Molecular Therapy

Article	Citations
Ad-justing macrophages for cancer immunotherapy	468
RNA interference targeting lipoprotein(a): A “big hit” against “little a”?	322
Response to: DNA transposon mechanisms and pathways of genotoxicity	288
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	281
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	269
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	248
From cortex to medulla: Navigating the thymic landscape of T cell education	188
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	182
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	182
Exploiting viral infection/vaccination to focus high-affinity T cell populations into tumors using oncolytic viro-immunotherapy	180
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	170
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	164
Precision A3G base editors for disease modeling and correction	156
A synthetic hyperglycemia-sensing gene circuit enhances blood glucose homeostasis in diabetic mice	150
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	148
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	133
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	132
Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	127
Ablation of dysmorphic neurons is a safe and effective treatment for focal cortical dysplasia II	120
ALCAT1 promotes diabetic cardiomyopathy by linking myocardial tetralinoleoyl cardiolipin deficiency to lipotoxicity	119
JMJD3 and UTX as key targets for gene-modified mesenchymal stem cell therapy in cartilage tissue engineering	118
Inhibition of NAD-GPx4 axis and MEK triggers ferroptosis to suppress pancreatic ductal adenocarcinoma	113
CAR-modified marrow infiltrating lymphocytes efficiently target malignant plasma cells with very low antigen density	112
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	112
Promotion or inhibition? This is a question in gene editing	111

Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	105
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	105
Reduced-intensity conditioning in LV-mediated gene therapy for Fabry disease targeting HSPCs	102
Targeting EBV gp42 for nasopharyngeal carcinoma prevention	98
Biologics-based technologies for highly efficient and targeted RNA delivery	98
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCT	97
To make biology programmable, we must master its generative grammar	96
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	90
Taming Autoimmunity: Alpha-1 Antitrypsin Overexpressing Mesenchymal Stem/Stromal Cells Promote Regulatory T Cell Crosstalk to Reverse Diabetes	87
CDK4/6 inhibition enhances CAR-T cell therapy in solid tumors	84
Structural basis of liver de-targeting and neuronal tropism of CNS-targeted AAV capsids	84
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	82
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	81
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	80
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	79
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	78
Recent progress and future challenges in structure-based protein-protein interaction prediction	77
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	77
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	77
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	75
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	75
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	73
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	71
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	69
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	69
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	69
How to democratize cell and gene therapy: A global approach	69
Molecular Therapy Family Highlights	67
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	67
miRNAs as neuro-oncologic therapeutics: A narrative review	67
Targeting of p53-Transcriptional Dysfunction by Conditionally Replicating Adenovirus Is Not Limited by p53-Homologues	65
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	65
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	64
Toward scalable helper T cells from iPSCs	64
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	64
Optogenetic vision restoration in the face of secondary and tertiary remodeling in the rd1 mouse retina	64
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	63
Development of an AAV-delivered microRNA gene therapy for myotonic dystrophy type 1	63
The phosphatase DUSP2 constrains lymphoid remodeling and immunotherapy response in lung squamous carcinoma	62
Smoking and tetramer tryptase accelerate intervertebral disc degeneration by inducing METTL14-mediated DIXDC1 m6 modification	62
Targeted long-read sequencing captures CRISPR editing and AAV integration outcomes in brain	62
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	62
FLT201, a novel liver-directed AAV gene therapy candidate for Gaucher disease type 1	62
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	61
From bench to bloodstream: Lipid nanoparticles drive on-demand CAR-T cells	61
Disease-modifying, multidimensional efficacy of putaminal CaV1.3-shRNA gene therapy in aged parkinsonism male and female macaques	61
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	61
A safer path to gene correction in junctional epidermolysis bullosa	59
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	59
Late-Breaking Abstracts I	59

Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche	58
Response: Promise and open questions of optogenetic vision restoration by MCO	58
Molecular Therapy’s growing influence	58
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	58
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	58
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	56
Modification of the VP1u region boosts transduction of adeno-associated virus vectors for ocular gene therapy	56
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	56
Dual AAV gene therapy using laminin-linking proteins ameliorates muscle and nerve defects in LAMA2-related muscular dystrophy	55
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	54
Intra-arterial transplantation of autologous mesoangioblasts in m.3243A>G mutation carriers is safe: First phase 1/2 human clinical study	54
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	53
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	53
Progress in skin gene therapy: From the inside and out	52
Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies	51
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	51
Transgene-induced cardiotoxicity in high-dose AAV gene transfer	51
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	51
Quo vadis American postdoc?	50
Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	50
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	50
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	50
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	50
Mucosal-associated invariant T cells for cancer immunotherapy	49
ASGCT 27th Annual Meeting Abstracts	49
Focused ultrasound as a delivery platform for CNS AAV gene therapy	49
Inflammatory mediators of mRNA vaccine-induced adverse reactions in mice	49
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	49
AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders	48
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	47
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	47
Managing allorejection in off-the-shelf CAR-engineered cell therapies	47
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	46
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	46
RNA editing: Expanding the potential of RNA therapeutics	46
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	45
A new advanced stem cell-based embryonic model: The ultimate model?	45
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	45
In this issue	45
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	45
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	45
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	44
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	44
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	43
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	43
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	43
Env-independent protection of intrarectal SIV challenge by vaccine induction of Gag/Vif-specific CD8+ T cells but not CD4+ T cells	43
Recent advances of engineered bacteria for therapeutic applications	42
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	42
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	42
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	42
Essential role of CD56dimNKG2C+ NK cells trained by SARS-CoV-2 vaccines in protecting against COVID-19	42
Host natural IgM shapes systemic efficacy of the oncolytic adenovirus	41
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	41
Computational structural optimization enhances IL13Rα2 – B7-H3 tandem CAR T cells to overcome antigen-heterogeneity-mediated tumor escape	41
GDF11: A promising new drug target for pulmonary fibrosis?	41
Efficient polymer nanoparticle-mediated delivery of gene editing reagents into human hematopoietic stem and progenitor cells	41
Deepm5C: A deep-learning-based hybrid framework for identifying human RNA N5-methylcytosine sites using a stacking strategy	41
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	40
Neonatal systemic gene therapy restores cardiorespiratory function in a rat model of Pompe disease	40
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	40
Preclinical quality, safety, and efficacy of a CGMP iPSC-derived myogenic progenitor product for the treatment of muscular dystrophies	40
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	40
Igniting CAR-NKT cells with IL-18	40
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	40
AAV-mediated DMPK silencing: A defining moment in myotonic dystrophy type 1 therapeutics	39
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	39
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	39
GDF11 Protects against Endothelial Injury and Reduces Atherosclerotic Lesion Formation in Apolipoprotein E-Null Mice	39
The curious case of AAV immunology	39
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	39
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	39
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	38
CD19xCD3 T cell engager shows therapeutic potential for refractory myasthenic syndromes	38
In this issue	38
Unlocking antigen flexibility with widened patient access: CD3FR in off-the-shelf CAR T cells	38
Second gene therapy for hemophilia B approved: More answers or questions?	37
From amputations to antibiotics: A future beyond “hacksaw” gene editing	37
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	37
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	37

Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	37
Harnessing engineered NK cells for refractory CD30+ lymphoma	37
Myeloid-lineage CAR knockin mice enable allogeneic immunotherapy for liver and lung fibrosis	36
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	36
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	36
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	36
In this issue	36
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	35
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	35
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	35
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	35
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	35
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	34
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	34
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	34
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	34
CD19xCD3 T cell engager blinatumomab effective in refractory generalized myasthenic syndromes	34
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	33
Current RNA strategies in treating cardiovascular diseases	33
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	33
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	33
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	33
2022 ASGCT Annual Meeting Abstracts	33
In this issue	32
An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	32
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors	32
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	32
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	32
Circulating urobilinogen augments inflammation and corticosteroid non-response in severe alcohol-induced hepatitis	32
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	32
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	32
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	32
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	32
Successful immunotherapy using adenovector gene therapy for the treatment of recurrent respiratory papillomatosis	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	31
Discovery, preclinical safety, and efficacy characterization of SMAC mimetic S-016-1348 as a potential cancer therapeutic	31
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	31
On-target/off-tumor toxicities following infusion of low-affinity Nectin-4-specific CAR T cells	31
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	31
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	31
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	31
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	31
In utero genetic therapy: Treatment of early onset neurological disorders before they start	31
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	30
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	30
Turning the tide in peritoneal metastases: Locoregional CAR-NK therapy primes systemic immunity in colorectal cancer	30
Nucleic acid modifications in self-nonself discrimination	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
Reprogramming the neuroblastoma tumor immune microenvironment to enhance GPC2 CAR T cells	30
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	30
Focused ultrasound expands intra-CSF AAV delivery to deep brain regions in rats and non-human primates	30
Gene replacement therapy for centronuclear myopathy: A breakthrough in complex genetic muscle disease	30
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	30
Repair Drive improves gene editing in the liver	30
Targeted therapy for rare lung cancers: Status, challenges, and prospects	30
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	30
New hope for older SMA patients with next-generation self-complementary AAV gene therapy	30
In this issue	30
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	29
Globin vector regulatory elements are active in early hematopoietic progenitor cells	29
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	29
Overcoming barriers to commercially pre-viable gene and cell therapies for rare and ultra-rare diseases	29
AAV-mediated genome editing is influenced by the formation of R-loops	29
Recovery of cone-mediated vision in Lebercilin associated retinal ciliopathy after gene therapy: One-year results of a phase I/II trial	29
The future of gene therapy: Safer vectors, sharper focus	29
An optimized SpCas9 high-fidelity variant for direct protein delivery	29
Phase 1 study of autologous T cells bearing fully human chimeric antigen receptors targeting mesothelin in mesothelin-expressing cancers	28
RAC1 directly phosphorylates both PKM2 and FBP1 to promote radioresistance in hepatocellular carcinoma	28
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	28
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	28
COVID-19 mRNA vaccines: Platforms and current developments	28
Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial	28
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	28
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	28
Identification of a therapeutic threshold for AAV-STXBP1 gene therapy in a rodent model of STXBP1 developmental and epileptic encephalopathy	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
Targeting cancer-induced nerve injury reverses immunotherapy resistance	27
CircRNA: Unlocking new frontiers in therapeutic and vaccine development	27
TOX-induced lnc-SUMF2-8 compromises antitumor function and anti-PD-1 response of CD8+ T cells via lysosome-dependent degradation of TCF-1	27
Promoting donor microglial replacement through augmented conditioning or radiation sensitivity	27
Celebrating 25 years of Molecular Therapy	27
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	27
Advances in stem cell and cellular therapeutics from the 9th Midwest Conference on Cell Therapy and Regenerative Medicine	27
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	27
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	27
In this issue	27
A sophisticated approach to targeting TERT activity in DLBCL	27
Clinical perspectives: Treating spinal muscular atrophy	27
Immunological responses and clinical outcomes in dogs with osteosarcoma receiving standard therapy and a Listeria vaccine expressing HER2	26
AAV gene therapy for GBA1-related diseases	26
Try before you buy: Empirical comparison of base editing approaches	26
Extracellular vesicle-mediated delivery of CRISPR machinery silences androgen receptor in castration-resistant prostate cancer cells	26
CRISPR-mediated MECOM depletion retards tumor growth by reducing cancer stem cell properties in lung squamous cell carcinoma	26
In this issue	26
Bispecific BAFF-R/BCMA CAR T cells control growth of heterogeneous plasma cells in multiple myeloma	26