Molecular Therapy

Article	Citations
CANT1 lncRNA Triggers Efficient Therapeutic Efficacy by Correcting Aberrant lncing Cascade in Malignant Uveal Melanoma	227
CRISPR-Cas9n-mediated ELANE promoter editing for gene therapy of severe congenital neutropenia	214
Reply to In vivo confusion over in vivo conversion	210
Engineering memory T cells as a platform for long-term enzyme replacement therapy in lysosomal storage disorders	195
Antitumor activity of a lectibody targeting cancer-associated high-mannose glycans	185
From cortex to medulla: Navigating the thymic landscape of T cell education	182
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques	174
Promotion or inhibition? This is a question in gene editing	168
IL-1Ra gene transfer potentiates BMP2-mediated bone healing by redirecting osteogenesis toward endochondral ossification	167
Response to: DNA transposon mechanisms and pathways of genotoxicity	152
A novel regulatory mechanism network mediated by lncRNA TUG1 that induces the impairment of spiral artery remodeling in preeclampsia	142
Getting the host antiviral machinery back on track: Targeting viral suppressors of RNA interference	140
Recombinant neutralizing secretory IgA antibodies for preventing mucosal acquisition and transmission of SARS-CoV-2	137
The neutralization effect of montelukast on SARS-CoV-2 is shown by multiscale in silico simulations and combined in vitro studies	136
Enhanced fatty acid oxidation via SCD1 downregulation fuels cardiac reprogramming	135
α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial	131
Generation of highly proliferative, rejuvenated cytotoxic T cell clones through pluripotency reprogramming for adoptive immunotherapy	130
RNAi mediated silencing of STAT3/PD-L1 in tumor-associated immune cells induces robust anti-tumor effects in immunotherapy resistant tumors	124
Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier	122
Reversion of metabolic dysfunction-associated steatohepatitis by skeletal muscle-directed FGF21 gene therapy	119
AAV vector manufacturing process design and scalability - Bending the trajectory to address vector-associated immunotoxicities	118
Preclinical lentiviral hematopoietic stem cell gene therapy corrects Pompe disease-related muscle and neurological manifestations	116
Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8	115
A highly stable human single-domain antibody-drug conjugate exhibits superior penetration and treatment of solid tumors	114
An engineered mitoCBE facilitates efficient mitochondrial DNA editing and modified mitochondrial transfer	113

Development of a peptide drug restoring AMPK and adipose tissue functionality in cancer cachexia	113
Safety concern of recombination between self-amplifying mRNA vaccines and viruses is mitigated in vivo	113
High-dose systemic adeno-associated virus vector administration causes liver and sinusoidal endothelial cell injury	113
miRNAs as neuro-oncologic therapeutics: A narrative review	107
The ARDS microenvironment enhances MSC-induced repair via VEGF in experimental acute lung inflammation	104
Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells	98
Asparagine endopeptidase protects podocytes in adriamycin-induced nephropathy by regulating actin dynamics through cleaving transgelin	96
Recent progress and future challenges in structure-based protein-protein interaction prediction	94
How to democratize cell and gene therapy: A global approach	92
Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo	90
Extracellular vesicles in neuroinflammation: Pathogenesis, diagnosis, and therapy	89
Biologics-based technologies for highly efficient and targeted RNA delivery	85
Roles of lncRNAs in brain development and pathogenesis: Emerging therapeutic opportunities	85
Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy	85
Spike mutation resilient scFv76 antibody counteracts SARS-CoV-2 lung damage upon aerosol delivery	85
Self-amplifying RNA vaccine protects mice against lethal Ebola virus infection	84
MicroRNA-124-3p-enriched small extracellular vesicles as a therapeutic approach for Parkinson’s disease	84
Modulation of NLRP3 inflammasomes activation contributes to improved survival and function of mesenchymal stromal cell spheroids	83
In This Issue	82
WDR5 facilitates EMT and metastasis of CCA by increasing HIF-1α accumulation in Myc-dependent and independent pathways	82
Late-Breaking Abstracts I	81
SINEUP RNA rescues molecular phenotypes associated with CHD8 suppression in autism spectrum disorder model systems	81
Efficient gene delivery admitted by small metabolites specifically targeting astrocytes in the mouse brain	80
In situ blockade of TNF-TNFR2 axis via oncolytic adenovirus improves antitumor efficacy in solid tumors	79
Gene editing efficiencies and hematopoietic stem cell fitness in sickle cell disease: A balancing act	76
Myeloma cell-intrinsic ANXA1 elevation and T cell dysfunction contribute to BCMA-negative relapse after CAR-T therapy	76
Progress in skin gene therapy: From the inside and out	76
A promoterless AAV6.2FF-based lung gene editing platform for the correction of surfactant protein B deficiency	74
Managing allorejection in off-the-shelf CAR-engineered cell therapies	74
Targeting cholesterol biosynthesis promotes anti-tumor immunity by inhibiting long noncoding RNA SNHG29-mediated YAP activation	71
AAV capsid engineering identified two novel variants with improved in vivo tropism for cardiomyocytes	70
Use of 2,6-diaminopurine as a potent suppressor of UGA premature stop codons in cystic fibrosis	70
Evaluation of repRNA vaccine for induction and in utero transfer of maternal antibodies in a pregnant rabbit model	68
Molecular Therapy Family Highlights	68
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice	66
Complete remission of tumors in mice with neoantigen-painted exosomes and anti-PD-1 therapy	66
ASGCT 27th Annual Meeting Abstracts	65
Autologous transplantation of mitochondria/rAAV IGF-I platforms in human osteoarthritic articular chondrocytes to treat osteoarthritis	64
Response: Promise and open questions of optogenetic vision restoration by MCO	64
Long Noncoding RNA-Maternally Expressed Gene 3 Contributes to Hypoxic Pulmonary Hypertension	63
Targeting astrocytes with in vivo gene addition: Can it rescue loss of brain myelin?	62
Retraction Notice to: The YY1-HOTAIR-MMP2 Signaling Axis Controls Trophoblast Invasion at the Maternal-Fetal Interface	62
Systemic AAV9.BVES delivery ameliorates muscular dystrophy in a mouse model of LGMDR25	62
Targeting PD-1+ T cells with small-format immunocytokines enhances IL-12 antitumor activity	62
Single-cell transcriptomics unveil a unique molecular profile of mesenchymal stem/stromal cell-induced myeloid-derived immune suppressor cells	61
Quo vadis American postdoc?	61
Low-inflammatory lipid nanoparticles facilitate safe mRNA vaccination against influenza virus infection	60
Molecular Therapy’s growing influence	60
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP	59
T-cell specific in vivo gene delivery with DART-AAVs targeted to CD8	58

Switch of ELF3 and ATF4 transcriptional axis programs the amino acid insufficiency-linked epithelial-to-mesenchymal transition	58
Fluoxetine ameliorates mucopolysaccharidosis type IIIA	57
Viral platform engineering for targeted gene delivery to human hematopoietic stem cells	57
Mesenchymal stromal cell delivery of oncolytic immunotherapy improves CAR-T cell antitumor activity	57
RNA m6A methylation regulates dissemination of cancer cells by modulating expression and membrane localization of β-catenin	56
RETRACTED: Hypoxia-induced lncHILAR promotes renal cancer metastasis via ceRNA for the miR-613/206/ 1-1-3p/Jagged-1/Notch/CXCR4 signaling pathway	56
Fast, accurate ranking of engineered proteins by target-binding propensity using structure modeling	55
Recombinant adeno-associated virus as a delivery platform for ocular gene therapy: A comprehensive review	54
ANKRD22 promotes resolution of psoriasiform skin inflammation by antagonizing NIK-mediated IL-23 production	54
Mucosal-associated invariant T cells for cancer immunotherapy	53
Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial	53
Engineering of efficiency-enhanced Cas9 and base editors with improved gene therapy efficacies	52
Superantigen-fused T cell engagers for tumor antigen-mediated robust T cell activation and tumor cell killing	51
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
The power of 2,6-diaminopurine in correcting UGA nonsense codons in CFTR mRNA	50
METTL3-m6A-Rubicon axis inhibits autophagy in nonalcoholic fatty liver disease	50
Carbon monoxide-induced autophagy enhances human mesenchymal stromal cell function via paracrine actions in murine polymicrobial sepsis	50
RNA editing: Expanding the potential of RNA therapeutics	50
The curious case of AAV immunology	49
Reconstructed glycosylase base editors GBE2.0 with enhanced C-to-G base editing efficiency and purity	49
Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models	49
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus	49
Chimeric oncolytic adenovirus to break away from neutralizing antibodies	48
A universal viral capsid protein based one step RNA synthesis and packaging system for rapid and efficient mRNA vaccine development	48
IL12 integrated into the CAR exodomain converts CD8+ T cells to poly-functional NK-like cells with superior killing of antigen-loss tumors	48
Transforming bacterial pathogens into wonder tools in cancer immunotherapy	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
LncRNA IFITM4P promotes immune escape by up-regulating PD-L1 via dual mechanism in oral carcinogenesis	47
Two cases of T cell lymphoma following Piggybac-mediated CAR T cell therapy	47
Structure-guided engineering of CD112 receptor variants for optimized immunotherapy	47
TIPE2 gene transfer ameliorates aging-associated osteoarthritis in a progeria mouse model by reducing inflammation and cellular senescence	47
ASGCT 2021: Time to celebrate and expand	47
The battle between host and SARS-CoV-2: Innate immunity and viral evasion strategies	47
In this issue	47
NIIMBL’s viral vector program: A cross-gene therapy community collaboration to transform viral vector development and manufacturing	46
Tailoring capsid-directed evolution technology for improved AAV-mediated CAR-T generation	46
A new advanced stem cell-based embryonic model: The ultimate model?	46
Inhibition of BTK and PI3Kδ impairs the development of human JMML stem and progenitor cells	46
Retraction Notice to: Comprehensive Analysis of the Expression and Prognosis for E2Fs in Human Breast Cancer	46
Env-independent protection of intrarectal SIV challenge by vaccine induction of Gag/Vif-specific CD8+ T cells but not CD4+ T cells	45
Potentiating CAR-T cell function in the immunosuppressive tumor microenvironment by inverting the TGF-β signal	45
The role of the mitochondrial protein VDAC1 in inflammatory bowel disease: a potential therapeutic target	44
A preclinical “magic bullet” against fibrolamellar hepatocellular carcinoma?	44
A localizing nanocarrier formulation enables multi-target immune responses to multivalent replicating RNA with limited systemic inflammation	44
The Molecular Therapy family of journals continues to lead the field of gene and cell therapy	44
An RNAi therapeutic targeting hepatic DGAT2 in a genetically obese mouse model of nonalcoholic steatohepatitis	44
Therapeutic base editing and prime editing of COL7A1 mutations in recessive dystrophic epidermolysis bullosa	43
Leukemia inhibitory factor, a double-edged sword with therapeutic implications in human diseases	43
Extracellular vesicles: Major actors of heterogeneity in tau spreading among human tauopathies	43
Live-attenuated RNA hybrid vaccine technology provides single-dose protection against Chikungunya virus	43
A comprehensive analysis of the efficacy and safety of COVID-19 vaccines	43
Intercellular transfer of miR-200c-3p impairs the angiogenic capacity of cardiac endothelial cells	43
Antiangiogenic Variant of TSP-1 Targets Tumor Cells in Glioblastomas	42
circRNA_0025202 Regulates Tamoxifen Sensitivity and Tumor Progression via Regulating the miR-182-5p/FOXO3a Axis in Breast Cancer	42
CAR T cell therapy for glioblastoma: A review of the first decade of clinical trials	42
Call for papers: A special issue on tackling emerging infectious diseases	42
From amputations to antibiotics: A future beyond “hacksaw” gene editing	42
Cleavage-free human genome editing	42
Cardiac reprogramming via chromatin remodeling by CRISPR activation	42
2022 ASGCT Annual Meeting Abstracts	41
A promising advance using oncolytic adenovirus to locally block tumorigenic TNF signaling	41
BETting on BRD4 inhibition to combat adaptive resistance to CAR T cell therapy in glioblastoma	41
Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec	41
Single AAV-mediated CRISPR-Nme2Cas9 efficiently reduces mutant hTTR expression in a transgenic mouse model of transthyretin amyloidosis	41
In this issue	41
Circular mRNA-based TCR-T offers a safe and effective therapeutic strategy for treatment of cytomegalovirus infection	40
Single-cell dissection of cellular and molecular features underlying mesenchymal stem cell therapy in ischemic acute kidney injury	40
Long-term reversal of chronic pain behavior in rodents through elevation of spinal agmatine	40
Pevonedistat, a first-in-class NEDD8-activating enzyme inhibitor, sensitizes cancer cells to VSVΔ51 oncolytic virotherapy	40
In memoriam: Kenneth I. Berns, MD, PhD (1938–2024)	40
Second gene therapy for hemophilia B approved: More answers or questions?	39
N1-methyladenosine formation, gene regulation, biological functions, and clinical relevance	39
Gene therapy using Aβ variants for amyloid reduction	39
Ex vivo and in vivo suppression of SARS-CoV-2 with combinatorial AAV/RNAi expression vectors	39
Neuroserpin gene therapy inhibits retinal ganglion cell apoptosis and promotes functional preservation in glaucoma	38
Protein expression/secretion boost by a novel unique 21-mer cis-regulatory motif (Exin21) via mRNA stabilization	38
Light-stimulated insulin secretion from pancreatic islet-like organoids derived from human pluripotent stem cells	37
Local magnetic delivery of adeno-associated virus AAV2(quad Y-F)-mediated BDNF gene therapy restores hearing after noise injury	37

An SCD1-dependent mechanoresponsive pathway promotes HCC invasion and metastasis through lipid metabolic reprogramming	37
AAV-mediated hepatic LPL expression ameliorates severe hypertriglyceridemia and acute pancreatitis in Gpihbp1 deficient mice and rats	37
ROCK inhibition enhanced hepatocyte liver engraftment by retaining membrane CD59 and attenuating complement activation	37
TNFAIP8L2 maintains hair cell function and regulates age-related hearing loss via mTORC1 signaling	37
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F	36
Enforced expression of Runx3 improved CAR-T cell potency in solid tumor via enhancing resistance to activation-induced cell death	36
Exploiting the biogenesis of extracellular vesicles for bioengineering and therapeutic cargo loading	36
Current RNA strategies in treating cardiovascular diseases	36
Immunomodulatory effect of locoregional therapy in the tumor microenvironment	35
PBAE-PEG-based lipid nanoparticles for lung cell-specific gene delivery	35
In this issue	35
Catalase, a therapeutic target in the reversal of estrogen-mediated aging	35
In this issue	34
Perioperative arginine prevents metastases by accelerating natural killer cell recovery after surgery	34
In this issue	34
Retraction Notice to: SET1A Cooperates With CUDR to Promote Liver Cancer Growth and Hepatocyte-like Stem Cell Malignant Transformation Epigenetically	34
Dibenzazepine-Loaded Nanoparticles Induce Local Browning of White Adipose Tissue to Counteract Obesity	34
LncRNA Osilr9 coordinates promoter DNA demethylation and the intrachromosomal loop structure required for maintaining stem cell pluripotency	34
C5a-C5aR1 axis controls mitochondrial fission to promote podocyte injury in lupus nephritis	34
Globin vector regulatory elements are active in early hematopoietic progenitor cells	34
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
Targeted therapy for rare lung cancers: Status, challenges, and prospects	33
Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption	33
In this issue	33
In vivo adenine base editing rescues adrenoleukodystrophy in a humanized mouse model	33
KCNN4 as a genomic determinant of cytosolic delivery by the attenuated cationic lytic peptide L17E	33
The 13th International Oncolytic Virus Conference: Powerful payloads gain clinical momentum	33
Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA	32
Dichotomous effects of cellular expression of STAT3 on tumor growth of HNSCC	32
Perinatal loss of galactosylceramidase in both oligodendrocytes and microglia is crucial for the pathogenesis of Krabbe disease in mice	32
Therapeutic application of extracellular vesicle-encapsulated CC16 in acute lung injury	32
Enforced dimerization between XBP1s and ATF6f enhances the protective effects of the UPR in models of neurodegeneration	32
AAV-mediated genome editing is influenced by the formation of R-loops	31
Repair Drive improves gene editing in the liver	31
Nucleic acid modifications in self-nonself discrimination	31
Enhancing VEGF therapy in T2D wounds with PLCγ2 epigenetic targeting	31
Immune tolerance induction by hepatic gene transfer: First-in-human evidence	31
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	30
A new MVA ancestor-derived oncolytic vaccinia virus induces immunogenic tumor cell death and robust antitumor immune responses	30
Extracellular vesicle-mediated communication between hepatocytes and natural killer cells promotes hepatocellular tumorigenesis	30
Cell type-selective targeted delivery of a recombinant lysosomal enzyme for enzyme therapies	30
Vision protection and robust axon regeneration in glaucoma models by membrane-associated Trk receptors	30
PRMT5 regulates RNA m6A demethylation for doxorubicin sensitivity in breast cancer	30
On- and off-target effects of paired CRISPR-Cas nickase in primary human cells	29
An optimized SpCas9 high-fidelity variant for direct protein delivery	29
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease	29
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates	29
Far-red light-activated human islet-like designer cells enable sustained fine-tuned secretion of insulin for glucose control	29
Tumor-derived exosomal microRNA-106b-5p activates EMT-cancer cell and M2-subtype TAM interaction to facilitate CRC metastasis	28
STING/ACSL4 axis-dependent ferroptosis and inflammation promote hypertension-associated chronic kidney disease	28
Critical role of TPRN rings in the stereocilia for hearing	28
Dual-antigen targeted iPSC-derived chimeric antigen receptor-T cell therapy for refractory lymphoma	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
Decoding the regulatory roles of non-coding RNAs in cellular metabolism and disease	28
P2Y12 inhibitor clopidogrel inhibits renal fibrosis by blocking macrophage-to-myofibroblast transition	28
COVID-19 mRNA vaccines: Platforms and current developments	28
In this issue	28
On your MARCKS, get set, deliver: Engineering extracellular vesicles	27
Anti-Cas9 immunity: A formidable challenge for muscle genome editing	27
Transient expression of factor VIII and a chronic high-fat diet induces ER stress and late hepatocyte oncogenesis	27
Gene augmentation therapy attenuates retinal degeneration in a knockout mouse model of Fam161a retinitis pigmentosa	27
Gene Therapy Restores Balance and Auditory Functions in a Mouse Model of Usher Syndrome	27
Knockdown of calpain1 in lumbar motoneurons reduces spasticity after spinal cord injury in adult rats	27
IL-10-Engineered Human CD4+ Tr1 Cells Eliminate Myeloid Leukemia in an HLA Class I-Dependent Mechanism	27
Versatile and efficient in vivo genome editing with compact Streptococcus pasteurianus Cas9	27
Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant	26
Cardiolipin remodeling by ALCAT1 links hypoxia to coronary artery disease by promoting mitochondrial dysfunction	26
Immunological responses and clinical outcomes in dogs with osteosarcoma receiving standard therapy and a Listeria vaccine expressing HER2	26
Structural and functional characterization of capsid binding by anti-AAV9 monoclonal antibodies from infants after SMA gene therapy	26
Reduction of circulating IgE and allergens by a pH-sensitive antibody with enhanced FcγRIIb binding	26
Celebrating 25 years of Molecular Therapy	26
In this issue	26
Try before you buy: Empirical comparison of base editing approaches	26
Clinical perspectives: Treating spinal muscular atrophy	26
Gene therapy then and now: A look back at changes in the field over the past 25 years	26
Combined intraocular and intravenous gene delivery for therapy of gyrate atrophy of the choroid and retina	26
Targeting tissue-resident memory CD8+ T cells in the kidney is a potential therapeutic strategy to ameliorate podocyte injury and glomerulosclerosis	26
The short inverted repeats-induced circEXOC6B inhibits prostate cancer metastasis by enhancing the binding of RBMS1 and HuR	26
DNA based Immunotherapy for Cancer: in vivo Approaches for Recalcitrant Targets	26
A BPTF-specific PROTAC degrader enhances NK cell-based cancer immunotherapy	26
Carrier-free mRNA vaccine induces robust immunity against SARS-CoV-2 in mice and non-human primates without systemic reactogenicity	26
Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder	26
In vivo gene editing via homology-independent targeted integration for adrenoleukodystrophy treatment	26
CRISPR-mediated MECOM depletion retards tumor growth by reducing cancer stem cell properties in lung squamous cell carcinoma	26
Genome editing in large animal models	26
The paradigm of immune escape by SARS-CoV-2 variants and strategies for repositioning subverted mAbs against escaped VOCs	25
Genetic and pharmacological inhibition of GRPR protects against acute kidney injury via attenuating renal inflammation and necroptosis	25
Lymph node macrophages drive innate immune responses to enhance the anti-tumor efficacy of mRNA vaccines	25
Feasibility and preclinical efficacy of CD7-unedited CD7 CAR T cells for T cell malignancies	25
Retraction Notice to: Precise editing of FGFR3-TACC3 fusion genes with CRISPR-Cas13a in glioblastoma	25
M2 macrophages secrete glutamate-containing extracellular vesicles to alleviate osteoporosis by reshaping osteoclast precursor fate	25
PD-1 and TIGIT downregulation distinctly affect the effector and early memory phenotypes of CD19-targeting CAR T cells	25
Visualizing lipid nanoparticle trafficking for mRNA vaccine delivery in non-human primates	25
Hitchhiking of Cas9 with nucleus-localized proteins impairs its controllability and leads to efficient genome editing of NLS-free Cas9	24
R-loops facilitate AAV-mediated nuclease-free gene targeting	24