Molecular Therapy

Article	Citations
Current Clinical Applications of In Vivo Gene Therapy with AAVs	454
Plant Exosome-like Nanovesicles: Emerging Therapeutics and Drug Delivery Nanoplatforms	281
CAR T Cell Therapy for Solid Tumors: Bright Future or Dark Reality?	225
A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties	180
xCT: A Critical Molecule That Links Cancer Metabolism to Redox Signaling	179
Emerging mechanisms and targeted therapy of ferroptosis in cancer	168
M2 Macrophage-Derived Exosomes Promote Angiogenesis and Growth of Pancreatic Ductal Adenocarcinoma by Targeting E2F2	167
A Nuclear Long Non-Coding RNA LINC00618 Accelerates Ferroptosis in a Manner Dependent upon Apoptosis	163
CRISPR-Cas9: A Preclinical and Clinical Perspective for the Treatment of Human Diseases	158
Personalized Cancer Vaccines: Clinical Landscape, Challenges, and Opportunities	150
METTL1/WDR4-mediated m7G tRNA modifications and m7G codon usage promote mRNA translation and lung cancer progression	139
AAV vectors: The Rubik’s cube of human gene therapy	133
COVID-19 mRNA vaccines: Platforms and current developments	133
Plant-derived exosomal microRNAs inhibit lung inflammation induced by exosomes SARS-CoV-2 Nsp12	129
lncRNA SNHG11 Promotes Gastric Cancer Progression by Activating the Wnt/β-Catenin Pathway and Oncogenic Autophagy	126
Lyophilization provides long-term stability for a lipid nanoparticle-formulated, nucleoside-modified mRNA vaccine	126
Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs	122
Role of exosomal non-coding RNAs from tumor cells and tumor-associated macrophages in the tumor microenvironment	119
A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice	114
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines	114
Tumor-derived exosomal microRNA-106b-5p activates EMT-cancer cell and M2-subtype TAM interaction to facilitate CRC metastasis	112
A SARS-CoV-2 targeted siRNA-nanoparticle therapy for COVID-19	110
Macrophages as a therapeutic target to promote diabetic wound healing	108
Gene therapy for ALS: A review	108
Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy	106

In vivo somatic cell base editing and prime editing	106
Allergic Reactions and Anaphylaxis to LNP-Based COVID-19 Vaccines	100
Evaluating the state of the science for adeno-associated virus integration: An integrated perspective	97
Microglia secrete miR-146a-5p-containing exosomes to regulate neurogenesis in depression	95
CART19-BE-01: A Multicenter Trial of ARI-0001 Cell Therapy in Patients with CD19+ Relapsed/Refractory Malignancies	95
Dual-AAV delivering split prime editor system for in vivo genome editing	95
Exosomal miR-208b related with oxaliplatin resistance promotes Treg expansion in colorectal cancer	94
lncRNA ITGB8-AS1 functions as a ceRNA to promote colorectal cancer growth and migration through integrin-mediated focal adhesion signaling	94
A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II	93
METTL3 restrains papillary thyroid cancer progression via m6A/c-Rel/IL-8-mediated neutrophil infiltration	93
circ-EIF6 encodes EIF6-224aa to promote TNBC progression via stabilizing MYH9 and activating the Wnt/beta-catenin pathway	88
Dysregulated m6A modification promotes lipogenesis and development of non-alcoholic fatty liver disease and hepatocellular carcinoma	86
CXCL11-armed oncolytic adenoviruses enhance CAR-T cell therapeutic efficacy and reprogram tumor microenvironment in glioblastoma	86
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations	85
Circular RNAs: Expression, localization, and therapeutic potentials	84
Exosome-derived circTRPS1 promotes malignant phenotype and CD8+ T cell exhaustion in bladder cancer microenvironments	83
Cancer-secreted exosomal miR-1468-5p promotes tumor immune escape via the immunosuppressive reprogramming of lymphatic vessels	83
Synergistic and Antagonistic Drug Combinations against SARS-CoV-2	80
METTL3-mediated m6A modification of TIMP2 mRNA promotes podocyte injury in diabetic nephropathy	78
Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency	78
AAV integration in human hepatocytes	76
Liver Injury Increases the Incidence of HCC following AAV Gene Therapy in Mice	73
The evolving landscape of N6-methyladenosine modification in the tumor microenvironment	72
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	71
Photodynamic therapy synergizes with PD-L1 checkpoint blockade for immunotherapy of CRC by multifunctional nanoparticles	70
Aptamers: Cutting edge of cancer therapies	69
Development of CAR T Cells Expressing a Suicide Gene Plus a Chimeric Antigen Receptor Targeting Signaling Lymphocytic-Activation Molecule F7	69
Exosomal miR-1246 from glioma patient body fluids drives the differentiation and activation of myeloid-derived suppressor cells	68
Extracellular vesicles: The next generation in gene therapy delivery	68
Emerging Role of Ubiquitination in the Regulation of PD-1/PD-L1 in Cancer Immunotherapy	67
circMRPS35 promotes malignant progression and cisplatin resistance in hepatocellular carcinoma	67
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	66
YAP Circular RNA, circYap, Attenuates Cardiac Fibrosis via Binding with Tropomyosin-4 and Gamma-Actin Decreasing Actin Polymerization	66
A Distinct Transcriptional Program in Human CAR T Cells Bearing the 4-1BB Signaling Domain Revealed by scRNA-Seq	66
Transfer RNA-Derived Small RNAs: Another Layer of Gene Regulation and Novel Targets for Disease Therapeutics	66
Olaparib Suppresses MDSC Recruitment via SDF1α/CXCR4 Axis to Improve the Anti-tumor Efficacy of CAR-T Cells on Breast Cancer in Mice	66
Ginseng-derived nanoparticles potentiate immune checkpoint antibody efficacy by reprogramming the cold tumor microenvironment	65
Bulk and single-cell transcriptome profiling reveal the metabolic heterogeneity in human breast cancers	64
Microglial vesicles improve post-stroke recovery by preventing immune cell senescence and favoring oligodendrogenesis	63
Adenylate Kinase 4 Modulates the Resistance of Breast Cancer Cells to Tamoxifen through an m6A-Based Epitranscriptomic Mechanism	63
Circular RNA circ-FoxO3 attenuates blood-brain barrier damage by inducing autophagy during ischemia/reperfusion	63
Pre-conditioning modifies the TME to enhance solid tumor CAR T cell efficacy and endogenous protective immunity	62
Self-amplifying loop of NF-κB and periostin initiated by PIEZO1 accelerates mechano-induced senescence of nucleus pulposus cells and intervertebral disc degeneration	62
FOXO3A-induced LINC00926 suppresses breast tumor growth and metastasis through inhibition of PGK1-mediated Warburg effect	62
mRNA-LNP vaccines tuned for systemic immunization induce strong antitumor immunity by engaging splenic immune cells	62
Engineered prime editors with PAM flexibility	61
The roles of PD-1/PD-L1 in the prognosis and immunotherapy of prostate cancer	60
Novel Genetic and Epigenetic Biomarkers of Prognostic and Predictive Significance in Stage II/III Colorectal Cancer	59
Inhibition of xCT suppresses the efficacy of anti-PD-1/L1 melanoma treatment through exosomal PD-L1-induced macrophage M2 polarization	59
Circular RNA Cdyl promotes abdominal aortic aneurysm formation by inducing M1 macrophage polarization and M1-type inflammation	58

A bispecific antibody targeting GPC3 and CD47 induced enhanced antitumor efficacy against dual antigen-expressing HCC	58
IL-15 Preconditioning Augments CAR T Cell Responses to Checkpoint Blockade for Improved Treatment of Solid Tumors	58
Clinical Applications of Single-Stranded Oligonucleotides: Current Landscape of Approved and In-Development Therapeutics	57
A flexible split prime editor using truncated reverse transcriptase improves dual-AAV delivery in mouse liver	57
mRNA-encoded, constitutively active STINGV155M is a potent genetic adjuvant of antigen-specific CD8+ T cell response	56
COVID-eVax, an electroporated DNA vaccine candidate encoding the SARS-CoV-2 RBD, elicits protective responses in animal models	56
Imbalance of TGF-β1/BMP-7 pathways induced by M2-polarized macrophages promotes hepatocellular carcinoma aggressiveness	55
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	55
Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	55
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	54
CircRNA Samd4 induces cardiac repair after myocardial infarction by blocking mitochondria-derived ROS output	54
Clinical CAR-T Cell and Oncolytic Virotherapy for Cancer Treatment	54
Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases	54
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	52
lnc-MAP3K13-7:1 Inhibits Ovarian GC Proliferation in PCOS via DNMT1 Downregulation-Mediated CDKN1A Promoter Hypomethylation	52
Cell-derived nanovesicle-mediated drug delivery to the brain: Principles and strategies for vesicle engineering	52
NONO Inhibits Lymphatic Metastasis of Bladder Cancer via Alternative Splicing of SETMAR	51
Innate Inhibiting Proteins Enhance Expression and Immunogenicity of Self-Amplifying RNA	51
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	50
Leukemic extracellular vesicles induce chimeric antigen receptor T cell dysfunction in chronic lymphocytic leukemia	50
Bioinspired artificial exosomes based on lipid nanoparticles carrying let-7b-5p promote angiogenesis in vitro and in vivo	50
First-in-Human Phase I Clinical Trial of an SFV-Based RNA Replicon Cancer Vaccine against HPV-Induced Cancers	49
ADAR2 increases in exercised heart and protects against myocardial infarction and doxorubicin-induced cardiotoxicity	49
Apoptotic vesicles activate autophagy in recipient cells to induce angiogenesis and dental pulp regeneration	49
FOSL1 promotes metastasis of head and neck squamous cell carcinoma through super-enhancer-driven transcription program	48
circEHBP1 promotes lymphangiogenesis and lymphatic metastasis of bladder cancer via miR-130a-3p/TGFβR1/VEGF-D signaling	48
In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model	48
Hierarchical graph attention network for miRNA-disease association prediction	47
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	47
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	47
The CD39+ HBV surface protein-targeted CAR-T and personalized tumor-reactive CD8+ T cells exhibit potent anti-HCC activity	47
Circular RNA circPDE4D Protects against Osteoarthritis by Binding to miR-103a-3p and Regulating FGF18	47
miR-204-3p/Nox4 Mediates Memory Deficits in a Mouse Model of Alzheimer’s Disease	47
A truncated reverse transcriptase enhances prime editing by split AAV vectors	46
Pros and Cons of Adenovirus-Based SARS-CoV-2 Vaccines	46
Early Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A	46
Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9	45
Docosanoic acid conjugation to siRNA enables functional and safe delivery to skeletal and cardiac muscles	45
miR-486 attenuates cardiac ischemia/reperfusion injury and mediates the beneficial effect of exercise for myocardial protection	44
LAG-3xPD-L1 bispecific antibody potentiates antitumor responses of T cells through dendritic cell activation	44
A self-amplifying mRNA SARS-CoV-2 vaccine candidate induces safe and robust protective immunity in preclinical models	44
m6A modification regulates lung fibroblast-to-myofibroblast transition through modulating KCNH6 mRNA translation	44
mRNALocater: Enhance the prediction accuracy of eukaryotic mRNA subcellular localization by using model fusion strategy	44
Locally secreted BiTEs complement CAR T cells by enhancing killing of antigen heterogeneous solid tumors	43
RNA editing: Expanding the potential of RNA therapeutics	43
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	43
MicroRNA-10 negatively regulates inflammation in diabetic kidney via targeting activation of the NLRP3 inflammasome	43
Beyond cancer cells: Targeting the tumor microenvironment with gene therapy and armed oncolytic virus	43
Single and Dual Vector Gene Therapy with AAV9-PHP.B Rescues Hearing in Tmc1 Mutant Mice	42
Microglial lnc-U90926 facilitates neutrophil infiltration in ischemic stroke via MDH2/CXCL2 axis	42
Single-Cell Transcriptomic Analysis Reveals BCMA CAR-T Cell Dynamics in a Patient with Refractory Primary Plasma Cell Leukemia	42
TRERNA1 upregulation mediated by HBx promotes sorafenib resistance and cell proliferation in HCC via targeting NRAS by sponging miR-22-3p	42
Durability of transgene expression after rAAV gene therapy	41
Advances in gene therapy hold promise for treating hereditary hearing loss	41
Debulking SARS-CoV-2 in saliva using angiotensin converting enzyme 2 in chewing gum to decrease oral virus transmission and infection	41
The emerging regulatory roles of long non-coding RNAs implicated in cancer metabolism	41
T Cells Expressing NKG2D CAR with a DAP12 Signaling Domain Stimulate Lower Cytokine Production While Effective in Tumor Eradication	41
Immunogenicity of a new gorilla adenovirus vaccine candidate for COVID-19	41
IL-4 polarized human macrophage exosomes control cardiometabolic inflammation and diabetes in obesity	40
Precision medicine: In vivo CAR therapy as a showcase for receptor-targeted vector platforms	40
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	39
siRNA Drugs: Here to Stay	39
Long noncoding RNA LINC01234 promotes hepatocellular carcinoma progression through orchestrating aspartate metabolic reprogramming	39
DPP4/CD32b/NF-κB Circuit: A Novel Druggable Target for Inhibiting CRP-Driven Diabetic Nephropathy	38
The rational development of CD5-targeting biepitopic CARs with fully human heavy-chain-only antigen recognition domains	38
Mesenchymal stromal cell delivery of oncolytic immunotherapy improves CAR-T cell antitumor activity	38
CRISPR technologies for the treatment of Duchenne muscular dystrophy	38
Eliciting anti-cancer immunity by genetically engineered multifunctional exosomes	38
Anti-atherosclerotic therapies: Milestones, challenges, and emerging innovations	37
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	37
Telomerase therapy attenuates cardiotoxic effects of doxorubicin	37
sEVs from tonsil-derived mesenchymal stromal cells alleviate activation of hepatic stellate cells and liver fibrosis through miR-486-5p	37
Gsx1 promotes locomotor functional recovery after spinal cord injury	37
Inhibition of Tau seeding by targeting Tau nucleation core within neurons with a single domain antibody fragment	37
Circular RNA circSnx5 Controls Immunogenicity of Dendritic Cells through the miR-544/SOCS1 Axis and PU.1 Activity Regulation	37
Gene editing in a Myo6 semi-dominant mouse model rescues auditory function	36
Iduronate-2-sulfatase fused with anti-hTfR antibody, pabinafusp alfa, for MPS-II: A phase 2 trial in Brazil	36
lncRNA ZNF649-AS1 Induces Trastuzumab Resistance by Promoting ATG5 Expression and Autophagy	36
A comprehensive study of a 29-capsid AAV library in a non-human primate central nervous system	36
NMNAT2 is downregulated in glaucomatous RGCs, and RGC-specific gene therapy rescues neurodegeneration and visual function	36

CRISPR-Cas9-Edited Tacrolimus-Resistant Antiviral T Cells for Advanced Adoptive Immunotherapy in Transplant Recipients	35
Dual Targeting of Mesothelin and CD19 with Chimeric Antigen Receptor-Modified T Cells in Patients with Metastatic Pancreatic Cancer	35
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells	35
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy	35
Safety and immunogenicity of ChAd63-KH vaccine in post-kala-azar dermal leishmaniasis patients in Sudan	35
An antisense circular RNA circSCRIB enhances cancer progression by suppressing parental gene splicing and translation	35
Predicting genotoxicity of viral vectors for stem cell gene therapy using gene expression-based machine learning	34
FTO promotes multiple myeloma progression by posttranscriptional activation of HSF1 in an m6A-YTHDF2-dependent manner	34
TGFB1/INHBA Homodimer/Nodal-SMAD2/3 Signaling Network: A Pivotal Molecular Target in PDAC Treatment	34
Prodrugs and prodrug-activated systems in gene therapy	34
COVID-19-Induced ARDS Is Associated with Decreased Frequency of Activated Memory/Effector T Cells Expressing CD11a++	34
Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease	34
Zebularine elevates STING expression and enhances cGAMP cancer immunotherapy in mice	33
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	33
Targeting of pancreatic cancer cells and stromal cells using engineered oncolytic Salmonella typhimurium	33
lncRNA lnc-POP1-1 upregulated by VN1R5 promotes cisplatin resistance in head and neck squamous cell carcinoma through interaction with MCM5	33
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B	33
Novel Long Non-coding RNA lncAMPC Promotes Metastasis and Immunosuppression in Prostate Cancer by Stimulating LIF/LIFR Expression	33
A pause in gene therapy: Reflecting on the unique challenges of sickle cell disease	33
Reshaping the Immune Microenvironment by Oncolytic Herpes Simplex Virus in Murine Pancreatic Ductal Adenocarcinoma	32
Feasibility and preclinical efficacy of CD7-unedited CD7 CAR T cells for T cell malignancies	32
Impacts and mechanisms of alternative mRNA splicing in cancer metabolism, immune response, and therapeutics	32
Clinical perspective: Antibody-drug conjugates for the treatment of HER2-positive breast cancer	31
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	31
The cardiac translational landscape reveals that micropeptides are new players involved in cardiomyocyte hypertrophy	31
Targeting Complement C5a Receptor 1 for the Treatment of Immunosuppression in Sepsis	31
Isoforms of MUC16 activate oncogenic signaling through EGF receptors to enhance the progression of pancreatic cancer	31
Lethal immunotoxicity in high-dose systemic AAV therapy	31
Self-assembled miRNA-switch nanoparticles target denuded regions and prevent restenosis	31
The m6A methyltransferase Mettl3 deficiency attenuates hepatic stellate cell activation and liver fibrosis	31
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	31
cGAS/STING cross-talks with cell cycle and potentiates cancer immunotherapy	31
Pharmacokinetics and Proceedings in Clinical Application of Nucleic Acid Therapeutics	31
RNA m6A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of β-catenin	31
Rational Selection of CRISPR-Cas9 Guide RNAs for Homology-Directed Genome Editing	31
CRISPR-Cas9-Mediated ELANE Mutation Correction in Hematopoietic Stem and Progenitor Cells to Treat Severe Congenital Neutropenia	30
Adenovirus-based vaccines—a platform for pandemic preparedness against emerging viral pathogens	30
Bone marrow mesenchymal stem cell-derived exosomal miR-34c-5p ameliorates RIF by inhibiting the core fucosylation of multiple proteins	30
Development of an mRNA-lipid nanoparticle vaccine against Lyme disease	30
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	30
CAR- and TRuC-redirected regulatory T cells differ in capacity to control adaptive immunity to FVIII	30
Mucosal-associated invariant T cells for cancer immunotherapy	30
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	30
EVs Containing Host Restriction Factor IFITM3 Inhibited ZIKV Infection of Fetuses in Pregnant Mice through Trans-placenta Delivery	29
A dual-antigen self-amplifying RNA SARS-CoV-2 vaccine induces potent humoral and cellular immune responses and protects against SARS-CoV-2 variants through T cell-mediated immunity	29
Centyrin ligands for extrahepatic delivery of siRNA	29
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis	29
Activation of NF-κB-Inducing Kinase in Islet β Cells Causes β Cell Failure and Diabetes	29
Universal allogeneic CAR T cells engineered with Sleeping Beauty transposons and CRISPR-CAS9 for cancer immunotherapy	29
AAV5 delivery of CRISPR-Cas9 supports effective genome editing in mouse lung airway	29
Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice	29
Fecal miR-142a-3p from dextran sulfate sodium-challenge recovered mice prevents colitis by promoting the growth of Lactobacillus reuteri	29
Cerebral dopamine neurotrophic factor reduces α-synuclein aggregation and propagation and alleviates behavioral alterations in vivo	29
AAV9 Gene Therapy Increases Lifespan and Treats Pathological and Behavioral Abnormalities in a Mouse Model of CLN8-Batten Disease	29
Targeted genomic translocations and inversions generated using a paired prime editing strategy	29
Pro-inflammatory concerns with lipid nanoparticles	29
N6-methyladenosine demethylase FTO enhances chemo-resistance in colorectal cancer through SIVA1-mediated apoptosis	28
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	28
Intranasal infusion of GD3 and GM1 gangliosides downregulates alpha-synuclein and controls tyrosine hydroxylase gene in a PD model mouse	28
Kidney single-cell transcriptome profile reveals distinct response of proximal tubule cells to SGLT2i and ARB treatment in diabetic mice	28
Tim-3 regulates sepsis-induced immunosuppression by inhibiting the NF-κB signaling pathway in CD4 T cells	28
FOSL1 promotes proneural-to-mesenchymal transition of glioblastoma stem cells via UBC9/CYLD/NF-κB axis	28
Targeting tissue-resident memory CD8+ T cells in the kidney is a potential therapeutic strategy to ameliorate podocyte injury and glomerulosclerosis	28
CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi	28
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus	28
PSCs Reveal PUFA-Provoked Mitochondrial Stress as a Central Node Potentiating RPE Degeneration in Bietti’s Crystalline Dystrophy	27
Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing	27
Genome editing in large animal models	27
Radiation enhances the efficacy of EGFR-targeted CAR-T cells against triple-negative breast cancer by activating NF-κB/Icam1 signaling	26
Correction of recessive dystrophic epidermolysis bullosa by homology-directed repair-mediated genome editing	26
Anti-tau scFvs Targeted to the Cytoplasm or Secretory Pathway Variably Modify Pathology and Neurodegenerative Phenotypes	26
Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction	26
Lipid-nanoparticle-encapsulated mRNA vaccines induce protective memory CD8 T cells against a lethal viral infection	26
sEVsRVG selectively delivers antiviral siRNA to fetus brain, inhibits ZIKV infection and mitigates ZIKV-induced microcephaly in mouse model	26
Inhibition of lncRNA MAAT Controls Multiple Types of Muscle Atrophy by cis- and trans-Regulatory Actions	26
Epigenetic strategies to boost CAR T cell therapy	26
Clearance of heparan sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice	26
Enforced dimerization between XBP1s and ATF6f enhances the protective effects of the UPR in models of neurodegeneration	26
ESC-sEVs Rejuvenate Aging Hippocampal NSCs by Transferring SMADs to Regulate the MYT1-Egln3-Sirt1 Axis	26
Targeted Delivery of CXCL9 and OX40L by Mesenchymal Stem Cells Elicits Potent Antitumor Immunity	26
Evidence in primates supporting the use of chemogenetics for the treatment of human refractory neuropsychiatric disorders	26
Recent advances in targeted delivery of non-coding RNA-based therapeutics for atherosclerosis	26
Th1 cytokine interferon gamma improves response in HER2 breast cancer by modulating the ubiquitin proteasomal pathway	26
Dual-antigen targeted iPSC-derived chimeric antigen receptor-T cell therapy for refractory lymphoma	25
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease	25
DUX4 Transcript Knockdown with Antisense 2′-O-Methoxyethyl Gapmers for the Treatment of Facioscapulohumeral Muscular Dystrophy	25
PROTAC therapy as a new targeted therapy for lung cancer	25
NLRC3 expression in macrophage impairs glycolysis and host immune defense by modulating the NF-κB-NFAT5 complex during septic immunosuppression	25
CRISPR activation of endogenous genes reprograms fibroblasts into cardiovascular progenitor cells for myocardial infarction therapy	25
Key benefits of dexamethasone and antibody treatment in COVID-19 hamster models revealed by single-cell transcriptomics	25
Systemic delivery of antagomirs during blood-brain barrier disruption is disease-modifying in experimental epilepsy	25
Melatonin Induction of APP Intracellular Domain 50 SUMOylation Alleviates AD through Enhanced Transcriptional Activation and Aβ Degradation	25
Limited astrocyte-to-neuron conversion in the mouse brain using NeuroD1 overexpression	25
Fetal and Maternal Safety Considerations for In Utero Therapy Clinical Trials: iFeTiS Consensus Statement	25
An armed oncolytic virus enhances the efficacy of tumor-infiltrating lymphocyte therapy by converting tumors to artificial antigen-presenting cells in situ	25