Molecular Therapy

Papers
(The TQCC of Molecular Therapy is 19. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2020-02-01 to 2024-02-01.)
ArticleCitations
Immune Responses to Viral Gene Therapy Vectors362
Current Clinical Applications of In Vivo Gene Therapy with AAVs359
AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer346
Plant Exosome-like Nanovesicles: Emerging Therapeutics and Drug Delivery Nanoplatforms192
CAR T Cell Therapy for Solid Tumors: Bright Future or Dark Reality?187
A Multi-Targeting, Nucleoside-Modified mRNA Influenza Virus Vaccine Provides Broad Protection in Mice176
Delivery of Oligonucleotides to the Liver with GalNAc: From Research to Registered Therapeutic Drug168
A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties140
RETRACTED: METTL14 Suppresses CRC Progression via Regulating N6-Methyladenosine-Dependent Primary miR-375 Processing134
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9131
A Nuclear Long Non-Coding RNA LINC00618 Accelerates Ferroptosis in a Manner Dependent upon Apoptosis130
Genetically Engineered Cell-Derived Nanoparticles for Targeted Breast Cancer Immunotherapy129
xCT: A Critical Molecule That Links Cancer Metabolism to Redox Signaling129
Treatment of a Mouse Model of ALS by In Vivo Base Editing127
M2 Macrophage-Derived Exosomes Promote Angiogenesis and Growth of Pancreatic Ductal Adenocarcinoma by Targeting E2F2122
Emerging mechanisms and targeted therapy of ferroptosis in cancer121
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B119
Personalized Cancer Vaccines: Clinical Landscape, Challenges, and Opportunities115
CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases115
METTL1/WDR4-mediated m7G tRNA modifications and m7G codon usage promote mRNA translation and lung cancer progression114
Increased Microglial Exosomal miR-124-3p Alleviates Neurodegeneration and Improves Cognitive Outcome after rmTBI112
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines104
lncRNA SNHG11 Promotes Gastric Cancer Progression by Activating the Wnt/β-Catenin Pathway and Oncogenic Autophagy101
A SARS-CoV-2 targeted siRNA-nanoparticle therapy for COVID-1997
circCAMSAP1 Promotes Tumor Growth in Colorectal Cancer via the miR-328-5p/E2F1 Axis97
Plant-derived exosomal microRNAs inhibit lung inflammation induced by exosomes SARS-CoV-2 Nsp1295
Enhanced Loading of Functional miRNA Cargo via pH Gradient Modification of Extracellular Vesicles95
Single-Cell Transcriptome Analysis Reveals Intratumoral Heterogeneity in ccRCC, which Results in Different Clinical Outcomes95
A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice93
Engineered Circular RNA Sponges Act as miRNA Inhibitors to Attenuate Pressure Overload-Induced Cardiac Hypertrophy90
Gene therapy for ALS: A review90
Lyophilization provides long-term stability for a lipid nanoparticle-formulated, nucleoside-modified mRNA vaccine89
COVID-19 mRNA vaccines: Platforms and current developments88
Allergic Reactions and Anaphylaxis to LNP-Based COVID-19 Vaccines86
Oncolytic Adenovirus Armed with BiTE, Cytokine, and Checkpoint Inhibitor Enables CAR T Cells to Control the Growth of Heterogeneous Tumors86
In vivo somatic cell base editing and prime editing83
METTL3 Promotes Tumorigenesis and Metastasis through BMI1 m6A Methylation in Oral Squamous Cell Carcinoma83
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy80
In Vivo Generation of CAR T Cells Selectively in Human CD4+ Lymphocytes77
Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones77
Tumor-derived exosomal microRNA-106b-5p activates EMT-cancer cell and M2-subtype TAM interaction to facilitate CRC metastasis77
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II76
Adiponectin Stimulates Exosome Release to Enhance Mesenchymal Stem-Cell-Driven Therapy of Heart Failure in Mice75
CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies74
Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs74
AAV vectors: The Rubik’s cube of human gene therapy73
METTL3 restrains papillary thyroid cancer progression via m6A/c-Rel/IL-8-mediated neutrophil infiltration73
GPR108 Is a Highly Conserved AAV Entry Factor73
Dual-AAV delivering split prime editor system for in vivo genome editing73
Exosomal miR-208b related with oxaliplatin resistance promotes Treg expansion in colorectal cancer72
Role of METTL3-Dependent N6-Methyladenosine mRNA Modification in the Promotion of Angiogenesis72
HLSC-Derived Extracellular Vesicles Attenuate Liver Fibrosis and Inflammation in a Murine Model of Non-alcoholic Steatohepatitis70
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations69
Current Status and Challenges of DNA Base Editing Tools69
MicroRNA 223 3p Negatively Regulates the NLRP3 Inflammasome in Acute and Chronic Liver Injury69
Cancer-secreted exosomal miR-1468-5p promotes tumor immune escape via the immunosuppressive reprogramming of lymphatic vessels68
Synergistic and Antagonistic Drug Combinations against SARS-CoV-267
m6A RNA Methylation in Cardiovascular Diseases66
HucMSC-Derived Exosomes Mitigate the Age-Related Retardation of Fertility in Female Mice66
A Molecular Revolution in the Treatment of Hemophilia65
Circular RNAs: Expression, localization, and therapeutic potentials64
Integrative Analysis of NSCLC Identifies LINC01234 as an Oncogenic lncRNA that Interacts with HNRNPA2B1 and Regulates miR-106b Biogenesis64
AAV integration in human hepatocytes63
Comprehensive Molecular Profiles of Functionally Effective MSC-Derived Extracellular Vesicles in Immunomodulation62
lncRNA ITGB8-AS1 functions as a ceRNA to promote colorectal cancer growth and migration through integrin-mediated focal adhesion signaling62
The evolving landscape of N6-methyladenosine modification in the tumor microenvironment62
circ-EIF6 encodes EIF6-224aa to promote TNBC progression via stabilizing MYH9 and activating the Wnt/beta-catenin pathway61
Role of exosomal non-coding RNAs from tumor cells and tumor-associated macrophages in the tumor microenvironment61
Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice59
The Circular RNA circSKA3 Binds Integrin β1 to Induce Invadopodium Formation Enhancing Breast Cancer Invasion59
A Safe and Reliable Technique for CNS Delivery of AAV Vectors in the Cisterna Magna58
YAP Circular RNA, circYap, Attenuates Cardiac Fibrosis via Binding with Tropomyosin-4 and Gamma-Actin Decreasing Actin Polymerization57
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective57
Development of CAR T Cells Expressing a Suicide Gene Plus a Chimeric Antigen Receptor Targeting Signaling Lymphocytic-Activation Molecule F757
Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency57
Microglia secrete miR-146a-5p-containing exosomes to regulate neurogenesis in depression56
Urinary Extracellular Vesicles Carrying Klotho Improve the Recovery of Renal Function in an Acute Tubular Injury Model56
Transfer RNA-Derived Small RNAs: Another Layer of Gene Regulation and Novel Targets for Disease Therapeutics56
Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV56
A Distinct Transcriptional Program in Human CAR T Cells Bearing the 4-1BB Signaling Domain Revealed by scRNA-Seq55
METTL3-mediated m6A modification of TIMP2 mRNA promotes podocyte injury in diabetic nephropathy54
Engineered prime editors with PAM flexibility53
Exosome-derived circTRPS1 promotes malignant phenotype and CD8+ T cell exhaustion in bladder cancer microenvironments53
Photodynamic therapy synergizes with PD-L1 checkpoint blockade for immunotherapy of CRC by multifunctional nanoparticles53
First-in-Human, First-in-Child Trial of Autologous MSCs Carrying the Oncolytic Virus Icovir-5 in Patients with Advanced Tumors52
Gene Therapy for Glaucoma by Ciliary Body Aquaporin 1 Disruption Using CRISPR-Cas952
Emerging Role of Ubiquitination in the Regulation of PD-1/PD-L1 in Cancer Immunotherapy52
Microglial vesicles improve post-stroke recovery by preventing immune cell senescence and favoring oligodendrogenesis51
The Emerging Role of Cytidine Deaminase in Human Diseases: A New Opportunity for Therapy?51
Aptamers: Cutting edge of cancer therapies51
Allogeneic FLT3 CAR T Cells with an Off-Switch Exhibit Potent Activity against AML and Can Be Depleted to Expedite Bone Marrow Recovery50
Dysregulated m6A modification promotes lipogenesis and development of non-alcoholic fatty liver disease and hepatocellular carcinoma49
Adenylate Kinase 4 Modulates the Resistance of Breast Cancer Cells to Tamoxifen through an m6A-Based Epitranscriptomic Mechanism49
Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing48
IL-15 Preconditioning Augments CAR T Cell Responses to Checkpoint Blockade for Improved Treatment of Solid Tumors48
COVID-eVax, an electroporated DNA vaccine candidate encoding the SARS-CoV-2 RBD, elicits protective responses in animal models48
Pre-conditioning modifies the TME to enhance solid tumor CAR T cell efficacy and endogenous protective immunity48
Novel Genetic and Epigenetic Biomarkers of Prognostic and Predictive Significance in Stage II/III Colorectal Cancer48
Inhibition of xCT suppresses the efficacy of anti-PD-1/L1 melanoma treatment through exosomal PD-L1-induced macrophage M2 polarization48
Olaparib Suppresses MDSC Recruitment via SDF1α/CXCR4 Axis to Improve the Anti-tumor Efficacy of CAR-T Cells on Breast Cancer in Mice48
Circular RNA circ-FoxO3 attenuates blood-brain barrier damage by inducing autophagy during ischemia/reperfusion48
Clinical CAR-T Cell and Oncolytic Virotherapy for Cancer Treatment47
Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway46
Codon Modification and PAMPs in Clinical AAV Vectors: The Tortoise or the Hare?46
circMRPS35 promotes malignant progression and cisplatin resistance in hepatocellular carcinoma46
Bulk and single-cell transcriptome profiling reveal the metabolic heterogeneity in human breast cancers45
Exosomal miR-1246 from glioma patient body fluids drives the differentiation and activation of myeloid-derived suppressor cells44
Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics44
Ginseng-derived nanoparticles potentiate immune checkpoint antibody efficacy by reprogramming the cold tumor microenvironment44
FOXO3A-induced LINC00926 suppresses breast tumor growth and metastasis through inhibition of PGK1-mediated Warburg effect44
Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR Correction44
Circular RNA circPDE4D Protects against Osteoarthritis by Binding to miR-103a-3p and Regulating FGF1843
Pre-arrayed Pan-AAV Peptide Display Libraries for Rapid Single-Round Screening43
First-in-Human Phase I Clinical Trial of an SFV-Based RNA Replicon Cancer Vaccine against HPV-Induced Cancers43
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases43
High Glucose Induces Mesangial Cell Apoptosis through miR-15b-5p and Promotes Diabetic Nephropathy by Extracellular Vesicle Delivery43
NONO Inhibits Lymphatic Metastasis of Bladder Cancer via Alternative Splicing of SETMAR43
Spatiotemporal PET Imaging Reveals Differences in CAR-T Tumor Retention in Triple-Negative Breast Cancer Models42
CD39 Produced from Human GMSCs Regulates the Balance of Osteoclasts and Osteoblasts through the Wnt/β-Catenin Pathway in Osteoporosis42
Pros and Cons of Adenovirus-Based SARS-CoV-2 Vaccines42
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK942
A Highly Efficacious PS Gene Editing System Corrects Metabolic and Neurological Complications of Mucopolysaccharidosis Type I42
A bispecific antibody targeting GPC3 and CD47 induced enhanced antitumor efficacy against dual antigen-expressing HCC42
In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model42
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer41
Bioinspired artificial exosomes based on lipid nanoparticles carrying let-7b-5p promote angiogenesis in vitro and in vivo41
Non-invasive Reporter Gene Imaging of Cell Therapies, including T Cells and Stem Cells41
Circular Noncoding RNA NR3C1 Acts as a miR-382-5p Sponge to Protect RPE Functions via Regulating PTEN/AKT/mTOR Signaling Pathway41
The Effect of CpG Sequences on Capsid-Specific CD8+ T Cell Responses to AAV Vector Gene Transfer40
Immune Modulation by Telomerase-Specific Oncolytic Adenovirus Synergistically Enhances Antitumor Efficacy with Anti-PD1 Antibody40
SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease40
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways40
mRNA-LNP vaccines tuned for systemic immunization induce strong antitumor immunity by engaging splenic immune cells40
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation40
CXCL11-armed oncolytic adenoviruses enhance CAR-T cell therapeutic efficacy and reprogram tumor microenvironment in glioblastoma39
Type I IFN Sensing by cDCs and CD4+ T Cell Help Are Both Requisite for Cross-Priming of AAV Capsid-Specific CD8+ T Cells39
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver39
Precise Targeting of miRNA Sites Restores CFTR Activity in CF Bronchial Epithelial Cells39
Imbalance of TGF-β1/BMP-7 pathways induced by M2-polarized macrophages promotes hepatocellular carcinoma aggressiveness39
Circular RNA Cdyl promotes abdominal aortic aneurysm formation by inducing M1 macrophage polarization and M1-type inflammation39
mRNA-encoded, constitutively active STINGV155M is a potent genetic adjuvant of antigen-specific CD8+ T cell response38
Debulking SARS-CoV-2 in saliva using angiotensin converting enzyme 2 in chewing gum to decrease oral virus transmission and infection38
Leukemic extracellular vesicles induce chimeric antigen receptor T cell dysfunction in chronic lymphocytic leukemia38
Innate Inhibiting Proteins Enhance Expression and Immunogenicity of Self-Amplifying RNA38
Ligand-Induced Degradation of a CAR Permits Reversible Remote Control of CAR T Cell Activity In Vitro and In Vivo38
Upregulating Lin28a Promotes Axon Regeneration in Adult Mice with Optic Nerve and Spinal Cord Injury38
lnc-MAP3K13-7:1 Inhibits Ovarian GC Proliferation in PCOS via DNMT1 Downregulation-Mediated CDKN1A Promoter Hypomethylation38
Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A38
circEHBP1 promotes lymphangiogenesis and lymphatic metastasis of bladder cancer via miR-130a-3p/TGFβR1/VEGF-D signaling38
Exosomes: A Potential Therapeutic Tool Targeting Communications between Tumor Cells and Macrophages37
The roles of PD-1/PD-L1 in the prognosis and immunotherapy of prostate cancer37
Macrophages as a therapeutic target to promote diabetic wound healing37
Coactivation of Endogenous Wnt10b and Foxc2 by CRISPR Activation Enhances BMSC Osteogenesis and Promotes Calvarial Bone Regeneration37
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease37
TGF-β1-Licensed Murine MSCs Show Superior Therapeutic Efficacy in Modulating Corneal Allograft Immune Rejection In Vivo37
AAV Capsid-Promoter Interactions Determine CNS Cell-Selective Gene Expression In Vivo37
mRNALocater: Enhance the prediction accuracy of eukaryotic mRNA subcellular localization by using model fusion strategy37
Self-amplifying loop of NF-κB and periostin initiated by PIEZO1 accelerates mechano-induced senescence of nucleus pulposus cells and intervertebral disc degeneration36
Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles36
Single-Cell Transcriptomic Analysis Reveals BCMA CAR-T Cell Dynamics in a Patient with Refractory Primary Plasma Cell Leukemia36
Immunogenicity of a new gorilla adenovirus vaccine candidate for COVID-1936
DPP4/CD32b/NF-κB Circuit: A Novel Druggable Target for Inhibiting CRP-Driven Diabetic Nephropathy36
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy35
miR-204-3p/Nox4 Mediates Memory Deficits in a Mouse Model of Alzheimer’s Disease35
Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa35
Efficient Gene Silencing by Adenine Base Editor-Mediated Start Codon Mutation35
Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety34
Microglial lnc-U90926 facilitates neutrophil infiltration in ischemic stroke via MDH2/CXCL2 axis34
Circular RNA circSnx5 Controls Immunogenicity of Dendritic Cells through the miR-544/SOCS1 Axis and PU.1 Activity Regulation34
The CD39+ HBV surface protein-targeted CAR-T and personalized tumor-reactive CD8+ T cells exhibit potent anti-HCC activity34
Adenovirus-Antibody Complexes Contributed to Lethal Systemic Inflammation in a Gene Therapy Trial33
siRNA Drugs: Here to Stay33
FOSL1 promotes metastasis of head and neck squamous cell carcinoma through super-enhancer-driven transcription program33
TRERNA1 upregulation mediated by HBx promotes sorafenib resistance and cell proliferation in HCC via targeting NRAS by sponging miR-22-3p33
Gene Therapy Intervention in Neovascular Eye Disease: A Recent Update33
The emerging regulatory roles of long non-coding RNAs implicated in cancer metabolism32
Upregulation of PUM1 Expression in Preeclampsia Impairs Trophoblast Invasion by Negatively Regulating the Expression of the lncRNA HOTAIR32
A truncated reverse transcriptase enhances prime editing by split AAV vectors32
Aerosol Delivery of Synthetic mRNA to Vaginal Mucosa Leads to Durable Expression of Broadly Neutralizing Antibodies against HIV32
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target32
Long noncoding RNA LINC01234 promotes hepatocellular carcinoma progression through orchestrating aspartate metabolic reprogramming32
Targeting CD33 in Chemoresistant AML Patient-Derived Xenografts by CAR-CIK Cells Modified with an Improved SB Transposon System32
ADAR2 increases in exercised heart and protects against myocardial infarction and doxorubicin-induced cardiotoxicity32
T Cells Expressing NKG2D CAR with a DAP12 Signaling Domain Stimulate Lower Cytokine Production While Effective in Tumor Eradication32
Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice31
MicroRNA-10 negatively regulates inflammation in diabetic kidney via targeting activation of the NLRP3 inflammasome31
CircRNA Samd4 induces cardiac repair after myocardial infarction by blocking mitochondria-derived ROS output31
COVID-19-Induced ARDS Is Associated with Decreased Frequency of Activated Memory/Effector T Cells Expressing CD11a++31
CRISPR/Cas9-Mediated miR-29b Editing as a Treatment of Different Types of Muscle Atrophy in Mice31
Novel Long Non-coding RNA lncAMPC Promotes Metastasis and Immunosuppression in Prostate Cancer by Stimulating LIF/LIFR Expression30
Extracellular vesicles: The next generation in gene therapy delivery30
Dual Targeting of Mesothelin and CD19 with Chimeric Antigen Receptor-Modified T Cells in Patients with Metastatic Pancreatic Cancer30
CRISPR technologies for the treatment of Duchenne muscular dystrophy30
Cell-derived nanovesicle-mediated drug delivery to the brain: Principles and strategies for vesicle engineering30
A self-amplifying mRNA SARS-CoV-2 vaccine candidate induces safe and robust protective immunity in preclinical models30
lncRNA ZNF649-AS1 Induces Trastuzumab Resistance by Promoting ATG5 Expression and Autophagy30
sEVs from tonsil-derived mesenchymal stromal cells alleviate activation of hepatic stellate cells and liver fibrosis through miR-486-5p30
Mesenchymal stromal cell delivery of oncolytic immunotherapy improves CAR-T cell antitumor activity30
Telomerase therapy attenuates cardiotoxic effects of doxorubicin29
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis29
m6A modification regulates lung fibroblast-to-myofibroblast transition through modulating KCNH6 mRNA translation29
Beyond cancer cells: Targeting the tumor microenvironment with gene therapy and armed oncolytic virus29
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil29
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system28
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function28
A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease28
Hierarchical graph attention network for miRNA-disease association prediction28
AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice28
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease28
HIV-Resistant and HIV-Specific CAR-Modified CD4+ T Cells Mitigate HIV Disease Progression and Confer CD4+ T Cell Help In Vivo28
Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics28
Single SERCA2a Therapy Ameliorated Dilated Cardiomyopathy for 18 Months in a Mouse Model of Duchenne Muscular Dystrophy28
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies28
Gentamicin Induces Laminin 332 and Improves Wound Healing in Junctional Epidermolysis Bullosa Patients with Nonsense Mutations27
Locally secreted BiTEs complement CAR T cells by enhancing killing of antigen heterogeneous solid tumors27
Safety, Tumor Reduction, and Clinical Impact of Zika Virus Injection in Dogs with Advanced-Stage Brain Tumors27
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice27
miR-486 attenuates cardiac ischemia/reperfusion injury and mediates the beneficial effect of exercise for myocardial protection27
RBP EIF2S2 Promotes Tumorigenesis and Progression by Regulating MYC-Mediated Inhibition via FHIT-Related Enhancers27
TGFB1/INHBA Homodimer/Nodal-SMAD2/3 Signaling Network: A Pivotal Molecular Target in PDAC Treatment26
Transient Inhibition of PI3Kδ Enhances the Therapeutic Effect of Intravenous Delivery of Oncolytic Vaccinia Virus26
Gsx1 promotes locomotor functional recovery after spinal cord injury26
Self-assembled miRNA-switch nanoparticles target denuded regions and prevent restenosis26
Cardiac Nestin+ Mesenchymal Stromal Cells Enhance Healing of Ischemic Heart through Periostin-Mediated M2 Macrophage Polarization26
Quantification of CpG Motifs in rAAV Genomes: Avoiding the Toll26
An antisense circular RNA circSCRIB enhances cancer progression by suppressing parental gene splicing and translation26
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells25
An Optimized Full-Length FLT3/CD3 Bispecific Antibody Demonstrates Potent Anti-leukemia Activity and Reversible Hematological Toxicity25
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease25
Safety and immunogenicity of ChAd63-KH vaccine in post-kala-azar dermal leishmaniasis patients in Sudan25
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway25
Rational Selection of CRISPR-Cas9 Guide RNAs for Homology-Directed Genome Editing25
Apoptotic vesicles activate autophagy in recipient cells to induce angiogenesis and dental pulp regeneration25
Precision medicine: In vivo CAR therapy as a showcase for receptor-targeted vector platforms25
Inhibition of lncRNA MAAT Controls Multiple Types of Muscle Atrophy by cis- and trans-Regulatory Actions25
FTO promotes multiple myeloma progression by posttranscriptional activation of HSF1 in an m6A-YTHDF2-dependent manner25
lncRNA lnc-POP1-1 upregulated by VN1R5 promotes cisplatin resistance in head and neck squamous cell carcinoma through interaction with MCM524
Targeting SOX2 Protein with Peptide Aptamers for Therapeutic Gains against Esophageal Squamous Cell Carcinoma24
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy24
LAG-3xPD-L1 bispecific antibody potentiates antitumor responses of T cells through dendritic cell activation24
CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia24
Impacts and mechanisms of alternative mRNA splicing in cancer metabolism, immune response, and therapeutics24
Reactive Myelopoiesis Triggered by Lymphodepleting Chemotherapy Limits the Efficacy of Adoptive T Cell Therapy24
Intrastromal Gene Therapy Prevents and Reverses Advanced Corneal Clouding in a Canine Model of Mucopolysaccharidosis I24
Targeting of pancreatic cancer cells and stromal cells using engineered oncolytic Salmonella typhimurium24
Intranasal infusion of GD3 and GM1 gangliosides downregulates alpha-synuclein and controls tyrosine hydroxylase gene in a PD model mouse23
The rational development of CD5-targeting biepitopic CARs with fully human heavy-chain-only antigen recognition domains23
Activation of NF-κB-Inducing Kinase in Islet β Cells Causes β Cell Failure and Diabetes23
CRISPR-Cas9-Edited Tacrolimus-Resistant Antiviral T Cells for Advanced Adoptive Immunotherapy in Transplant Recipients23
Glycine Enhances Satellite Cell Proliferation, Cell Transplantation, and Oligonucleotide Efficacy in Dystrophic Muscle23
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