Molecular Therapy-Methods & Clinical Development

Article	Citations
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	105
Thank you to our 2024 reviewers	89
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	74
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	71
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	71
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	69
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	67
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	67
First use of adeno-associated viruses in the human inner ear	63
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	59
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	57
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	57
Molecular earplugs to protect the inner ear	55
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	48
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	47
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	47
Unfolding of viral protein 1 N-termini facilitates genome ejection from recombinant adeno-associated virus serotype 8	45
Genetic surgery for a cystic fibrosis-causing splicing mutation	45
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	41
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	40
Exploring human plasma proteomic variations in mucolipidosis type IV	40
Differential T cell immune responses to deamidated adeno-associated virus vector	39
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	38
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	37
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	36

Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	36
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	35
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	34
The unknown impact of conditioning on HSC engraftment and clonal dynamics	33
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	33
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	32
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	32
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	32
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	31
Novel transferrin receptor-mediated enzyme replacement therapy efficiently treats myogenic and neurogenic aspects of Pompe disease in mice	31
Modulation of AAV transduction and integration targeting by topoisomerase poisons	31
Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo	31
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	31
AAV-mediated inner ear gene delivery triggers mild host immune responses in the mammalian inner ear	31
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	31
Dose-finding and in vivo safety study of an adipose targeted leptin gene therapy for congenital leptin deficiency	30
Applying a clinical lens to animal models of CAR-T cell therapies	30
Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates	30
Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy	29
Defining the optimal dose and therapeutic window in SMA with respiratory distress type I model mice, FVB/NJ-Ighmpb2	29
CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice	29
Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling	29
A combinatorial CRISPR-Cas12a attack on HIV DNA	29
A lentiviral vector B cell gene therapy platform for the delivery of the anti-HIV-1 eCD4-Ig-knob-in-hole-reversed immunoadhesin	28
Inducible caspase 9-mediated suicide gene therapy using AAV6 vectors in a murine model of breast cancer	28
Development of novel lipoplex formulation methodologies to improve large-scale transient transfection for lentiviral vector manufacture	28
Predicted deleterious variants in the human genome relevant to gene therapy with adeno-associated virus vectors	27
Nonviral delivery of nCas9 for “safe harbor” integration to treat MPS IVA	27
Co-transducing B7H3 CAR-NK cells with the DNR preserves their cytolytic function against GBM in the presence of exogenous TGF-β	26
The longitudinal kinetics of AAV5 vector integration profiles and evaluation of clonal expansion in mice	26
Suppression of toxic transgene expression by optimized artificial miRNAs increases AAV vector yields in HEK-293 cells	25
Sustained high expression of human FVII following AAV8-mediated gene delivery in mice	25
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A	25
AAV yield, bioactivity, and particle heterogeneity are impacted by genome size and non-coding DNA elements	25
In vitro and in vivo expansion of CD33/HBG promoter-edited HSPCs with Mylotarg	24
Preclinical pharmacology and safety studies to support an AAV9 NGLY1 gene therapy clinical trial for the treatment of NGLY1 deficiency	24
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications	24
Manufacturing DNA in E. coli yields higher-fidelity DNA than in vitro enzymatic synthesis	24
Transcriptomic analysis reveals optimal cytokine combinations for SARS-CoV-2-specific T cell therapy products	24
Modulation of the pharmacokinetics of soluble ACE2 decoy receptors through glycosylation	24
A novel approach to quantitate biodistribution and transduction of adeno-associated virus gene therapy using radiolabeled AAV vectors in mice	24
Bringing base editing to the clinic: The next generation of genome editors	23
Development of circular AAV cargos for targeted seamless insertion with large serine integrases	23
Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models	23
Biodistribution and safety of a single rAAV3B-AAT vector for silencing and replacement of alpha-1 antitrypsin in Cynomolgus macaques	23
A linear DNA encoding the SARS-CoV-2 receptor binding domain elicits potent immune response and neutralizing antibodies in domestic cats	22
Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model	22
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8	22
Confirmatory detection of neutralizing antibodies to AAV gene therapy using a cell-based transduction inhibition assay	22
Linker-specific monoclonal antibodies present a simple and reliable detection method for scFv-based CAR NK cells	22

Monitoring CAR T cell generation with a CD8-targeted lentiviral vector by single-cell transcriptomics	22
In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans	22
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice	22
AAV8BP2 and AAV8 transduce the mammalian cochlear lateral wall and endolymphatic sac with high efficiency	22
Lipid nanoparticles: Composition, formulation, and application	21
Research progress of mosquito-borne virus mRNA vaccines	21
Emerging therapeutic potential of adeno-associated virus-mediated gene therapy in liver fibrosis	21
Ionizable lipid nanoparticles with functionalized PEG-lipids increase retention in the tumor microenvironment	21
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	21
Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors	21
Toward lentiviral vectors for antiangiogenic ocular gene therapy	20
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device	20
Non-canonical capsid engineering highlights new possibilities for AAV vectorology	20
Gene editing-based targeted integration for correction of Wiskott-Aldrich syndrome	20
Efficient and sustained FOXP3 locus editing in hematopoietic stem cells as a therapeutic approach for IPEX syndrome	20
Optimizing AAV2/6 microglial targeting identified enhanced efficiency in the photoreceptor degenerative environment	20
Optimization of hypo-alloimmunogenic multispecific CAR-T and SARS-CoV-2-specific T cells for off-the-shelf adoptive cell therapy	20
Toward effective hematopoietic stem cell gene therapies: Optimized conditioning regimen and stem cell source in harmony	20
Starburst amacrine cells amplify optogenetic visual restoration through gap junctions	20
Universal ddPCR-based assay for the determination of lentivirus infectious titer and lenti-modified cell vector copy number	20
Perineural delivery of AAV2/9 in non-human primates is a safe and efficient route for gene therapy in Charcot-Marie-Tooth diseases	19
Human amnionic progenitor cell secretome mitigates the consequence of traumatic optic neuropathy in a mouse model	19
Development of cell-based assay for detecting replication-competent adeno-associated virus by qPCR	19
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors	19
Development of LC-MS methods for AAV capsid protein quantification and host cell protein profiling	19
Durable tissue-specific transgene expression in newborn mice following intraperitoneal delivery of non-cytotoxic HSV vectors	19
Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6	18
Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity	18
Molecular dynamics of genome editing with CRISPR-Cas9 and rAAV6 virus in human HSPCs to treat sickle cell disease	18
Extracellular vesicles ameliorates sleep deprivation induced anxiety-like behavior and cognitive impairment in mice	18
Simultaneous engineering of natural killer cells for CAR transgenesis and CRISPR-Cas9 knockout using retroviral particles	18
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease	18
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry	18
Assessment of adeno-associated virus purity by capillary electrophoresis-based western	18
Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy	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
Brain-targeted ex vivo lentiviral gene therapy: Implications for MPS and beyond	17
Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model	17
VikAD, a Vika site-specific recombinase-based system for efficient and scalable helper-dependent adenovirus production	17
Transduction characteristics of alternative adeno-associated virus serotypes in the cat brain by intracisternal delivery	17
Engineered mesenchymal stromal cell therapy during human lung ex vivo lung perfusion is compromised by acidic lung microenvironment	17
It’s all about location: Targeting the right spot for Wiskott-Aldrich syndrome	17
Continuous manufacturing of lentiviral vectors using a stable producer cell line in a fixed-bed bioreactor	17
Gene replacement therapy in a schwannoma mouse model of neurofibromatosis type 2	17
Necessity of strengthening the current clinical regulatory for companion diagnostics: An institutional comparison of the FDA, EMA, and MFDS	17
Lentiviral vector mediated gene therapy for type I Dent disease ameliorates Dent disease-like phenotypes for three months in ClC-5 null mice	17
Induction of antigen-specific tolerance by hepatic AAV immunotherapy regardless of T cell epitope usage or mouse strain background	16
Reversal of neuroinflammation in novel GS model mice by single i.c.v. administration of CHO-derived rhCTSA precursor protein	16
FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving adeno-associated virus gene transfer efficacy	16
Rationally engineered novel AAV capsids for intra-articular gene delivery	16
A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR	16
Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies	16
Reversing PAI-1 deficiency in blood using mRNA lipid nanoparticles	16
Improving cell-specific recombination using AAV vectors in the murine CNS by capsid and expression cassette optimization	16
Atelocollagen supports three-dimensional culture of human induced pluripotent stem cells	16
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease	16
mRNA-LNP vaccine strategies: Effects of adjuvants on non-parenchymal liver cells and tolerance	16
Targeted biallelic integration of an inducible Caspase 9 suicide gene in iPSCs for safer therapies	15
In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9	15
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers	15
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform	15
Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes	15
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid	15
A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery	15
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	15
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model	15
Perspectives of the Friedreich ataxia community on gene therapy clinical trials	15
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells	15
Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations	15
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis	15
Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection	15
Gene therapy for Friedreich ataxia: Too much, too little, or just right?	15
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions	14
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy	14
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	14
A rescue fanconi anemia humanized mouse model with endogenous FA mutation and high human hematopoietic stem cell chimerism	14
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine	14
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	14
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	14

Persistent tailoring of MSC activation through genetic priming	14
Engineering a highly durable adeno-associated virus receptor for analytical applications	14
Safety through design: Expanding options for spinal muscular atrophy gene therapy	14
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life	14
Efficient autocrine and paracrine signaling explain the osteogenic superiority of transgenic BMP-2 over rhBMP-2	13
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	13
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	13
A clinically viable approach to restoring visual function using optogenetic gene therapy	13
A positive take on negative selection for CAR-T manufacturing	13
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine	13
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells	13
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts	13
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	13
PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development	13
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics	13
Deciphering key parameters enhancing lentiviral vector producer cells yields: Vector components copy number and expression	13
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	13
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	13
From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia	12
Can mitochondria brown the lower-limb adipocytes?	12
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study	12
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned	12
An automated and high-throughput approach for enhanced precision of adenoviral titering	12
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity	12
Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo	12
Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy	12
Measurement solutions and standards for advanced therapy	12
AAV-shDUX4 provides short-term benefits but limited long-term efficacy in a DUX4 mouse model of FSHD	12
Metabolic priming of GD2 TRAC-CAR T cells during manufacturing promotes memory phenotypes while enhancing persistence	12
Self-amplifying mRNA bicistronic influenza vaccines raise cross-reactive immune responses in mice and prevent infection in ferrets	12
Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia	12
Evaluation of efficacy and safety of AAV8-ΔC4ATP7B gene therapy in a mutant mouse model of Wilson’s disease	12
Harnessing mRNA-lipid nanoparticles as innovative therapies for autoimmune diseases	12
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study	12
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II	12
Innate immune response to AAV-based gene therapy vectors: Mechanisms of complement activation and cytokine release	12
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice	12
Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma	12
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy	11
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors	11
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency	11
mRNA-based therapy proves superior to the standard of care for treating hereditary tyrosinemia 1 in a mouse model	11
Implementing a robust platform analytical procedure for measuring adeno-associated virus vector genome titer	11
A novel FOXP3 knockout-humanized mouse model for pre-clinical safety and efficacy evaluation of Treg-like cell products	11
Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia	11
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver	11
CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa	11
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters	11
Nonclinical strategies and considerations to enable the redosing of gene therapies	11
Identification of a novel neutralization epitope in rhesus AAVs	11
DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome	11
Stimulation of the immune system by a tumor antigen-bearing adenovirus-inspired VLP allows control of melanoma growth	11
Self-amplifying mRNA SARS-CoV-2 vaccines raise cross-reactive immune response to variants and prevent infection in animal models	11
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1	11
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone	11
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	11
Macrophage manufacturing and engineering with 5′-Cap1 and N1-methylpseudouridine-modified mRNA	11
PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington’s disease	11
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs	11