Molecular Therapy

Article	Citations
Retraction Notice to: Risk-Associated Long Noncoding RNA FOXD3-AS1 Inhibits Neuroblastoma Progression by Repressing PARP1-Mediated Activation of CTCF	214
Retraction Notice to: Long Noncoding RNA LINC00673 Is Activated by SP1 and Exerts Oncogenic Properties by Interacting with LSD1 and EZH2 in Gastric Cancer	205
Retraction Notice to: The Pseudogene DUXAP8 Promotes Non-small-cell Lung Cancer Cell Proliferation and Invasion by Epigenetically Silencing EGR1 and RHOB	204
Retraction Notice to: METTL14 Suppresses CRC Progression via Regulating N6-Methyladenosine-Dependent Primary miR-375 Processing	184
lncRNA Linc00173 modulates glucose metabolism and multidrug chemoresistance inSCLC:Potentialmolecular panel for targeted therapy	178
Retraction Notice to: lncRNA RMST Enhances DNMT3 Expression through Interaction with HuR	177
The impact of revised regenerative medicine regulations on unapproved gene therapies in Japan	165
Deficiency of galactosyl-ceramidase in adult oligodendrocytes worsens disease severity during chronic experimental allergic encephalomyelitis	165
Dasatinib-resistant universal CAR-T cells proliferate in the presence of host immune cells and exhibit antitumor activity	159
Response to: DNA transposon mechanisms and pathways of genotoxicity	148
CRISPR-Cas9 as a Trojan horse	137
ATGL regulates renal fibrosis by reprogramming lipid metabolism during the transition from AKI to CKD	136
Controlling intracellular protein delivery, tumor colonization and tissue distribution using flhDC in clinically relevant ΔsseJ Salmonella	131
Inverted HA-EV immunization elicits stalk-specific influenza immunity and cross-protection in mice	129
Inhibition of adenylyl cyclase 8 prevents the upregulation of Orai1 channel, which improves cardiac function after myocardial infarction	129
FOXC1 modulates stem-like cell properties and chemoresistance through hedgehog and EMT signaling in gastric adenocarcinoma	129
Y4 RNA fragment alleviates myocardial injury in heart transplantation via SNRNP200 to enhance IL-10 mRNA splicing	126
Promotion or inhibition? This is a question in gene editing	123
RNA and epigenetic editing: The new frontier in gene and cell therapy	116
The emerging regulatory roles of long non-coding RNAs implicated in cancer metabolism	114
Call for papers: Exploiting extracellular vesicles as therapeutic agents	112
Reversing the path: Mesenchymal-to-mesothelial transition as a novel target in liver fibrosis	112
Base editing the synapse: Modeling a complex neurological disorder in non-human primates	111
In this issue	111
In this issue	111

Retraction Notice to: Role of HCP5-miR-139-RUNX1 Feedback Loop in Regulating Malignant Behavior of Glioma Cells	111
2022 ASGCT Annual Meeting Late-breaking Abstracts	110
ASGCT 27th Annual Meeting Late-Breaking Abstracts	109
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	103
Accessorizing viral vectors with extracellular vesicles for enhanced performance	100
Heal thyself: The promise of autologous hematopoietic stem cell gene therapy in neurometabolic disorders	98
Mavis Agbandje-McKenna’s lifelong commitment to teaching and research	96
Financial considerations in expanded access policy for gene therapies: A tough nut to crack?	94
The first medical education program on gene and gene-modified cell therapies for Latin America	92
In vivo genome editing for inherited retinal disease: Opportunities and challenges	91
In this issue	87
Rationally designed DNA therapeutics can modulate human TH expression by controlling specific GQ formation in its promoter	86
AAV and hepatitis: Cause or coincidence?	83
Isoforms of MUC16 activate oncogenic signaling through EGF receptors to enhance the progression of pancreatic cancer	82
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	82
Systematic comparison of rAAV vectors manufactured using large-scale suspension cultures of Sf9 and HEK293 cells	81
CasRx-based Wnt activation promotes alveolar regeneration while ameliorating pulmonary fibrosis in a mouse model of lung injury	81
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	81
Systemic delivery of antagomirs during blood-brain barrier disruption is disease-modifying in experimental epilepsy	80
Engineering small-molecule and protein drugs for targeting bone tumors	80
Targeting RNA oxidation by ISG20-mediated degradation is a potential therapeutic strategy for acute kidney injury	80
The efficacy of COVID-19 vaccines against the B.1.617.2 (delta) variant	79
Advanced gene-editing strategy for epidermolysis bullosa simplex	77
A new immune pathway mediating AAV immune responses	75
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	74
Dual targeting of CD19 and CD22 against B-ALL using a novel high-sensitivity aCD22 CAR	74
miRNA discovery to therapy: The field is sufficiently mature to assess the value of miRNA-based therapeutics	74
In this issue	74
VCAM-targeting lipid platform brings new hope to the treatment of ischemic stroke	73
Anti-microRNA-378a Enhances Wound Healing Process by Upregulating Integrin Beta-3 and Vimentin	73
Novel insights into the ROCK-JAK-STAT signaling pathway in upper respiratory tract infections and neurodegenerative diseases	73
Large-scale falsification of research papers risks public trust in biomedical sciences	72
Immune responses to cells and proteins after hematopoietic stem cell gene therapy for inherited diseases: A cause for concern	69
Ligand-activated RXFP1 gene therapy ameliorates pressure overload-induced cardiac dysfunction	69
An armed oncolytic virus enhances the efficacy of tumor-infiltrating lymphocyte therapy by converting tumors to artificial antigen-presenting cells in situ	65
Single-cell data-driven design of armed oncolytic virus to boost cooperative innate-adaptive immunity against cancer	65
From cortex to medulla: Navigating the thymic landscape of T cell education	63
Immunotherapeutic HCW9218 augments anti-tumor activity of chemotherapy via NK cell-mediated reduction of therapy-induced senescent cells	63
Global challenges in preparedness and response to epidemic infectious diseases	63
Precision spinal gene delivery-induced functional switch in nociceptive neurons reverses neuropathic pain	63
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	63
Regulation of activated microglia and macrophages by systemically administered DNA/RNA heteroduplex oligonucleotides	61
Innovative stroke intervention: Harnessing cerebral dopamine neurotrophic factor	60
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	60
Evidence in primates supporting the use of chemogenetics for the treatment of human refractory neuropsychiatric disorders	59
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	59
Expanding CAR-engineered cell therapies into autoimmune diseases	59
An aptamer-mediated base editing platform for simultaneous knockin and multiple gene knockout for allogeneic CAR-T cells generation	58
The rational development of CD5-targeting biepitopic CARs with fully human heavy-chain-only antigen recognition domains	58
Efficient T cell adoptive transfer in lymphoreplete hosts mediated by transient activation of Stat5 signaling	58

Host liver-derived extracellular vesicles deliver miR-142a-3p induces neutrophil extracellular traps via targeting WASL to block the development of Schistosoma japonicum	58
Tubular-specific CDK12 knockout causes a defect in urine concentration due to premature cleavage of the slc12a1 gene	57
Targeting inflammatory macrophages rebuilds therapeutic efficacy of DOT1L inhibition in hepatocellular carcinoma	56
Targeting Lin28 axis enhances glypican-3-CAR T cell efficacy against hepatic tumor initiating cell population	56
Extracellular vesicle-based delivery of silencing sequences for the treatment of Machado-Joseph disease/spinocerebellar ataxia type 3	54
Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases	54
Beyond the unfolded protein response: Disclosing the role of XBP1s in Alzheimer’s disease	52
Emerging roles of the crosstalk between non-coding RNAs and m6A modification in cancers	52
In vivo selection of anti-HIV-1 gene-modified human hematopoietic stem/progenitor cells to enhance engraftment and HIV-1 inhibition	51
Development of a TNF-α-mediated Trojan Horse for bacteria-based cancer therapy	51
Allele-specific CRISPR-Cas9 editing of dominant epidermolysis bullosa simplex in human epidermal stem cells	51
Precise excision of HTLV-1 provirus with a designer-recombinase	51
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	50
In vivo genome editing shows promise for treating pulmonary diseases	50
Clarin-2 gene supplementation durably preserves hearing in a model of progressive hearing loss	50
Three recent breakthroughs in CAR T cells for the treatment of glioblastoma: Is it the light at the end of the tunnel?	49
Hitchhiking of Cas9 with nucleus-localized proteins impairs its controllability and leads to efficient genome editing of NLS-free Cas9	49
First in vivo base-editing trial shows promise	49
SOD2 promotes the immunosuppressive function of mesenchymal stem cells at the expense of adipocyte differentiation	49
Advances and transgressions of nuclear transport checkpoint inhibitors	49
Expanding strategies to resolve psoriasis-like inflammation: Non-canonical NF-κB signaling controls IL-23 production in dendritic cells	49
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	48
High-throughput screening identifies ibuprofen as an sEV PD-L1 inhibitor for synergistic cancer immunotherapy	47
Defining the activity of pro-reparative extracellular vesicles in wound healing based on miRNA payloads and cell type-specific lineage mapping	47
Ultrasound-mediated gene delivery specifically targets liver sinusoidal endothelial cells for sustained FVIII expression in hemophilia A mice	47
Mechanical force determines chimeric antigen receptor microclustering and signaling	47
Activation of the YAP/KLF5 transcriptional cascade in renal tubular cells aggravates kidney injury	47
Single-cell RNA sequencing reveals anti-tumor potency of CD56+ NK cells and CD8+ T cells in humanized mice via PD-1 and TIGIT co-targeting	46
Prevention of prostate cancer metastasis by a CRISPR-delivering nanoplatform for interleukin-30 genome editing	46
Blunting specific T-dependent antibody responses with engineered “decoy” B cells	46
AAV vector-derived elements integrate into Cas9-generated double-strand breaks and disrupt gene transcription	46
Mapping the cancer surface proteome in search of target antigens for immunotherapy	45
Clinical immunogenicity outcomes from GENEr8-1, a phase 3 study of valoctocogene roxaparvovec, an AAV5-vectored gene therapy for hemophilia A	45
Integrative proteomic and metabolomic elucidation of cardiomyopathy with in vivo and in vitro models and clinical samples	45
Discovery of a novel inhibitor of macropinocytosis with antiviral activity	45
Evaluation of [18F]fluoroestradiol and ChRERα as a gene expression PET reporter system in rhesus monkey brain	45
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	45
A functional RNA-origami as direct thrombin inhibitor with fast-acting and specific single-molecule reversal agents in vivo model	45
Immunosuppressant therapy averts rejection of allogeneic FKBP1A-disrupted CAR-T cells	45
A platform to deliver single and bi-specific Cas9/guide RNA to perturb genes in vitro and in vivo	44
CITED4 gene therapy: Can it be cited for preventing maladaptive post-ischemic cardiac remodeling?	44
The rise of cochlear gene therapy	44
The CD39+ HBV surface protein-targeted CAR-T and personalized tumor-reactive CD8+ T cells exhibit potent anti-HCC activity	43
Enhanced mucosal SARS-CoV-2 immunity after heterologous intramuscular mRNA prime/intranasal protein boost vaccination with a combination adjuvant	43
Microglial lnc-U90926 facilitates neutrophil infiltration in ischemic stroke via MDH2/CXCL2 axis	43
Longitudinal imaging of therapeutic enzyme expression after gene therapy for Fabry disease using positron emission tomography and the radiotracer [18F]AGAL	43
Promoting readthrough of nonsense mutations in CF mouse model: Biodistribution and efficacy of NV848 in rescuing CFTR protein expression	43
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	43
Long-term safety and efficacy of the fully human CAR-T therapy CT103A in relapsed/refractory multiple myeloma	43
mRNALocater: Enhance the prediction accuracy of eukaryotic mRNA subcellular localization by using model fusion strategy	42
Incorporation of bacterial immunoevasins to protect cell therapies from host antibody-mediated immune rejection	42
A chemokine regulatory loop induces cholesterol synthesis in lung-colonizing triple-negative breast cancer cells to fuel metastatic growth	42
Targeting of miR-33 ameliorates phenotypes linked to age-related macular degeneration	42
Targeting KIT by frameshifting mRNA transcripts as a therapeutic strategy for aggressive mast cell neoplasms	42
Gsx1 promotes locomotor functional recovery after spinal cord injury	42
Immunogenicity of a new gorilla adenovirus vaccine candidate for COVID-19	42
Immune profile and responses of a novel dengue DNA vaccine encoding an EDIII-NS1 consensus design based on Indo-African sequences	42
Sphingosine-1-phosphate transporter spinster homolog 2 is essential for iron-regulated metastasis of hepatocellular carcinoma	41
The antitumoral activity of TLR7 ligands is corrupted by the microenvironment of pancreatic tumors	41
Reply to In vivo confusion over in vivo conversion	41
Suppression of Fli-1 protects against pericyte loss and cognitive deficits in Alzheimer's disease	41
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	41
Exploiting the tumor-suppressive activity of the androgen receptor by CDK4/6 inhibition in castration-resistant prostate cancer	41
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	40
Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs	40
R-loops facilitate AAV-mediated nuclease-free gene targeting	40
Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials	40
The establishment of CDK9/RNA PolII/H3K4me3/DNA methylation feedback promotes HOTAIR expression by RNA elongation enhancement in cancer	40
Mesenchymal stromal cell delivery of oncolytic immunotherapy improves CAR-T cell antitumor activity	40
RNA therapeutics targeting PD-L1 is a promising immune-activation strategy against difficult-to-treat cancers	39
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	39
Corneal fibrosis abrogation by a localized AAV-mediated inhibitor of differentiation 3 (Id3) gene therapy in rabbit eyes in vivo	39
Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV	39
Valproic Acid Confers Functional Pluripotency to Human Amniotic Fluid Stem Cells in a Transgene-free Approach	39
Antisense-mediated exon skipping targeting EZH2 suppresses tumor growth in a xenograft mouse model of hepatocellular carcinoma	38
Apoptotic MSCs, COX2/PGE2 and clinical efficacy in Crohn fistula	38
Turning “trashed” genomic loci into treasurable sites for integrating chimeric antigen receptors in T and NK cells	38
Retraction Notice to: Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	38
AAVrh.10 delivery of novel APOE2-Christchurch variant suppresses amyloid and Tau pathology in Alzheimer’s disease mice	38
Collaborative science to advance gene therapies in resource-limited parts of the world	37

Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	37
circEHBP1 promotes lymphangiogenesis and lymphatic metastasis of bladder cancer via miR-130a-3p/TGFβR1/VEGF-D signaling	36
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	36
A broad and systematic approach to identify B cell malignancy-targeting TCRs for multi-antigen-based T cell therapy	36
m6A RNA hypermethylation-induced BACE2 boosts intracellular calcium release and accelerates tumorigenesis of ocular melanoma	36
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	36
Downregulation of a mitochondrial micropeptide, MPM, promotes hepatoma metastasis by enhancing mitochondrial complex I activity	36
Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1	35
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	35
Retraction Notice to: Precise editing of FGFR3-TACC3 fusion genes with CRISPR-Cas13a in glioblastoma	35
Evaluation of safety and early efficacy of AAV gene therapy in mouse models of vanishing white matter disease	34
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	34
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	34
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	34
Building a novel TRUCK by harnessing the endogenous IFN-gamma promoter for cytokine expression	34
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	33
Presidential Symposium and Presentation of Top Abstracts	33
Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson’s disease	33
miRNAs as neuro-oncologic therapeutics: A narrative review	33
FOSL1 promotes metastasis of head and neck squamous cell carcinoma through super-enhancer-driven transcription program	33
An optimized IgG-based B7-H3xCD3 bispecific antibody for treatment of gastrointestinal cancers	33
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	33
Imagine CRISPR cures	32
Locally secreted BiTEs complement CAR T cells by enhancing killing of antigen heterogeneous solid tumors	32
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	32
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	32
FTO promotes multiple myeloma progression by posttranscriptional activation of HSF1 in an m6A-YTHDF2-dependent manner	32
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	31
Dual neutralization of influenza virus hemagglutinin and neuraminidase by a bispecific antibody leads to improved antiviral activity	31
SOCS3 regulates pathological retinal angiogenesis through modulating SPP1 expression in microglia and macrophages	31
AAT-MSC-EVs: Novel implications for suppressing ferroptosis, fibrosis and pain associated with chronic pancreatitis	31
Prospects and challenges of tissue-derived extracellular vesicles	31
Balancing activation and co-stimulation of CAR tunes signaling dynamics and enhances therapeutic potency	30
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	30
Tissue nanotransfection causes tumor regression by its effect on nanovesicle cargo that alters microenvironmental macrophage state	30
Preclinical evaluation of ADVM-062, a novel intravitreal gene therapy vector for the treatment of blue cone monochromacy	30
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	30
CTLA-4 blockade induces tumor pyroptosis via CD8+ T cells in head and neck squamous cell carcinoma	30
Sirt6 protects retinal ganglion cells and optic nerve from degeneration during aging and glaucoma	29
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	29
Genetically engineered macrophages reverse the immunosuppressive tumor microenvironment and improve immunotherapeutic efficacy in TNBC	29
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	29
Small non-coding RNA YF1: A new dawn in heart transplantation	29
Biologics-based technologies for highly efficient and targeted RNA delivery	29
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	28
BCKDK modification enhances the anticancer efficacy of CAR-T cells by reprogramming branched chain amino acid metabolism	28
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	28
Successful large gene augmentation of USH2A with non-viral episomal vectors	28
CTLA-4: Checkpoints beyond the membrane	28
Extracellular vesicle-transmitted miR-671-5p alleviates lung inflammation and injury by regulating the AAK1/NF-κB axis	28
TLR9-independent CD8+ T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling	28
Atractylodinol prevents pulmonary fibrosis through inhibiting TGF-β receptor 1 recycling by stabilizing vimentin	28
Envelope protein-specific B cell receptors direct lentiviral vector tropism in vivo	28
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	27
Adoptive transfer of ex vivo expanded regulatory T cells improves immune cell engraftment and therapy-refractory chronic GvHD	27
Dual targeting of CD19 and CD22 against B-ALL using a novel high-sensitivity aCD22 CAR	27
Antisense oligonucleotide-based treatment of retinitis pigmentosa caused by USH2A exon 13 mutations	27
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	27
A self-amplifying mRNA SARS-CoV-2 vaccine candidate induces safe and robust protective immunity in preclinical models	27
An antisense circular RNA circSCRIB enhances cancer progression by suppressing parental gene splicing and translation	27
Immuno-oncology recapitulates ontogeny: Modern cell and gene therapy for cancer	27
Genetically modified ZIKA virus as a microRNA-sensitive oncolytic virus against central nervous system tumors	27
Armored bicistronic CAR T cells with dominant-negative TGF-β receptor II to overcome resistance in glioblastoma	26
CircRNAs regulate the crosstalk between inflammation and tumorigenesis: The bilateral association and molecular mechanisms	26
Treatment-induced hemophilic thrombosis?	26
The landscape of nanoparticle-based siRNA delivery and therapeutic development	26
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	26
miR-99b-5p, miR-380-3p, and miR-485-3p are novel chemosensitizing miRNAs in high-risk neuroblastoma	26
Stem cell-derived CAR T cells show greater persistence, trafficking, and viral control compared to ex vivo transduced CAR T cells	26
Leukemic extracellular vesicles induce chimeric antigen receptor T cell dysfunction in chronic lymphocytic leukemia	26
Inhibitory CARs fail to protect from immediate T cell cytotoxicity	26
XGBoost framework with feature selection for the prediction of RNA N5-methylcytosine sites	26
Targeting lipid nanoparticles to the blood-brain barrier to ameliorate acute ischemic stroke	26
Epigenetic strategies to boost CAR T cell therapy	26
FOSL1 promotes proneural-to-mesenchymal transition of glioblastoma stem cells via UBC9/CYLD/NF-κB axis	26
Human cochlear diffusion from the cerebrospinal fluid space with gadolinium contrast	25
Preclinical evidence in the assembly of mammalian SWI/SNF complexes: Epigenetic insights and clinical perspectives in human lung disease therapy	25
Regulatory approval of islet transplantation for treatment of type 1 diabetes: Implications and what is on the horizon	25
A first-in-human trial on the safety and immunogenicity of COVID-eVax, a cellular response-skewed DNA vaccine against COVID-19	25
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	25
Engineering of potent CAR NK cells using non-viral Sleeping Beauty transposition from minimalistic DNA vectors	25
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	25
A Precision Medicine Approach to Primary Immunodeficiency Disease: Ataluren Strikes Nonsense Mutations Once Again	25
PROTAC therapy as a new targeted therapy for lung cancer	25
Bulk and single-cell transcriptome profiling reveal the metabolic heterogeneity in human breast cancers	25
Therapeutic potential of RNA-enriched extracellular vesicles: The next generation in RNA delivery via biogenic nanoparticles	25
Targeting S100A8/A9-NCF1 axis in tumor microenvironment to prevent tumor metastasis by self-assembled peptide nanofibers	24
The high efficacy of claudin18.2-targeted CAR-T cell therapy in advanced pancreatic cancer with an antibody-dependent safety strategy	24
NKG2A genetic deletion promotes human primary NK cell anti-tumor responses better than an anti-NKG2A monoclonal antibody	24
WITHDRAWN: FOXC1 modulates stem-like cell properties and chemoresistance through hedgehog and EMT signaling in gastric adenocarcinoma	24
Role of exosomal non-coding RNAs from tumor cells and tumor-associated macrophages in the tumor microenvironment	24
The long road traveled in hematopoietic stem cell gene therapy	24
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	24
Therapeutic targeting of argininosuccinate synthase 1 (ASS1)-deficient pulmonary fibrosis	24
Cell-penetrating peptide-conjugated Morpholino rescues SMA in a symptomatic preclinical model	24