Molecular Therapy

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	306
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	238
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	219
Promotion or inhibition? This is a question in gene editing	215
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	206
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	192
From cortex to medulla: Navigating the thymic landscape of T cell education	176
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	170
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	161
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	149
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	148
Reply to In vivo confusion over in vivo conversion	147
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	141
Response to: DNA transposon mechanisms and pathways of genotoxicity	138
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	130
Biologics-based technologies for highly efficient and targeted RNA delivery	129
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	129
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	128
How to democratize cell and gene therapy: A global approach	126
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	126
Recent progress and future challenges in structure-based protein-protein interaction prediction	124
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	121
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	120
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	108
Ad-justing macrophages for cancer immunotherapy	102

Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	101
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	101
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	100
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	100
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	99
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	99
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	97
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	97
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	92
miRNAs as neuro-oncologic therapeutics: A narrative review	91
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	91
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	87
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	86
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	85
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	85
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	85
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	84
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	84
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	84
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	83
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	78
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	76
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	76
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	74
Late-Breaking Abstracts I	73
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	72
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	71
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	70
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	70
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	69
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	69
Quo vadis American postdoc?	68
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	68
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	68
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	67
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	65
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	65
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	64
Molecular Therapy’s growing influence	64
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	64
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	63
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	62
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	62
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	61
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	61
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	60
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	59
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	58
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	56
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	55

Response: Promise and open questions of optogenetic vision restoration by MCO	55
Managing allorejection in off-the-shelf CAR-engineered cell therapies	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	54
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	54
RETRACTED: Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	54
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	53
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension	53
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	53
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface	53
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	52
Molecular Therapy Family Highlights	52
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	52
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	52
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	52
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	52
RNA m6A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of β-catenin	52
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	51
ASGCT 27th Annual Meeting Abstracts	51
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	51
Progress in skin gene therapy: From the inside and out	51
RNA editing: Expanding the potential of RNA therapeutics	51
Fluoxetine ameliorates mucopolysaccharidosis type IIIA	51
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	50
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	50
Mucosal-associated invariant T cells for cancer immunotherapy	49
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	48
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	47
In this issue	47
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	47
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	47
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	47
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	47
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	46
A new advanced stem cell-based embryonic model: The ultimate model?	46
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	46
Env-independent protection of intrarectal SIV challenge by vaccine induction of Gag/Vif-specific CD8+ T cells but not CD4+ T cells	46
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	45
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	45
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	45
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	44
Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy	44
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	44
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	44
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	43
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	43
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	43
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	43
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	43
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines	43
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	42
Computational structural optimization enhances IL13Rα2 – B7-H3 tandem CAR T cells to overcome antigen-heterogeneity-mediated tumor escape	42
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	42
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	41
IL12 integrated into the CAR exodomain converts CD8+ T cells to poly-functional NK-like cells with superior killing of antigen-loss tumors	41
Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells	41
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis	41
Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies	40
The curious case of AAV immunology	40
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	40
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	40
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	39
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	39
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	39
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	39
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus	39
Cardiac reprogramming via chromatin remodeling by CRISPR activation	38
In this issue	37
circRNA_0025202 Regulates Tamoxifen Sensitivity and Tumor Progression via Regulating the miR-182-5p/FOXO3a Axis in Breast Cancer	37
BETting on BRD4 inhibition to combat adaptive resistance to CAR T cell therapy in glioblastoma	37
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	37
2022 ASGCT Annual Meeting Abstracts	37
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	36
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	36
Live-attenuated RNA hybrid vaccine technology provides single-dose protection against Chikungunya virus	36
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	36
Harnessing engineered NK cells for refractory CD30+ lymphoma	36
In this issue	36
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	36
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	36
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	36

Immunomodulatory effect of locoregional therapy in the tumor microenvironment	35
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	35
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	35
Cleavage-free human genome editing	35
Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis	35
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors	35
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	35
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	35
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	34
Second gene therapy for hemophilia B approved: More answers or questions?	34
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	34
From amputations to antibiotics: A future beyond “hacksaw” gene editing	34
Catalase, a therapeutic target in the reversal of estrogen-mediated aging	34
CD19xCD3 T cell engager shows therapeutic potential for refractory myasthenic syndromes	33
Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells	33
Current RNA strategies in treating cardiovascular diseases	33
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	33
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	32
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	32
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	32
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	32
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	32
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	32
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	32
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	32
Local magnetic delivery of adeno-associated virus AAV2(quad Y-F)-mediated BDNF gene therapy restores hearing after noise injury	32
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	31
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	31
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	31
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	31
In this issue	31
In this issue	31
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	31
The 13th International Oncolytic Virus Conference: Powerful payloads gain clinical momentum	31
In this issue	30
Repair Drive improves gene editing in the liver	30
An optimized SpCas9 high-fidelity variant for direct protein delivery	30
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	30
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	30
Far-red light-activated human islet-like designer cells enable sustained fine-tuned secretion of insulin for glucose control	30
AAV-mediated genome editing is influenced by the formation of R-loops	30
Nucleic acid modifications in self-nonself discrimination	30
In utero genetic therapy: Treatment of early onset neurological disorders before they start	30
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	30
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	30
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein	30
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	29
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	29
Dibenzazepine-Loaded Nanoparticles Induce Local Browning of White Adipose Tissue to Counteract Obesity	29
Extracellular vesicle-mediated communication between hepatocytes and natural killer cells promotes hepatocellular tumorigenesis	29
Globin vector regulatory elements are active in early hematopoietic progenitor cells	29
Targeted therapy for rare lung cancers: Status, challenges, and prospects	29
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	29
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	29
Dual-antigen targeted iPSC-derived chimeric antigen receptor-T cell therapy for refractory lymphoma	29
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	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
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	28
Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies	28
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	28
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	28
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	27
COVID-19 mRNA vaccines: Platforms and current developments	27
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	27
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	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
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	27
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	27
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	27
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	27
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	27
In this issue	26
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells	26
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	26
In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment	26
In this issue	26
Critical role of TPRN rings in the stereocilia for hearing	26
Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome	26
Gene therapy then and now: A look back at changes in the field over the past 25 years	26
Highly conserved brain vascular receptor ALPL mediates transport of engineered AAV vectors across the blood-brain barrier	26
Knockdown of calpain1 in lumbar motoneurons reduces spasticity after spinal cord injury in adult rats	26
Osteoarthritis gene therapy: Expanding the scope of genetic therapies	26
IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism	26
Targeting tissue-resident memory CD8+ T cells in the kidney is a potential therapeutic strategy to ameliorate podocyte injury and glomerulosclerosis	26
Advances in stem cell and cellular therapeutics from the 9th Midwest Conference on Cell Therapy and Regenerative Medicine	26
M2 macrophages secrete glutamate-containing extracellular vesicles to alleviate osteoporosis by reshaping osteoclast precursor fate	26
Celebrating 25 years of Molecular Therapy	26
Clinical perspectives: Treating spinal muscular atrophy	26
Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction	25
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	25
Reduction of circulating IgE and allergens by a pH-sensitive antibody with enhanced FcγRIIb binding	25
Gene augmentation therapy attenuates retinal degeneration in a knockout mouse model of Fam161a retinitis pigmentosa	25
Try before you buy: Empirical comparison of base editing approaches	25
DNA-based immunotherapy for cancer: In vivo approaches for recalcitrant targets	25