Molecular Therapy

Article	Citations
Ad-justing macrophages for cancer immunotherapy	482
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	328
Response to: DNA transposon mechanisms and pathways of genotoxicity	291
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	253
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	195
From cortex to medulla: Navigating the thymic landscape of T cell education	195
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	184
Precision A3G base editors for disease modeling and correction	183
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	183
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	167
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	160
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	155
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	151
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	135
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	134
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	130
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	123
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	123
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	121
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	121
To make biology programmable, we must master its generative grammar	121
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	115
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	115
Taming autoimmunity: Alpha-1 antitrypsin overexpressing mesenchymal stromal cells promote regulatory T cell crosstalk to reverse diabetes	113
Promotion or inhibition? This is a question in gene editing	108

Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	107
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	104
Biologics-based technologies for highly efficient and targeted RNA delivery	100
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	100
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	98
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	91
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	90
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well tolerated and generates sustained neurodevelopment without HSCT	88
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	85
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	85
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	83
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	82
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	82
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	80
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	79
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	78
miRNAs as neuro-oncologic therapeutics: A narrative review	78
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	78
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	76
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	73
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	73
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	73
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	70
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	70
Recent progress and future challenges in structure-based protein-protein interaction prediction	69
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	69
How to democratize cell and gene therapy: A global approach	69
Programmable miRNA-Guided RNA-Toxin Switch for Selective Elimination of Cancer Cells	69
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	69
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	69
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	68
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	67
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	66
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	66
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	65
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells	64
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	64
Late-Breaking Abstracts I	64
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	64
A safer path to gene correction in junctional epidermolysis bullosa	63
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	62
Transgene-induced cardiotoxicity in high-dose AAV gene transfer	61
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	60
Inflammatory mediators of mRNA vaccine-induced adverse reactions in mice	60
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	60
Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapy	60
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	60
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies	60
Toward scalable helper T cells from iPSCs	59
Response: Promise and open questions of optogenetic vision restoration by MCO	58

Focused ultrasound as a delivery platform for CNS AAV gene therapy	58
Molecular Therapy Family Highlights	57
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	56
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	55
Quo vadis American postdoc?	55
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	55
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	55
ASGCT 27th Annual Meeting Abstracts	54
Molecular Therapy’s growing influence	54
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	53
Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	53
Mucosal-associated invariant T cells for cancer immunotherapy	52
AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders	52
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	52
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	51
Managing allorejection in off-the-shelf CAR-engineered cell therapies	51
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	51
RNA editing: Expanding the potential of RNA therapeutics	51
The phosphatase DUSP2 constrains lymphoid remodeling and immunotherapy response in lung squamous carcinoma	50
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	50
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	50
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	50
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	50
Dual AAV gene therapy using laminin-linking proteins ameliorates muscle and nerve defects in LAMA2-related muscular dystrophy	49
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	49
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	49
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	48
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	47
Progress in skin gene therapy: From the inside and out	47
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	47
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	47
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	47
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	46
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	46
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	45
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	45
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	45
In this issue	45
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	45
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	44
Igniting CAR-NKT cells with IL-18	44
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	44
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	44
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	44
Recent advances of engineered bacteria for therapeutic applications	43
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	43
GDF11: A promising new drug target for pulmonary fibrosis?	43
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	43
A new advanced stem cell-based embryonic model: The ultimate model?	43
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	43
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	43
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	43
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	42
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	42
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	42
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	41
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	41
The curious case of AAV immunology	41
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	41
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	41
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	40
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	40
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	40
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	40
Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells	39
AAVrh32.33 capsid demonstrates unexpected dermal tropism regardless of immunodominant epitope	39
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	39
Retraction Notice to: Smad7 suppresses renal fibrosis via altering expression of TGFβ/Smad3-regulated microRNAs	39
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	39
Host natural IgM shapes systemic efficacy of the oncolytic adenovirus	39
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	38
AAV-mediated DMPK silencing: A defining moment in myotonic dystrophy type 1 therapeutics	38
Computational structural optimization enhances IL13Rα2 – B7-H3 tandem CAR T cells to overcome antigen-heterogeneity-mediated tumor escape	38
CD19xCD3 T cell engager shows therapeutic potential for refractory myasthenic syndromes	38
Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells	38
From amputations to antibiotics: A future beyond “hacksaw” gene editing	38
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	37
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	37
In this issue	37
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	37

A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	36
In this issue	36
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	36
Harnessing engineered NK cells for refractory CD30+ lymphoma	36
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	36
Myeloid-lineage CAR knockin mice enable allogeneic immunotherapy for liver and lung fibrosis	36
Second gene therapy for hemophilia B approved: More answers or questions?	35
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	35
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	35
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	35
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	34
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	34
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	34
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	34
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	34
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	34
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	34
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	34
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	34
Death following high-dose AAV9 gene therapy in a patient with advanced SMA-PME	33
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	33
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	33
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	33
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	33
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	33
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	32
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	32
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	32
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	32
In this issue	32
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	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
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	32
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	32
Current RNA strategies in treating cardiovascular diseases	32
In this issue	32
On-target/off-tumor toxicities following infusion of low-affinity Nectin-4-specific CAR T cells	32
Targeted therapy for rare lung cancers: Status, challenges, and prospects	31
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	31
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	31
Turning the tide in peritoneal metastases: Locoregional CAR-NK therapy primes systemic immunity in colorectal cancer	31
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	31
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	31
New hope for older SMA patients with next-generation self-complementary AAV gene therapy	31
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	31
Discovery, preclinical safety, and efficacy characterization of SMAC mimetic S-016-1348 as a potential cancer therapeutic	31
In vitro and in vivo validation of the antiviral effect of hCypA against SARS-CoV-2 via binding to the RBD of spike protein	31
AAV-mediated genome editing is influenced by the formation of R-loops	30
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	30
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	30
Globin vector regulatory elements are active in early hematopoietic progenitor cells	30
Nucleic acid modifications in self-nonself discrimination	30
Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial	30
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	30
Phase 1 study of autologous T cells bearing fully human chimeric antigen receptors targeting mesothelin in mesothelin-expressing cancers	29
Promoting donor microglial replacement through augmented conditioning or radiation sensitivity	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
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	29
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases	29
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	29
An optimized SpCas9 high-fidelity variant for direct protein delivery	29
Repair Drive improves gene editing in the liver	29
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	29
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	29
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	29
The future of gene therapy: Safer vectors, sharper focus	29
Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy	29
RAC1 directly phosphorylates both PKM2 and FBP1 to promote radioresistance in hepatocellular carcinoma	29
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	29
Successful immunotherapy using adenovector gene therapy for the treatment of recurrent respiratory papillomatosis	29
Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates	29
In utero genetic therapy: Treatment of early onset neurological disorders before they start	28
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	28
IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism	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
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	28
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	28
DNA-based immunotherapy for cancer: In vivo approaches for recalcitrant targets	28
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	28
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	28
A sophisticated approach to targeting TERT activity in DLBCL	28
In this issue	28
Critical role of TPRN rings in the stereocilia for hearing	27
Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina	27
Immunological responses and clinical outcomes in dogs with osteosarcoma receiving standard therapy and a Listeria vaccine expressing HER2	27
Advances in stem cell and cellular therapeutics from the 9th Midwest Conference on Cell Therapy and Regenerative Medicine	27
CircRNA: Unlocking new frontiers in therapeutic and vaccine development	27
In this issue	27
Knockdown of calpain1 in lumbar motoneurons reduces spasticity after spinal cord injury in adult rats	27
Osteoarthritis gene therapy: Expanding the scope of genetic therapies	27
Try before you buy: Empirical comparison of base editing approaches	27
A BPTF-specific PROTAC degrader enhances NK cell-based cancer immunotherapy	26
Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model	26
Reduction of circulating IgE and allergens by a pH-sensitive antibody with enhanced FcγRIIb binding	26