Molecular Therapy

Article	Citations
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	385
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	280
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	252
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	249
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	243
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	200
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	186
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	170
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	160
Response to: DNA transposon mechanisms and pathways of genotoxicity	159
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	152
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	150
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	143
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	143
From cortex to medulla: Navigating the thymic landscape of T cell education	136
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	136
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	135
Reply to In vivo confusion over in vivo conversion	135
Biologics-based technologies for highly efficient and targeted RNA delivery	133
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	125
Ad-justing macrophages for cancer immunotherapy	123
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	119
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	118
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	112
How to democratize cell and gene therapy: A global approach	111

Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	111
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	107
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	105
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	103
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	103
Promotion or inhibition? This is a question in gene editing	100
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	100
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	99
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	93
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	92
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	92
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	90
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	88
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	86
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	86
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	86
miRNAs as neuro-oncologic therapeutics: A narrative review	85
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	84
Recent progress and future challenges in structure-based protein-protein interaction prediction	84
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	84
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	84
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	82
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	77
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	76
Molecular Therapy Family Highlights	75
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	74
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface	73
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	73
Quo vadis American postdoc?	73
Response: Promise and open questions of optogenetic vision restoration by MCO	71
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells	71
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	69
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	69
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	68
Progress in skin gene therapy: From the inside and out	68
Late-Breaking Abstracts I	67
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension	65
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	64
Molecular Therapy’s growing influence	64
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	63
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	63
ASGCT 27th Annual Meeting Abstracts	63
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	63
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	62
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	61
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	61
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	61
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	61
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	60
AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders	59

In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	59
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	59
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	58
RNA m6A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of β-catenin	58
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	58
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	57
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	57
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	56
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	55
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	55
Managing allorejection in off-the-shelf CAR-engineered cell therapies	54
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	54
Transgene-Induced Cardiotoxicity In High-Dose AAV Gene Transfer	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
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	54
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	53
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	53
Fluoxetine ameliorates mucopolysaccharidosis type IIIA	53
Mucosal-associated invariant T cells for cancer immunotherapy	53
RNA editing: Expanding the potential of RNA therapeutics	53
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	52
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	52
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	51
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	50
In this issue	50
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	50
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	50
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	50
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	50
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	49
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	49
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	49
A new advanced stem cell-based embryonic model: The ultimate model?	49
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis	48
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	48
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	48
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	48
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	47
Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells	47
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	47
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	47
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	47
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	46
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	46
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	46
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	46
IL12 integrated into the CAR exodomain converts CD8+ T cells to poly-functional NK-like cells with superior killing of antigen-loss tumors	45
AAV-mediated DMPK silencing: A defining moment in myotonic dystrophy type 1 therapeutics	45
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	45
Computational structural optimization enhances IL13Rα2 – B7-H3 tandem CAR T cells to overcome antigen-heterogeneity-mediated tumor escape	45
Recent advances of engineered bacteria for therapeutic applications	43
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	43
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	43
GDF11: A promising new drug target for pulmonary fibrosis?	43
Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies	42
The curious case of AAV immunology	42
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	42
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus	42
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	42
Env-independent protection of intrarectal SIV challenge by vaccine induction of Gag/Vif-specific CD8+ T cells but not CD4+ T cells	42
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	42
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	42
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	41
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	41
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	40
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	40
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	40
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	40
In this issue	40
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	40
In this issue	40
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	40
Second gene therapy for hemophilia B approved: More answers or questions?	39
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors	39
Current RNA strategies in treating cardiovascular diseases	39
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	39
Cardiac reprogramming via chromatin remodeling by CRISPR activation	38
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	38
2022 ASGCT Annual Meeting Abstracts	38
Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells	38

Cleavage-free human genome editing	38
Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis	37
Harnessing engineered NK cells for refractory CD30+ lymphoma	37
From amputations to antibiotics: A future beyond “hacksaw” gene editing	37
CD19xCD3 T cell engager shows therapeutic potential for refractory myasthenic syndromes	37
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	37
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	37
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	37
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	36
Local magnetic delivery of adeno-associated virus AAV2(quad Y-F)-mediated BDNF gene therapy restores hearing after noise injury	36
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	36
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	36
Catalase, a therapeutic target in the reversal of estrogen-mediated aging	36
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	36
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	35
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	35
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	35
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	35
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	35
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	34
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	34
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	34
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	34
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	33
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	33
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	33
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein	33
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	33
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	33
In this issue	33
Far-red light-activated human islet-like designer cells enable sustained fine-tuned secretion of insulin for glucose control	33
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	33
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	32
Turning the tide in peritoneal metastases: Locoregional CAR-NK therapy primes systemic immunity in colorectal cancer	32
Repair Drive improves gene editing in the liver	32
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	32
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	32
Nucleic acid modifications in self-nonself discrimination	31
Targeted therapy for rare lung cancers: Status, challenges, and prospects	31
New hope for older SMA patients with next-generation self-complementary AAV gene therapy	31
AAV-mediated genome editing is influenced by the formation of R-loops	31
In this issue	30
In this issue	30
RAC1 directly phosphorylates both PKM2 and FBP1 to promote radioresistance in hepatocellular carcinoma	30
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	30
Dibenzazepine-Loaded Nanoparticles Induce Local Browning of White Adipose Tissue to Counteract Obesity	30
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	30
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	30
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	30
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	30
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	30
The 13th International Oncolytic Virus Conference: Powerful payloads gain clinical momentum	30
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	30
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	30
Successful immunotherapy using adenovector gene therapy for the treatment of recurrent respiratory papillomatosis	29
The future of gene therapy: Safer vectors, sharper focus	29
In utero genetic therapy: Treatment of early onset neurological disorders before they start	29
On-target/off-tumor toxicities following infusion of low-affinity Nectin-4-specific CAR T cells	29
Globin vector regulatory elements are active in early hematopoietic progenitor cells	29
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	29
Dual-antigen targeted iPSC-derived chimeric antigen receptor-T cell therapy for refractory lymphoma	29
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases	29
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	29
An optimized SpCas9 high-fidelity variant for direct protein delivery	29
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	29
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	29
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	28
Self-amplifying loop of NF-κB and periostin initiated by PIEZO1 accelerates mechano-induced senescence of nucleus pulposus cells and intervertebral disc degeneration	28
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	28
In this issue	28
COVID-19 mRNA vaccines: Platforms and current developments	28
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	28
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	28
Dichotomous effects of cellular expression of STAT3 on tumor growth of HNSCC	28
IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism	28
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	28
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	28
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	28
Extracellular vesicle-mediated communication between hepatocytes and natural killer cells promotes hepatocellular tumorigenesis	28
Celebrating 25 years of Molecular Therapy	28
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	28
Clinical perspectives: Treating spinal muscular atrophy	27
Extracellular vesicle-mediated delivery of CRISPR machinery silences androgen receptor in castration-resistant prostate cancer cells	27
Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9	27
Anti-Cas9 immunity: A formidable challenge for muscle genome editing	27
A sophisticated approach to targeting TERT activity in DLBCL	27
CRISPR-mediated MECOM depletion retards tumor growth by reducing cancer stem cell properties in lung squamous cell carcinoma	27
CircRNA: Unlocking new frontiers in therapeutic and vaccine development	27
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	27
Knockdown of calpain1 in lumbar motoneurons reduces spasticity after spinal cord injury in adult rats	27
Try before you buy: Empirical comparison of base editing approaches	27
Gene augmentation therapy attenuates retinal degeneration in a knockout mouse model of Fam161a retinitis pigmentosa	26
Visualizing lipid nanoparticle trafficking for mRNA vaccine delivery in non-human primates	26
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells	26
Targeting tissue-resident memory CD8+ T cells in the kidney is a potential therapeutic strategy to ameliorate podocyte injury and glomerulosclerosis	26