Molecular Therapy

Article	Citations
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	171127347
Current Clinical Applications of In Vivo Gene Therapy with AAVs	374
Immune Responses to Viral Gene Therapy Vectors	374
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer	357
Plant Exosome-like Nanovesicles: Emerging Therapeutics and Drug Delivery Nanoplatforms	200
CAR T Cell Therapy for Solid Tumors: Bright Future or Dark Reality?	189
A Multi-Targeting, Nucleoside-Modified mRNA Influenza Virus Vaccine Provides Broad Protection in Mice	186
Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug	175
A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties	147
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9	135
xCT: A Critical Molecule That Links Cancer Metabolism to Redox Signaling	134
A Nuclear Long Non-Coding RNA LINC00618 Accelerates Ferroptosis in a Manner Dependent upon Apoptosis	132
Treatment of a Mouse Model of ALS by In Vivo Base Editing	129
M2 Macrophage-Derived Exosomes Promote Angiogenesis and Growth of Pancreatic Ductal Adenocarcinoma by Targeting E2F2	129
Emerging mechanisms and targeted therapy of ferroptosis in cancer	127
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B	124
Personalized Cancer Vaccines: Clinical Landscape, Challenges, and Opportunities	121
CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases	119
METTL1/WDR4-mediated m7G tRNA modifications and m7G codon usage promote mRNA translation and lung cancer progression	114
lncRNA SNHG11 Promotes Gastric Cancer Progression by Activating the Wnt/β-Catenin Pathway and Oncogenic Autophagy	105
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines	104
A SARS-CoV-2 targeted siRNA-nanoparticle therapy for COVID-19	99
Plant-derived exosomal microRNAs inhibit lung inflammation induced by exosomes SARS-CoV-2 Nsp12	98
Enhanced Loading of Functional miRNA Cargo via pH Gradient Modification of Extracellular Vesicles	98
circCAMSAP1 Promotes Tumor Growth in Colorectal Cancer via the miR-328-5p/E2F1 Axis	98

A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice	97
Single-Cell Transcriptome Analysis Reveals Intratumoral Heterogeneity in ccRCC, which Results in Different Clinical Outcomes	97
COVID-19 mRNA vaccines: Platforms and current developments	96
Lyophilization provides long-term stability for a lipid nanoparticle-formulated, nucleoside-modified mRNA vaccine	96
Gene therapy for ALS: A review	93
Engineered Circular RNA Sponges Act as miRNA Inhibitors to Attenuate Pressure Overload-Induced Cardiac Hypertrophy	92
Allergic Reactions and Anaphylaxis to LNP-Based COVID-19 Vaccines	89
METTL3 Promotes Tumorigenesis and Metastasis through BMI1 m6A Methylation in Oral Squamous Cell Carcinoma	88
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy	87
Oncolytic Adenovirus Armed with BiTE, Cytokine, and Checkpoint Inhibitor Enables CAR T Cells to Control the Growth of Heterogeneous Tumors	87
In vivo somatic cell base editing and prime editing	84
Tumor-derived exosomal microRNA-106b-5p activates EMT-cancer cell and M2-subtype TAM interaction to facilitate CRC metastasis	84
AAV vectors: The Rubik’s cube of human gene therapy	82
Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs	82
Exosomal miR-208b related with oxaliplatin resistance promotes Treg expansion in colorectal cancer	81
Dual-AAV delivering split prime editor system for in vivo genome editing	80
Adiponectin Stimulates Exosome Release to Enhance Mesenchymal Stem-Cell-Driven Therapy of Heart Failure in Mice	80
In Vivo Generation of CAR T Cells Selectively in Human CD4+ Lymphocytes	79
CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies	78
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II	77
Role of METTL3-Dependent N6-Methyladenosine mRNA Modification in the Promotion of Angiogenesis	74
METTL3 restrains papillary thyroid cancer progression via m6A/c-Rel/IL-8-mediated neutrophil infiltration	73
Current Status and Challenges of DNA Base Editing Tools	72
Cancer-secreted exosomal miR-1468-5p promotes tumor immune escape via the immunosuppressive reprogramming of lymphatic vessels	71
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations	71
Role of exosomal non-coding RNAs from tumor cells and tumor-associated macrophages in the tumor microenvironment	70
Synergistic and Antagonistic Drug Combinations against SARS-CoV-2	70
m6A RNA Methylation in Cardiovascular Diseases	69
Comprehensive Molecular Profiles of Functionally Effective MSC-Derived Extracellular Vesicles in Immunomodulation	67
lncRNA ITGB8-AS1 functions as a ceRNA to promote colorectal cancer growth and migration through integrin-mediated focal adhesion signaling	67
circ-EIF6 encodes EIF6-224aa to promote TNBC progression via stabilizing MYH9 and activating the Wnt/beta-catenin pathway	67
HucMSC-Derived Exosomes Mitigate the Age-Related Retardation of Fertility in Female Mice	67
A Molecular Revolution in the Treatment of Hemophilia	65
Integrative Analysis of NSCLC Identifies LINC01234 as an Oncogenic lncRNA that Interacts with HNRNPA2B1 and Regulates miR-106b Biogenesis	65
Circular RNAs: Expression, localization, and therapeutic potentials	65
Microglia secrete miR-146a-5p-containing exosomes to regulate neurogenesis in depression	64
AAV integration in human hepatocytes	64
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective	63
The evolving landscape of N6-methyladenosine modification in the tumor microenvironment	62
Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice	61
Development of CAR T Cells Expressing a Suicide Gene Plus a Chimeric Antigen Receptor Targeting Signaling Lymphocytic-Activation Molecule F7	60
Exosome-derived circTRPS1 promotes malignant phenotype and CD8+ T cell exhaustion in bladder cancer microenvironments	59
The Circular RNA circSKA3 Binds Integrin β1 to Induce Invadopodium Formation Enhancing Breast Cancer Invasion	59
YAP Circular RNA, circYap, Attenuates Cardiac Fibrosis via Binding with Tropomyosin-4 and Gamma-Actin Decreasing Actin Polymerization	58
Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency	57
Transfer RNA-Derived Small RNAs: Another Layer of Gene Regulation and Novel Targets for Disease Therapeutics	57
METTL3-mediated m6A modification of TIMP2 mRNA promotes podocyte injury in diabetic nephropathy	57
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV	56
A Distinct Transcriptional Program in Human CAR T Cells Bearing the 4-1BB Signaling Domain Revealed by scRNA-Seq	56
Engineered prime editors with PAM flexibility	55

Photodynamic therapy synergizes with PD-L1 checkpoint blockade for immunotherapy of CRC by multifunctional nanoparticles	55
Emerging Role of Ubiquitination in the Regulation of PD-1/PD-L1 in Cancer Immunotherapy	54
Microglial vesicles improve post-stroke recovery by preventing immune cell senescence and favoring oligodendrogenesis	54
First-in-Human, First-in-Child Trial of Autologous MSCs Carrying the Oncolytic Virus Icovir-5 in Patients with Advanced Tumors	53
Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas9	52
Aptamers: Cutting edge of cancer therapies	51
Dysregulated m6A modification promotes lipogenesis and development of non-alcoholic fatty liver disease and hepatocellular carcinoma	51
Olaparib Suppresses MDSC Recruitment via SDF1α/CXCR4 Axis to Improve the Anti-tumor Efficacy of CAR-T Cells on Breast Cancer in Mice	50
Allogeneic FLT3 CAR T Cells with an Off-Switch Exhibit Potent Activity against AML and Can Be Depleted to Expedite Bone Marrow Recovery	50
Adenylate Kinase 4 Modulates the Resistance of Breast Cancer Cells to Tamoxifen through an m6A-Based Epitranscriptomic Mechanism	49
IL-15 Preconditioning Augments CAR T Cell Responses to Checkpoint Blockade for Improved Treatment of Solid Tumors	49
Circular RNA circ-FoxO3 attenuates blood-brain barrier damage by inducing autophagy during ischemia/reperfusion	49
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing	49
COVID-eVax, an electroporated DNA vaccine candidate encoding the SARS-CoV-2 RBD, elicits protective responses in animal models	49
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	49
Novel Genetic and Epigenetic Biomarkers of Prognostic and Predictive Significance in Stage II/III Colorectal Cancer	49
circMRPS35 promotes malignant progression and cisplatin resistance in hepatocellular carcinoma	48
Ginseng-derived nanoparticles potentiate immune checkpoint antibody efficacy by reprogramming the cold tumor microenvironment	48
Pre-conditioning modifies the TME to enhance solid tumor CAR T cell efficacy and endogenous protective immunity	48
Inhibition of xCT suppresses the efficacy of anti-PD-1/L1 melanoma treatment through exosomal PD-L1-induced macrophage M2 polarization	48
Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction	47
FOXO3A-induced LINC00926 suppresses breast tumor growth and metastasis through inhibition of PGK1-mediated Warburg effect	47
Bulk and single-cell transcriptome profiling reveal the metabolic heterogeneity in human breast cancers	47
Clinical CAR-T Cell and Oncolytic Virotherapy for Cancer Treatment	47
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?	46
High Glucose Induces Mesangial Cell Apoptosis through miR-15b-5p and Promotes Diabetic Nephropathy by Extracellular Vesicle Delivery	46
Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	46
Exosomal miR-1246 from glioma patient body fluids drives the differentiation and activation of myeloid-derived suppressor cells	45
Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics	45
CXCL11-armed oncolytic adenoviruses enhance CAR-T cell therapeutic efficacy and reprogram tumor microenvironment in glioblastoma	45
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases	45
First-in-Human Phase I Clinical Trial of an SFV-Based RNA Replicon Cancer Vaccine against HPV-Induced Cancers	44
Circular RNA Cdyl promotes abdominal aortic aneurysm formation by inducing M1 macrophage polarization and M1-type inflammation	44
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver	44
Pros and Cons of Adenovirus-Based SARS-CoV-2 Vaccines	44
A bispecific antibody targeting GPC3 and CD47 induced enhanced antitumor efficacy against dual antigen-expressing HCC	44
Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A	43
NONO Inhibits Lymphatic Metastasis of Bladder Cancer via Alternative Splicing of SETMAR	43
Spatiotemporal PET Imaging Reveals Differences in CAR-T Tumor Retention in Triple-Negative Breast Cancer Models	43
A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I	43
Macrophages as a therapeutic target to promote diabetic wound healing	43
Circular RNA circPDE4D Protects against Osteoarthritis by Binding to miR-103a-3p and Regulating FGF18	43
The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer	43
Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening	43
Ligand-Induced Degradation of a CAR Permits Reversible Remote Control of CAR T Cell Activity In Vitro and In Vivo	42
In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model	42
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9	42
Bioinspired artificial exosomes based on lipid nanoparticles carrying let-7b-5p promote angiogenesis in vitro and in vivo	42
CD39 Produced from Human GMSCs Regulates the Balance of Osteoclasts and Osteoblasts through the Wnt/β-Catenin Pathway in Osteoporosis	42
mRNA-LNP vaccines tuned for systemic immunization induce strong antitumor immunity by engaging splenic immune cells	42
Imbalance of TGF-β1/BMP-7 pathways induced by M2-polarized macrophages promotes hepatocellular carcinoma aggressiveness	41
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	41
Non-invasive Reporter Gene Imaging of Cell Therapies, including T Cells and Stem Cells	41
Circular Noncoding RNA NR3C1 Acts as a miR-382-5p Sponge to Protect RPE Functions via Regulating PTEN/AKT/mTOR Signaling Pathway	41
Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells	40
circEHBP1 promotes lymphangiogenesis and lymphatic metastasis of bladder cancer via miR-130a-3p/TGFβR1/VEGF-D signaling	40
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	40
Immune Modulation by Telomerase-Specific Oncolytic Adenovirus Synergistically Enhances Antitumor Efficacy with Anti-PD1 Antibody	40
lnc-MAP3K13-7:1 Inhibits Ovarian GC Proliferation in PCOS via DNMT1 Downregulation-Mediated CDKN1A Promoter Hypomethylation	40
Leukemic extracellular vesicles induce chimeric antigen receptor T cell dysfunction in chronic lymphocytic leukemia	40
The roles of PD-1/PD-L1 in the prognosis and immunotherapy of prostate cancer	39
miR-204-3p/Nox4 Mediates Memory Deficits in a Mouse Model of Alzheimer’s Disease	39
mRNA-encoded, constitutively active STINGV155M is a potent genetic adjuvant of antigen-specific CD8+ T cell response	39
Precise Targeting of miRNA Sites Restores CFTR Activity in CF Bronchial Epithelial Cells	39
mRNALocater: Enhance the prediction accuracy of eukaryotic mRNA subcellular localization by using model fusion strategy	39
Self-amplifying loop of NF-κB and periostin initiated by PIEZO1 accelerates mechano-induced senescence of nucleus pulposus cells and intervertebral disc degeneration	39
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	39
Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa	38
Single-Cell Transcriptomic Analysis Reveals BCMA CAR-T Cell Dynamics in a Patient with Refractory Primary Plasma Cell Leukemia	38
Debulking SARS-CoV-2 in saliva using angiotensin converting enzyme 2 in chewing gum to decrease oral virus transmission and infection	38
Exosomes: A Potential Therapeutic Tool Targeting Communications between Tumor Cells and Macrophages	38
Upregulating Lin28a Promotes Axon Regeneration in Adult Mice with Optic Nerve and Spinal Cord Injury	38
Innate Inhibiting Proteins Enhance Expression and Immunogenicity of Self-Amplifying RNA	38
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	38
AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo	37
TRERNA1 upregulation mediated by HBx promotes sorafenib resistance and cell proliferation in HCC via targeting NRAS by sponging miR-22-3p	37
TGF-β1-Licensed Murine MSCs Show Superior Therapeutic Efficacy in Modulating Corneal Allograft Immune Rejection In Vivo	37
Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles	37
A truncated reverse transcriptase enhances prime editing by split AAV vectors	36
The CD39+ HBV surface protein-targeted CAR-T and personalized tumor-reactive CD8+ T cells exhibit potent anti-HCC activity	36

DPP4/CD32b/NF-κB Circuit: A Novel Druggable Target for Inhibiting CRP-Driven Diabetic Nephropathy	36
Immunogenicity of a new gorilla adenovirus vaccine candidate for COVID-19	36
Aerosol Delivery of Synthetic mRNA to Vaginal Mucosa Leads to Durable Expression of Broadly Neutralizing Antibodies against HIV	36
FOSL1 promotes metastasis of head and neck squamous cell carcinoma through super-enhancer-driven transcription program	36
Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update	35
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	35
Microglial lnc-U90926 facilitates neutrophil infiltration in ischemic stroke via MDH2/CXCL2 axis	35
Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial	35
siRNA Drugs: Here to Stay	35
Long noncoding RNA LINC01234 promotes hepatocellular carcinoma progression through orchestrating aspartate metabolic reprogramming	34
Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety	34
Circular RNA circSnx5 Controls Immunogenicity of Dendritic Cells through the miR-544/SOCS1 Axis and PU.1 Activity Regulation	34
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	34
T Cells Expressing NKG2D CAR with a DAP12 Signaling Domain Stimulate Lower Cytokine Production While Effective in Tumor Eradication	33
The emerging regulatory roles of long non-coding RNAs implicated in cancer metabolism	33
MicroRNA-10 negatively regulates inflammation in diabetic kidney via targeting activation of the NLRP3 inflammasome	33
Extracellular vesicles: The next generation in gene therapy delivery	33
Cell-derived nanovesicle-mediated drug delivery to the brain: Principles and strategies for vesicle engineering	33
ADAR2 increases in exercised heart and protects against myocardial infarction and doxorubicin-induced cardiotoxicity	32
miR-486 attenuates cardiac ischemia/reperfusion injury and mediates the beneficial effect of exercise for myocardial protection	32
sEVs from tonsil-derived mesenchymal stromal cells alleviate activation of hepatic stellate cells and liver fibrosis through miR-486-5p	32
lncRNA ZNF649-AS1 Induces Trastuzumab Resistance by Promoting ATG5 Expression and Autophagy	32
A self-amplifying mRNA SARS-CoV-2 vaccine candidate induces safe and robust protective immunity in preclinical models	32
Targeting CD33 in Chemoresistant AML Patient-Derived Xenografts by CAR-CIK Cells Modified with an Improved SB Transposon System	32
CircRNA Samd4 induces cardiac repair after myocardial infarction by blocking mitochondria-derived ROS output	32
Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice	32
Novel Long Non-coding RNA lncAMPC Promotes Metastasis and Immunosuppression in Prostate Cancer by Stimulating LIF/LIFR Expression	32
COVID-19-Induced ARDS Is Associated with Decreased Frequency of Activated Memory/Effector T Cells Expressing CD11a++	31
CRISPR technologies for the treatment of Duchenne muscular dystrophy	31
CRISPR/Cas9-Mediated miR-29b Editing as a Treatment of Different Types of Muscle Atrophy in Mice	31
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil	31
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function	31
m6A modification regulates lung fibroblast-to-myofibroblast transition through modulating KCNH6 mRNA translation	31
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	31
Hierarchical graph attention network for miRNA-disease association prediction	30
Mesenchymal stromal cell delivery of oncolytic immunotherapy improves CAR-T cell antitumor activity	30
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice	30
Locally secreted BiTEs complement CAR T cells by enhancing killing of antigen heterogeneous solid tumors	30
Apoptotic vesicles activate autophagy in recipient cells to induce angiogenesis and dental pulp regeneration	30
Beyond cancer cells: Targeting the tumor microenvironment with gene therapy and armed oncolytic virus	30
Dual Targeting of Mesothelin and CD19 with Chimeric Antigen Receptor-Modified T Cells in Patients with Metastatic Pancreatic Cancer	30
Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics	29
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system	29
Telomerase therapy attenuates cardiotoxic effects of doxorubicin	29
Safety, Tumor Reduction, and Clinical Impact of Zika Virus Injection in Dogs with Advanced-Stage Brain Tumors	29
HIV-Resistant and HIV-Specific CAR-Modified CD4+ T Cells Mitigate HIV Disease Progression and Confer CD4+ T Cell Help In Vivo	29
Inhibition of Tau seeding by targeting Tau nucleation core within neurons with a single domain antibody fragment	29
Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy	29
TGFB1/INHBA Homodimer/Nodal-SMAD2/3 Signaling Network: A Pivotal Molecular Target in PDAC Treatment	29
Transient Inhibition of PI3Kδ Enhances the Therapeutic Effect of Intravenous Delivery of Oncolytic Vaccinia Virus	28
Gsx1 promotes locomotor functional recovery after spinal cord injury	28
Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll	28
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease	28
An antisense circular RNA circSCRIB enhances cancer progression by suppressing parental gene splicing and translation	28
Gentamicin Induces Laminin 332 and Improves Wound Healing in Junctional Epidermolysis Bullosa Patients with Nonsense Mutations	28
A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease	28
LAG-3xPD-L1 bispecific antibody potentiates antitumor responses of T cells through dendritic cell activation	27
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice	27
Cardiac Nestin+ Mesenchymal Stromal Cells Enhance Healing of Ischemic Heart through Periostin-Mediated M2 Macrophage Polarization	27
Safety and immunogenicity of ChAd63-KH vaccine in post-kala-azar dermal leishmaniasis patients in Sudan	27
RBP EIF2S2 Promotes Tumorigenesis and Progression by Regulating MYC-Mediated Inhibition via FHIT-Related Enhancers	27
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells	27
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease	27
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	27
lncRNA lnc-POP1-1 upregulated by VN1R5 promotes cisplatin resistance in head and neck squamous cell carcinoma through interaction with MCM5	26
CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia	26
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease	26
Self-assembled miRNA-switch nanoparticles target denuded regions and prevent restenosis	26
Targeting SOX2 Protein with Peptide Aptamers for Therapeutic Gains against Esophageal Squamous Cell Carcinoma	26
FTO promotes multiple myeloma progression by posttranscriptional activation of HSF1 in an m6A-YTHDF2-dependent manner	26
Reactive Myelopoiesis Triggered by Lymphodepleting Chemotherapy Limits the Efficacy of Adoptive T Cell Therapy	26
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	26
Precision medicine: In vivo CAR therapy as a showcase for receptor-targeted vector platforms	26
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy	25
EVs Containing Host Restriction Factor IFITM3 Inhibited ZIKV Infection of Fetuses in Pregnant Mice through Trans-placenta Delivery	25
CAR- and TRuC-redirected regulatory T cells differ in capacity to control adaptive immunity to FVIII	25
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway	25
Advances in gene therapy hold promise for treating hereditary hearing loss	25
Rational Selection of CRISPR-Cas9 Guide RNAs for Homology-Directed Genome Editing	25
Prodrugs and prodrug-activated systems in gene therapy	25
An Optimized Full-Length FLT3/CD3 Bispecific Antibody Demonstrates Potent Anti-leukemia Activity and Reversible Hematological Toxicity	25
Inhibition of lncRNA MAAT Controls Multiple Types of Muscle Atrophy by cis- and trans-Regulatory Actions	25
NMNAT2 is downregulated in glaucomatous RGCs, and RGC-specific gene therapy rescues neurodegeneration and visual function	25
Impacts and mechanisms of alternative mRNA splicing in cancer metabolism, immune response, and therapeutics	25
IL-4 polarized human macrophage exosomes control cardiometabolic inflammation and diabetes in obesity	24
CRISPR-Cas9-Edited Tacrolimus-Resistant Antiviral T Cells for Advanced Adoptive Immunotherapy in Transplant Recipients	24
Glycine Enhances Satellite Cell Proliferation, Cell Transplantation, and Oligonucleotide Efficacy in Dystrophic Muscle	24
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	24
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning	24
Long Noncoding RNA NHEG1 Drives β-Catenin Transactivation and Neuroblastoma Progression through Interacting with DDX5	24
Activation of NF-κB-Inducing Kinase in Islet β Cells Causes β Cell Failure and Diabetes	24
Anti-tau scFvs Targeted to the Cytoplasm or Secretory Pathway Variably Modify Pathology and Neurodegenerative Phenotypes	24
Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I	24
Targeting of pancreatic cancer cells and stromal cells using engineered oncolytic Salmonella typhimurium	24
The rational development of CD5-targeting biepitopic CARs with fully human heavy-chain-only antigen recognition domains	24