diseases of the bone	24
Non-cytolytic re-engineering of a viral vaccine vector enables durable effector-memory T cell immunity by reinforcing type I IFN induction	24
An acyclic nucleic acid-modified siRNA targeting CAG expansions for polyglutamine disease treatment	24
CRISPR-Cas9-mediated homology-directed repair for precise gene editing	24
A model system for antiviral siRNA therapeutics using exosome-based delivery	24
Rethinking CRISPR delivery for liver-targeted gene editing: The case for spatially fractionated intra-arterial approaches	24
Gene network landscape of mouse splenocytes reveals integrin complex as the A151 ODN-responsive hub molecule in the immune transcriptome	23
Rational design and applications of piperazine and cyclohexane ionizable lipids for PKU and SSADH deficiency	23
Direct delivery of Cas9 or base editor protein and guide RNA complex enables genome editing in the retina	23
Retraction Notice to: TNFAIP8 Promotes Cisplatin Chemoresistance in Triple-Negative Breast Cancer by Repressing p53-Mediated miR-205-5p Expression	23
High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells	23
Retraction Notice to: Schizandrin A Protects Human Retinal Pigment Epithelial Cell Line ARPE-19 against HG-Induced Cell Injury by Regulation of miR-145	23

Restoration of brain dystrophin using tricyclo-DNA ASOs restores neurobehavioral deficits in DMD mice	22
Plug-and-play nucleic acid-mediated multimerization of biparatopic nanobodies for molecular imaging	22
Intratumoral electroporation of a self-amplifying RNA expressing IL-12 induces antitumor effects in mouse models of cancer	22
Engineering RsDddA as mitochondrial base editor with wide target compatibility and enhanced activity	22
Selection of DNA aptamers that prevent the fibrillization of α-synuclein protein in cellular and mouse models	22
Antisense oligonucleotide targeting nicotinamide N-methyltransferase exhibits antitumor effects	22
Pharmacoepitranscriptomic landscape revealing m6A modification could be a drug-effect biomarker for cancer treatment	22
Axl deficiency promotes preeclampsia and vascular malformations in mice	22
Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations	22
Modulation of miR-181 influences dopaminergic neuronal degeneration in a mouse model of Parkinson’s disease	22
Human opsin restoration by histone methylation using methyltransferase fusion protein SETD7-dCas9	21
Directed evolution of novel AAV capsids for enhanced delivery to mouse and human Schwann cells	21
Unveiling the role of PUS7-mediated pseudouridylation in host protein interactions specific for the SARS-CoV-2 RNA genome	21
Multi-layer molecular profiling defines an immune-active colorectal cancer subtype with therapeutic relevance	21
Dendritic siRNA conjugate riding albumin for targeted delivery to solid tumors	21
FUBP3: A new player in HIV-1 transcriptional activation and immune regulation	21
An instructive attempt on developing aptamer-constructed PROTAC for breast cancer treatment	21
A pipeline for identifying guide RNA sequences that promote RNA editing of nonsense mutations that cause inherited retinal diseases	21
Using muscle homing peptide CyPep10 to deliver phosphorodiamidate morpholino oligomers in the mdx mouse	21
Aberrant HSF1 signaling activation underlies metformin amelioration of myocardial infarction in mice	21
Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV	21
MicroRNA-378 contributes to osteoarthritis by regulating chondrocyte autophagy and bone marrow mesenchymal stem cell chondrogenesis	20
Promising strategies employing nucleic acids as antimicrobial drugs	20
SINEUPs to boost translation	20
Cis-Cardio: A comprehensive analysis platform for cardiovascular-relavant cis-regulation in human and mouse	20
Enhancing mRNA translation efficiency by introducing sequence optimized AU-rich elements in 3′ UTR via HuR anchorage	20
Manipulating the delivery and immunogenicity of DNA vaccines through the addition of CB[8] to cationic polymers	20
Lipid nanoparticle-encapsulated microRNA-192: An anti-inflammatory adjuvant that enhances vaccine efficacy in aged mice	20
Biodistribution of mRNA vaccines in rats: Enrichment in injection site and lymph tissues and rapid clearance without tissue persistence	20
Behavioral improvement in dystrophic mdx23 mouse following repeated antisense oligonucleotides injections	20
Adenovirus expressing nc886, an anti-interferon and anti-apoptotic non-coding RNA, is an improved gene delivery vector	20
Opposing impacts of DNA polyplex crosslinking on delivery efficiency and vaccine responses	20
Advancements and challenges in mRNA and ribonucleoprotein-based therapies: From delivery systems to clinical applications	20
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1	20
Base editing in humanized dystrophic mice	20
A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy	20
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
Bile acid accumulation induced by miR-122 deficiency in liver parenchyma promotes cancer cell growth in hepatocellular carcinoma	19
Overexpression of lncRNA EPB41L4A-AS1 Induces Metabolic Reprogramming in Trophoblast Cells and Placenta Tissue of Miscarriage	19
Long noncoding RNA LUCAT1 enhances the survival and therapeutic effects of mesenchymal stromal cells post-myocardial infarction	19
A potential therapeutic approach for tauopathies	19
Royal jelly extracellular vesicles promote wound healing by modulating underlying cellular responses	19
mRNA vaccine expressing enterovirus D68 virus-like particles induces potent neutralizing antibodies and protects against infection	19
A novel multitargeted self-assembling peptide-siRNA complex for simultaneous inhibition of SARS-CoV-2-host cell interaction and replication	19
Broadly neutralizing aptamers to SARS-CoV-2: A diverse panel of modified DNA antiviral agents	19
Retraction Notice to: Overexpression of microRNA-203 Suppresses Proliferation, Invasion, and Migration while Accelerating Apoptosis of CSCC Cell Line SCL-1	19
Replication not required: mRNA vaccines take on non-enveloped viruses	19
Secondary follicles enable efficient germline mtDNA base editing at hard-to-edit site	19
Retraction Notice to: Exosome miR-155 Derived from Gastric Carcinoma Promotes Angiogenesis by Targeting the c-MYB/VEGF Axis of Endothelial Cells	19
Exon skipping induces uniform dystrophin rescue with dose-dependent restoration of serum miRNA biomarkers and muscle biophysical properties	19
Analytical approach for identification and mechanistic insights into mRNA-lipid adduct formation	19
Oversized liposomes boost macrophage-targeted RNA delivery to regulate macrophage polarity	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
Role of long non-coding RNAs in cancer: From subcellular localization to nanoparticle-mediated targeted regulation	18
Inducible miR-1224 silences cerebrovascular Serpine1 and restores blood flow to the stroke-affected site of the brain	18
Transient and tunable CRISPRa regulation of APOBEC/AID genes for targeting hepatitis B virus	18
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF	18
Conjugated STING agonists	18
Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice	18
Cell-type-specific alternative polyadenylation as a therapeutic biomarker in lung cancer progression	18
RNA activation of CEBPA improves leukemia treatment	18
Restoration of myogenesis in ALS-myocytes through miR-26a-5p-mediated Smad4 inhibition and its impact on motor neuron development	18
Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection	18
The potential and challenges of circular RNA in the development of vaccines and drugs for emerging infectious diseases	18
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells	18
A cystic fibrosis gene editing approach that is on target	18
Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes	18
Self-attention enabled deep learning of dihydrouridine (D) modification on mRNAs unveiled a distinct sequence signature from tRNAs	18
Delivering therapeutic RNA into the brain using extracellular vesicles	18
Blood pressure reduction through brain delivery of nanoparticles loaded with plasmid DNA encoding angiotensin receptor shRNA	18
N1MΨU-modified mRNA vaccines break the mold in fish by enhancing innate immune activation	18
Recent applications, future perspectives, and limitations of the CRISPR-Cas system	18
Unveiling the emerging functions of nuclear actin in gene function regulation	18
Translatomics to explore dynamic differences in immunocytes in the tumor microenvironment	17
HIF-1α or HOTTIP/CTCF Promotes Head and Neck Squamous Cell Carcinoma Progression and Drug Resistance by Targeting HOXA9	17
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model	17
EPRIM: An approach of identifying cancer immune-related epigenetic regulators	17
Exosome-like nanovesicles from acerola for CRISPR-Cas9 ribonucleoprotein delivery to the central nervous system	17
Modulating G-quadruplexes for therapeutic intervention: Structural diversity, stability, and emerging nucleic-acid-based strategies	17
Targeted allele-specific FGFR2 knockdown via human recombinant ferritin nanoparticles for personalized treatment of Crouzon syndrome	17
Gal-3 activates Tyro3 to ameliorate ferroptosis of hippocampal neurons after traumatic brain injury	17
Retraction Notice to: MicroRNA-140 Inhibits the Epithelial-Mesenchymal Transition and Metastasis in Colorectal Cancer	17
MicroRNA-451a inhibits gemcitabine-refractory biliary tract cancer progression by suppressing the MIF-mediated PI3K/AKT pathway	17
ACE-2 blockade and TMPRSS2 inhibition mitigate SARS-CoV-2 severity following cigarette smoke exposure in airway epithelial cells in vitro	17
Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4	17
Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping	17
Restoration of functional PAX6 in aniridia patient iPSC-derived ocular tissue models using repurposed nonsense suppression drugs	17
Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide	17
miR-429 RNA therapy as generic strategy to protect against photoreceptor loss	17
Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform	17
Toward learning the rules that predict siRNA efficacy	17
Cell-penetrating antibody enhances nuclear delivery of triplex-forming oligonucleotides targeting HER2-positive cancers	17
Novel Fas-TNFR chimeras that prevent Fas ligand-mediated kill and signal synergistically to enhance CAR T cell efficacy	17