Molecular Therapy-Methods & Clinical Development

Article	Citations
Thank you to our 2024 reviewers	135
Exploring human plasma proteomic variations in mucolipidosis type IV	104
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	99
Molecular earplugs to protect the inner ear	92
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	90
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	85
Genetic surgery for a cystic fibrosis-causing splicing mutation	77
Genome Editing in Patient iPSCs Corrects the Most Prevalent USH2A Mutations and Reveals Intriguing Mutant mRNA Expression Profiles	69
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	63
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	61
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	57
First use of adeno-associated viruses in the human inner ear	48
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	46
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	45
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	45
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	44
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	44
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	44
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	43
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	42
Biodistribution of AAV1, AAV5, AAV9, and AAVDJ serotypes after intra-cisterna magna delivery in non-human primates	42
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	40
Molecular Therapy Advances: Building the bridge between discovery and cure	39
The unknown impact of conditioning on HSC engraftment and clonal dynamics	38
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	37

Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice	36
Dose-finding and in vivo safety study of an adipose targeted leptin gene therapy for congenital leptin deficiency	34
Modulation of AAV transduction and integration targeting by topoisomerase poisons	34
Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo	33
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	33
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	32
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	32
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	32
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	31
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	31
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	31
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	29
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	29
Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling	28
Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates	28
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	28
Applying a clinical lens to animal models of CAR-T cell therapies	28
Sustained high expression of human FVII following AAV8-mediated gene delivery in mice	27
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice	27
Development of novel lipoplex formulation methodologies to improve large-scale transient transfection for lentiviral vector manufacture	27
Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency	27
Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA	27
Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer	26
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells	26
Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy	26
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice	26
A lentiviral vector B cell gene therapy platform for the delivery of the anti-HIV-1 eCD4-Ig-knob-in-hole-reversed immunoadhesin	26
AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements	25
A novel approach to quantitate biodistribution and transduction of adeno-associated virus gene therapy using radiolabeled AAV vectors in mice	25
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models	25
Co-transducing B7H3 CAR-NK cells with the DNR preserves their cytolytic function against GBM in the presence of exogenous TGF-β	25
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications	25
Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors	25
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model	25
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques	24
Ionizable lipid nanoparticles with functionalized PEG-lipids increase retention in the tumor microenvironment	24
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice	24
A linear DNA encoding the SARS-CoV-2 receptor binding domain elicits potent immune response and neutralizing antibodies in domestic cats	24
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation	24
Linker-specific monoclonal antibodies present a simple and reliable detection method for scFv-based CAR NK cells	23
Development of circular AAV cargos for targeted seamless insertion with large serine integrases	23
Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis	23
Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis	23
In vitro and in vivo expansion of CD33/HBG promoter-edited HSPCs with Mylotarg	22
Lipid nanoparticles: Composition, formulation, and application	22
Research progress of mosquito-borne virus mRNA vaccines	22
AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency	22
Bringing base editing to the clinic: The next generation of genome editors	22
Comparative analysis of cell-specific promoters in AAV9-mediated gene therapy targeting the central nervous system	22
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans	22

Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number	21
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	21
Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome	21
Landscape of ex vivo gene therapies: Technological trends and future prospects	21
Optimization of hypo-alloimmunogenic multispecific CAR-T and SARS-CoV-2-specific T cells for off-the-shelf adoptive cell therapy	21
Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony	21
Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity	21
Human amnionic progenitor cell secretome mitigates the consequence of traumatic optic neuropathy in a mouse model	21
Perineural delivery of AAV2/9 in non-human primates is a safe and efficient route for gene therapy in Charcot-Marie-Tooth diseases	21
Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome	21
Durable tissue-specific transgene expression in newborn mice following intraperitoneal delivery of non-cytotoxic HSV vectors	20
Non-canonical capsid engineering highlights new possibilities for AAV vectorology	20
Starburst amacrine cells amplify optogenetic visual restoration through gap junctions	20
Characterization of adeno-associated viral transduction of retinal ganglion cells in adult and old mice	20
Development of cell-based assay for detecting replication-competent adeno-associated virus by qPCR	20
Toward CAR-B cells for HIV-1 therapy	20
Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery	19
Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy	19
Toward lentiviral vectors for antiangiogenic ocular gene therapy	19
Development of LC-MS methods for AAV capsid protein quantification and host cell protein profiling	19
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6	19
It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome	19
Assessment of adeno-associated virus purity by capillary electrophoresis-based western	19
Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice	19
Rationally engineered novel AAV capsids for intra-articular gene delivery	19
Reversing PAI-1 deficiency in blood using mRNA lipid nanoparticles	19
Simultaneous engineering of natural killer cells for CAR transgenesis and CRISPR-Cas9 knockout using retroviral particles	19
Adeno-associated virus serotype 9 structural heterogeneity and stability characterized by charge detection mass spectrometry	19
Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond	18
Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of the FDA, EMA, and MFDS	18
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid	18
Targeted biallelic integration of an inducible Caspase 9 suicide gene in iPSCs for safer therapies	18
Lentiviral vector mediated gene therapy for type I Dent disease ameliorates Dent disease-like phenotypes for three months in ClC-5 null mice	18
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR	18
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2	18
Induction of antigen-specific tolerance by hepatic AAV immunotherapy regardless of T cell epitope usage or mouse strain background	18
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells	18
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease	18
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies	17
Identifying MAGE-A4-positive tumors for TCR T cell therapies in HLA-A∗02-eligible patients	17
Immunosuppression reduces rAAV2.5T neutralizing antibodies that limit efficacy following repeat dosing to ferret lungs	17
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor	17
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model	17
Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization	17
FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy	17
Perspectives of the Friedreich ataxia community on gene therapy clinical trials	16
Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes	16
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	16
mRNA-LNP vaccine strategies: Effects of adjuvants on non-parenchymal liver cells and tolerance	16
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	16
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	16
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	16
Deciphering key parameters enhancing lentiviral vector producer cells yields: Vector components copy number and expression	16
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine	16
A positive take on negative selection for CAR-T manufacturing	15
AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration	15
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics	15
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform	15
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	15
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells	15
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model	15
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions	15
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	15
Engineering a highly durable adeno-associated virus receptor for analytical applications	15
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	15
A clinically viable approach to restoring visual function using optogenetic gene therapy	15
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy	15
A rescue fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism	15
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis	14
An automated and high-throughput approach for enhanced precision of adenoviral titering	14
Self-amplifying mRNA bicistronic influenza vaccines raise cross-reactive immune responses in mice and prevent infection in ferrets	14
Persistent tailoring of MSC activation through genetic priming	14
Safety through design: Expanding options for spinal muscular atrophy gene therapy	14
Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma	14
Can mitochondria brown the lower-limb adipocytes?	14
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	14
Efficient autocrine and paracrine signaling explain the osteogenic superiority of transgenic BMP-2 over rhBMP-2	14
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life	14
Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection	14
From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia	14

Measurement solutions and standards for advanced therapy	14
PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development	14
Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia	14
Elucidation of the binding interaction interface between AAV serotype 11 capsid protein and host nuclear import proteins	13
mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model	13
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters	13
Nonclinical strategies and considerations to enable the redosing of gene therapies	13
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study	13
When pullulanase needs a little push: MyoAAV capsids enhance gene therapy for GSD IIIa	13
CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa	13
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy	13
Innate immune response to AAV-based gene therapy vectors: Mechanisms of complement activation and cytokine release	13
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study	13
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II	13
Evaluation of efficacy and safety of AAV8-ΔC4ATP7B gene therapy in a mutant mouse model of Wilson’s disease	13
Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence	13
GENE TARGET: A framework for evaluating Mendelian neurodevelopmental disorders for gene therapy	12
Whole-body galactose oxidation as a robust functional assay to assess the efficacy of gene-based therapies in a mouse model of Galactosemia	12
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency	12
Why regulatory T cells love lactic acid	12
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors	12
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned	12
A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products	12
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper	12
Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia	12
AAV-shDUX4 provides short-term benefits but limited long-term efficacy in a DUX4 mouse model of FSHD	12
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity	12
Harnessing mRNA-lipid nanoparticles as innovative therapies for autoimmune diseases	12
Orthogonal approaches to AAV vector characterization: Validating quantitative TEM for partially filled particles	12
DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome	12
The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs	12
Identification of a novel neutralization epitope in rhesus AAVs	12
Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo	12
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs	12
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver	12
Implementing a robust platform analytical procedure for measuring adeno-associated virus vector genome titer	12