Molecular Therapy

Article	Citations
Response to: DNA transposon mechanisms and pathways of genotoxicity	522
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	349
From cortex to medulla: Navigating the thymic landscape of T cell education	304
Precision A3G base editors for disease modeling and correction	276
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	219
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	209
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	208
To make biology programmable, we must master its generative grammar	199
Promotion or inhibition? This is a question in gene editing	190
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	180
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	175
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	160
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	155
miRNAs as neuro-oncologic therapeutics: A narrative review	142
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	139
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT	138
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	136
Taming autoimmunity: Alpha-1 antitrypsin overexpressing mesenchymal stromal cells promote regulatory T cell crosstalk to reverse diabetes	133
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	132
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	132
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	121
Ad-justing macrophages for cancer immunotherapy	119
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	117
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	108
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	104

Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	101
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	101
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	98
Targeting of Aberrant αvβ6 Integrin Expression in Solid Tumors Using Chimeric Antigen Receptor-Engineered T Cells	96
Programmable miRNA-guided RNA-toxin switch for selective elimination of cancer cells	95
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	94
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	92
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	92
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	84
C9orf72-associated poly-GR in skeletal muscle leads to neuromuscular junction deficits and muscle atrophy	83
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	82
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	81
How to democratize cell and gene therapy: A global approach	80
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	80
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	80
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	79
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	78
Biologics-based technologies for highly efficient and targeted RNA delivery	76
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	75
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	75
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	74
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	73
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	73
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	72
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	72
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	72
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	72
Recent progress and future challenges in structure-based protein-protein interaction prediction	71
Plugging the diastolic calcium leak: MYBPC3 fragment for CPVT gene therapy	71
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	70
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	68
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	67
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	66
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	66
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	65
A safer path to gene correction in junctional epidermolysis bullosa	64
Inflammatory mediators of mRNA vaccine-induced adverse reactions in mice	64
Late-Breaking Abstracts I	64
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	64
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	64
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	63
Molecular Therapy Family Highlights	63
Quo vadis American postdoc?	62
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	62
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	61
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	60
Molecular Therapy’s growing influence	60
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	60
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	59
Progress in skin gene therapy: From the inside and out	58

In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	58
The phosphatase DUSP2 constrains lymphoid remodeling and immunotherapy response in lung squamous carcinoma	57
Response: Promise and open questions of optogenetic vision restoration by MCO	57
Promises of co-stimulatory inhibition for durable and repeatable AAV gene therapy	57
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	57
Antibody format matters: A comparative analysis of VHH and scFv domains reveals superior in vivo CAR T cell function with VHH domains	57
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	57
Dual AAV gene therapy using laminin-linking proteins ameliorates muscle and nerve defects in LAMA2-related muscular dystrophy	56
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	56
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	56
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	54
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	53
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	53
ASGCT 27th Annual Meeting Abstracts	53
AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders	52
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	52
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	52
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	52
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	52
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	52
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	51
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	51
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	50
Managing allorejection in off-the-shelf CAR-engineered cell therapies	50
Focused ultrasound as a delivery platform for CNS AAV gene therapy	50
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	50
Toward scalable helper T cells from iPSCs	50
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	49
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	49
Transgene-induced cardiotoxicity in high-dose AAV gene transfer	49
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	48
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	48
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	48
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	48
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	48
Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapy	48
Mucosal-associated invariant T cells for cancer immunotherapy	47
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	47
RNA editing: Expanding the potential of RNA therapeutics	46
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	46
Tissue-Specific Silencing of Synthetic mRNAs By De-Targeting Elements Maps Vaccination-Competent Tissues, and Allows Cas9 De-Immunization	46
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies	46
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells	46
Defining therapeutic thresholds for STXBP1 gene therapy: From gene delivery to network rescue in neurodevelopmental disorders	45
Nicotinamide riboside enhances adoptive T cell therapy by promoting memory differentiation and metabolic fitness	45
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	45
AAVrh32.33 capsid demonstrates unexpected dermal tropism regardless of immunodominant epitope	45
Closing the loop: An RNA-to-RNA pipeline for on-demand antiviral antibodies	45
In this issue	45
GDF11: A promising new drug target for pulmonary fibrosis?	45
A new advanced stem cell-based embryonic model: The ultimate model?	44
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	43
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	43
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	43
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	43
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	43
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	43
Retraction Notice to: Smad7 suppresses renal fibrosis via altering expression of TGFβ/Smad3-regulated microRNAs	42
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	42
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	42
Host natural IgM shapes systemic efficacy of the oncolytic adenovirus	42
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	42
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	42
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	42
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	42
AAV-mediated DMPK silencing: A defining moment in myotonic dystrophy type 1 therapeutics	42
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	41
HSP90α-USP7-DNMT1 axis drives HCC recurrence after microwave ablation by disrupting ACSS3-mediated propionate metabolism	41
Igniting CAR-NKT cells with IL-18	40
Computational structural optimization enhances IL13Rα2 – B7-H3 tandem CAR T cells to overcome antigen-heterogeneity-mediated tumor escape	40
From silence to signal: Redirecting apoptotic cell clearance to fuel antitumor T cell immunity	40
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	40
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	40
Recent advances of engineered bacteria for therapeutic applications	39
The curious case of AAV immunology	39
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	38
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	38
From amputations to antibiotics: A future beyond “hacksaw” gene editing	38
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	38
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	38

Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	38
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	38
In this issue	38
In this issue	37
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	37
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	37
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	37
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	37
A new era for myotonic dystrophy: Improved delivery of a DMPK-targeted oligonucleotide modulates muscle transcriptomes and function	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
Harnessing engineered NK cells for refractory CD30+ lymphoma	36
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	36
Second gene therapy for hemophilia B approved: More answers or questions?	36
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	35
Endothelial injury with capillary leak: A final common pathway in acute AAV toxicity?	35
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	35
Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME	35
Myeloid-lineage CAR knockin mice enable allogeneic immunotherapy for liver and lung fibrosis	35
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	35
CD19xCD3 T cell engager shows therapeutic potential for refractory myasthenic syndromes	35
Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells	35
FGF21 gene therapy for healthy aging: Great promise, important questions	35
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	34
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	34
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	34
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	34
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	34
Nicotinamide riboside tunes adoptive T cells for memory and metabolic fitness	34
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	34
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	33
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	33
In vivo-directed evolution identifies AAV-WM04 as a next-generation vector for potent and sustained hearing restoration in DFNB9	33
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	33
Recent progress in the rational design of mRNA vaccines	32
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	32
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	32
In this issue	32
Nucleic acid modifications in self-nonself discrimination	32
Triple AAV Intein-Mediated Gene Therapy Ameliorates Dystrophic Phenotype in MDC1A Mice	32
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	32
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	32
Discovery, preclinical safety, and efficacy characterization of SMAC mimetic S-016-1348 as a potential cancer therapeutic	32
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	32
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	32
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	32
Current RNA strategies in treating cardiovascular diseases	32
Successful immunotherapy using adenovector gene therapy for the treatment of recurrent respiratory papillomatosis	32
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein	32
Targeting emerging respiratory pathogens with clamp peptides: Broad-spectrum inhibition of viral entry in 3D human lung models	31
Turning the tide in peritoneal metastases: Locoregional CAR-NK therapy primes systemic immunity in colorectal cancer	31
Repair Drive improves gene editing in the liver	31
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	31
Phase 1 study of autologous T cells bearing fully human chimeric antigen receptors targeting mesothelin in mesothelin-expressing cancers	31
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	31
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	31
Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial	31
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	31
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	31
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	30
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	30
The future of gene therapy: Safer vectors, sharper focus	30
Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy	30
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	30
In this issue	30
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	30
New hope for older SMA patients with next-generation self-complementary AAV gene therapy	30
Focused CRISPR screening to design fit-for-purpose CAR T cell therapies	30
Promoting donor microglial replacement through augmented conditioning or radiation sensitivity	29
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	29
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	29
Targeted therapy for rare lung cancers: Status, challenges, and prospects	29
Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates	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
Treatment with the Nox1/4 inhibitor Setanaxib ameliorates cardiac function in mouse models of Duchenne muscular dystrophy	29
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	29
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	28
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases	28
On-target/off-tumor toxicities following infusion of low-affinity Nectin-4-specific CAR T cells	28
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	28
Evaluating cases of possible insertional mutagenesis after AAV gene therapy	28
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	28
RAC1 directly phosphorylates both PKM2 and FBP1 to promote radioresistance in hepatocellular carcinoma	28
In utero genetic therapy: Treatment of early onset neurological disorders before they start	28
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	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
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	27
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy	27
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	27
In this issue	27
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	27
AAV-mediated genome editing is influenced by the formation of R-loops	27
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	27
Try before you buy: Empirical comparison of base editing approaches	27