Molecular Therapy

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	289
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	228
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	210
Promotion or inhibition? This is a question in gene editing	205
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	201
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	185
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	182
Recent progress and future challenges in structure-based protein-protein interaction prediction	170
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	161
Response to: DNA transposon mechanisms and pathways of genotoxicity	155
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	147
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	146
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	145
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	138
From cortex to medulla: Navigating the thymic landscape of T cell education	131
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	129
Reply to In vivo confusion over in vivo conversion	124
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	124
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	124
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	124
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	122
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	121
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	118
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	115
miRNAs as neuro-oncologic therapeutics: A narrative review	112

Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	107
Ad-justing macrophages for cancer immunotherapy	100
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	100
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	99
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	97
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	96
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	96
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	95
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	95
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	94
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	93
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	91
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	90
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	86
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	84
How to democratize cell and gene therapy: A global approach	84
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	83
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	80
Biologics-based technologies for highly efficient and targeted RNA delivery	80
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	79
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	79
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	77
Late-Breaking Abstracts I	74
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	74
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	73
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	72
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	70
Quo vadis American postdoc?	69
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	69
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	69
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	68
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	68
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	68
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	68
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	68
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	67
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	67
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	65
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	65
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension	64
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	63
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface	63
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	62
Response: Promise and open questions of optogenetic vision restoration by MCO	61
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	61
Molecular Therapy’s growing influence	60
Molecular Therapy Family Highlights	60
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	59
ASGCT 27th Annual Meeting Abstracts	59
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	59

Fluoxetine ameliorates mucopolysaccharidosis type IIIA	58
Managing allorejection in off-the-shelf CAR-engineered cell therapies	54
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	54
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	54
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	53
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	53
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	53
Progress in skin gene therapy: From the inside and out	52
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	52
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	52
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	52
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe – first phase I/II human clinical study	52
RNA editing: Expanding the potential of RNA therapeutics	52
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	52
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	51
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	51
Mucosal-associated invariant T cells for cancer immunotherapy	50
RNA m6A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of β-catenin	50
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	50
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	49
RETRACTED: Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	49
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	49
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	49
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	49
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	48
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	48
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis	48
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	48
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	48
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	48
Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells	46
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	46
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	46
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus	46
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	45
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	45
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	45
In this issue	45
A new advanced stem cell-based embryonic model: The ultimate model?	45
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	45
Env-independent protection of intrarectal SIV challenge by vaccine induction of Gag/Vif-specific CD8+ T cells but not CD4+ T cells	44
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	44
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	44
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	44
Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies	44
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	44
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	43
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	43
Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy	43
The curious case of AAV immunology	43
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	43
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	42
IL12 integrated into the CAR exodomain converts CD8+ T cells to poly-functional NK-like cells with superior killing of antigen-loss tumors	42
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	41
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	41
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	41
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines	40
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	40
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	40
Call for papers: A special issue on tackling emerging infectious diseases	39
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	39
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	39
Cardiac reprogramming via chromatin remodeling by CRISPR activation	39
circRNA_0025202 Regulates Tamoxifen Sensitivity and Tumor Progression via Regulating the miR-182-5p/FOXO3a Axis in Breast Cancer	39
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	39
In this issue	38
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors	38
BETting on BRD4 inhibition to combat adaptive resistance to CAR T cell therapy in glioblastoma	38
2022 ASGCT Annual Meeting Abstracts	38
Harnessing engineered NK cells for refractory CD30+ lymphoma	38
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	37
In this issue	37
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	37
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	37
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	37
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	36
Live-attenuated RNA hybrid vaccine technology provides single-dose protection against Chikungunya virus	36
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	36
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	36
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	35

From amputations to antibiotics: A future beyond “hacksaw” gene editing	35
Cleavage-free human genome editing	35
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	35
Catalase, a therapeutic target in the reversal of estrogen-mediated aging	34
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	34
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	34
Second gene therapy for hemophilia B approved: More answers or questions?	34
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	34
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	34
Local magnetic delivery of adeno-associated virus AAV2(quad Y-F)-mediated BDNF gene therapy restores hearing after noise injury	34
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	34
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	33
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	33
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	33
Current RNA strategies in treating cardiovascular diseases	33
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	33
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	32
In this issue	32
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	32
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	32
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	32
Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis	32
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	32
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	32
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	32
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	31
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	31
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	31
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	31
Extracellular vesicle-mediated communication between hepatocytes and natural killer cells promotes hepatocellular tumorigenesis	31
In this issue	31
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	30
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	30
In utero genetic therapy: Treatment of early onset neurological disorders before they start	30
The 13th International Oncolytic Virus Conference: Powerful payloads gain clinical momentum	30
Repair Drive improves gene editing in the liver	30
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	30
In this issue	30
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	30
Nucleic acid modifications in self-nonself discrimination	29
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein	29
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	29
Far-red light-activated human islet-like designer cells enable sustained fine-tuned secretion of insulin for glucose control	29
An optimized SpCas9 high-fidelity variant for direct protein delivery	29
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	29
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	29
Dibenzazepine-Loaded Nanoparticles Induce Local Browning of White Adipose Tissue to Counteract Obesity	29
AAV-mediated genome editing is influenced by the formation of R-loops	29
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	29
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	29
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	28
Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies	28
Retraction Notice to: Lentivirus Mediated Delivery of Neurosin Promotes Clearance of Wild-type α-Synuclein and Reduces the Pathology in an α-Synuclein Model of LBD	28
Dichotomous effects of cellular expression of STAT3 on tumor growth of HNSCC	28
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	28
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	28
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	28
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	28
Dual-antigen targeted iPSC-derived chimeric antigen receptor-T cell therapy for refractory lymphoma	27
Targeted therapy for rare lung cancers: Status, challenges, and prospects	27
Self-amplifying loop of NF-κB and periostin initiated by PIEZO1 accelerates mechano-induced senescence of nucleus pulposus cells and intervertebral disc degeneration	27
COVID-19 mRNA vaccines: Platforms and current developments	27
Globin vector regulatory elements are active in early hematopoietic progenitor cells	27
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	27
In this issue	27
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	27
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	27
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	27
Critical role of TPRN rings in the stereocilia for hearing	27
Reduction of circulating IgE and allergens by a pH-sensitive antibody with enhanced FcγRIIb binding	26
Celebrating 25 years of Molecular Therapy	26
In this issue	26
A BPTF-specific PROTAC degrader enhances NK cell-based cancer immunotherapy	26
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant	26
Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier	26
Immunological responses and clinical outcomes in dogs with osteosarcoma receiving standard therapy and a Listeria vaccine expressing HER2	26
Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina	26
Knockdown of calpain1 in lumbar motoneurons reduces spasticity after spinal cord injury in adult rats	26
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	26
IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism	26
Clinical perspectives: Treating spinal muscular atrophy	26
Gene therapy then and now: A look back at changes in the field over the past 25 years	26
Try before you buy: Empirical comparison of base editing approaches	26
Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome	26
Visualizing lipid nanoparticle trafficking for mRNA vaccine delivery in non-human primates	25
Gene augmentation therapy attenuates retinal degeneration in a knockout mouse model of Fam161a retinitis pigmentosa	25
Genome editing in large animal models	25
In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment	25
Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder	25
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	25
Genetic and pharmacological inhibition of GRPR protects against acute kidney injury via attenuating renal inflammation and necroptosis	25
DNA-based immunotherapy for cancer: In vivo approaches for recalcitrant targets	25
Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction	25
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy	25
Carrier-free mRNA vaccine induces robust immunity against SARS-CoV-2 in mice and non-human primates without systemic reactogenicity	25