Molecular Therapy-Methods & Clinical Development

Article	Citations
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes	107
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	90
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics	72
Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts	70
αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues	69
Recombinant adeno-associated virus batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution at single molecule resolution	60
A novel platform for engineered AAV-based vaccines	55
Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutions	47
Thank you to our 2024 reviewers	47
Characterization of drusen formation in a primary porcine tissue culture model of dry AMD	46
Size-exclusion chromatography as a multi-attribute method for process and product characterization of adeno-associated virus	45
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	45
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells	44
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	42
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	42
Off-the-shelf allogeneic natural killer cells for the treatment of COVID-19	42
AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection	41
Systemic Delivery of AAV5, AAV8, and AAV9 Packaging a C5-12-Micro-dystrophin-Flag Expression Cassette in Non-Human Primates	38
Molecular earplugs to protect the inner ear	37
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	36
Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation	35
Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells	35
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine	35
Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain	34
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	33

Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	33
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy	32
Generation of hepatitis C virus–resistant liver cells by genome editing–mediated stable expression of RNA aptamer	32
Deferoxamine mesylate improves splicing and GAA activity of the common c.-32-13T>G allele in late-onset PD patient fibroblasts	31
Inhibition of indoleamine 2,3-dioxygenase 1 synergizes with oxaliplatin for efficient colorectal cancer therapy	31
Evaluation of parameters for efficient purification and long-term storage of herpes simplex virus-based vectors	30
Expression of NMNAT1 in the photoreceptors is sufficient to prevent NMNAT1-associated retinal degeneration	29
Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice	29
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment	29
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	29
Efficient autocrine and paracrine signaling explain the osteogenic superiority of transgenic BMP-2 over rhBMP-2	29
Gene therapy for Friedreich ataxia: Too much, too little, or just right?	29
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	29
Dasatinib is a potent enhancer for CAR T cell generation by CD3-targeted lentiviral vectors	28
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine	28
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	27
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method	27
Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease	27
Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expression vector system	27
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease	27
Broadly active zinc finger protein-guided transcriptional activation of HIV-1	26
A pro-inflammatory mediator USP11 enhances the stability of p53 and inhibits KLF2 in intracerebral hemorrhage	26
Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL	25
A T cell-targeted multi-antigen vaccine generates robust cellular and humoral immunity against SARS-CoV-2 infection	25
Directed Evolution of AAV Serotype 5 for Increased Hepatocyte Transduction and Retained Low Humoral Seroreactivity	25
Spatiotemporal in vivo tracking of polyclonal human regulatory T cells (Tregs) reveals a role for innate immune cells in Treg transplant recruitment	24
Decrease in Angiotensin-Converting Enzyme activity but not concentration in plasma/lungs in COVID-19 patients offers clues for diagnosis/treatment	24
UTX/KDM6A deletion promotes the recovery of spinal cord injury by epigenetically triggering intrinsic neural regeneration	24
Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein assembly	24
Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model	24
Multiparametric domain insertional profiling of adeno-associated virus VP1	24
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1	23
Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies	23
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice	23
SAG therapy restores bone growth and reduces enchondroma incidence in a model of skeletal chondrodysplasias caused by Ihh deficiency	23
A positive take on negative selection for CAR-T manufacturing	23
Channelrhodopsin fluorescent tag replacement for clinical translation of optogenetic hearing restoration	23
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	23
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life	22
Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations	22
Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production	22
Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study	22
Glutaredoxin-1 modulates the NF-κB signaling pathway to activate inducible nitric oxide synthase in experimental necrotizing enterocolitis	22
TAZing down metabolic mayhem: siRNAs against liver inflammation and fibrosis in humanized mice	21
Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics	21
Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells	21
First use of adeno-associated viruses in the human inner ear	21
Ad- and AAV8-mediated ABCA1 gene therapy in a murine model with retinal ischemia/reperfusion injuries	21
Genetic surgery for a cystic fibrosis-causing splicing mutation	21
Combined clarification and affinity capture using magnetic resin enables efficient separation of rAAV5 from cell lysate	21

Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	20
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	20
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs	20
CReVIS-Seq: A highly accurate and multiplexable method for genome-wide mapping of lentiviral integration sites	20
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	20
Cryopreserved anti-CD22 and bispecific anti-CD19/22 CAR T cells are as effective as freshly infused cells	20
Exosomes and organ-specific metastasis	19
Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates	19
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	18
AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice	18
Anti-tau intrabodies: From anti-tau immunoglobulins to the development of functional scFv intrabodies	18
Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment	18
Engineering a highly durable adeno-associated virus receptor for analytical applications	18
A clinically viable approach to restoring visual function using optogenetic gene therapy	17
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series	17
Perspectives of the Friedreich ataxia community on gene therapy clinical trials	17
Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome	17
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	17
Tumor antigen-loaded AAV vaccine drives protective immunity in a melanoma animal model	17
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal	17
Monitoring of tisagenlecleucel transgene DNA using a quantitative polymerase chain reaction assay	17
Targeted knockdown of the adenosine A2A receptor by lipid NPs rescues the chemotaxis of head and neck cancer memory T cells	17
Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease	17
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	17
Efficient treatment of a preclinical inflammatory bowel disease model with engineered bacteria	17
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	17
Hexon modification of human adenovirus type 5 vectors enables efficient transduction of human multipotent mesenchymal stromal cells	16
Differential T cell immune responses to deamidated adeno-associated virus vector	16
A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes	16
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	16
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts	16
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	16
Hypothalamic AAV-BDNF gene therapy improves metabolic function and behavior in the Magel2-null mouse model of Prader-Willi syndrome	16
AAV-mediated expression of HLA-G for the prevention of experimental ocular graft vs. host disease	16
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning	16
Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues	16
Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements	16
Large-scale manufacturing of base-edited chimeric antigen receptor T cells	16
A C1qTNF3 collagen domain fusion chaperones diverse secreted proteins and anti-Aβ scFvs: Applications for gene therapies	15
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	15
Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection	15
Preclinical specificity & activity of a fully human 41BB-expressing anti-CD19 CART- therapy for treatment-resistant autoimmune disease	15
Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard	15
Nanodysferlins support membrane repair and binding to TRIM72/MG53 but do not localize to t-tubules or stabilize Ca2+ signaling	15
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	15
Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses	15
Genome length determination in adeno-associated virus vectors with mass photometry	15
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	15
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions	15
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	15
Fast HPLC-based affinity method to determine capsid titer and full/empty ratio of adeno-associated viral vectors	15
Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis	14
miRNA-mediated control of exogenous OCT4 during mesenchymal-epithelial transition increases measles vector reprogramming efficiency	14
A third dose of the unmodified COVID-19 mRNA vaccine CVnCoV enhances quality and quantity of immune responses	14
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	14
AAV vector production: Troublesome host innate responses in another setting	14
Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs	14
Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions	14
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors	14
Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity	14
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	14
PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development	14
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice	14
AAV vector production: Troublesome host innate responses in another setting	14
An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome	13
Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein	13
Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency	13
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis	13
Improvement of HSV-1 based amplicon vectors for a safe and long-lasting gene therapy in non-replicating cells	13
Large-scale generation of IL-12 secreting macrophages from human pluripotent stem cells for cancer therapy	13
Targeting the Apoa1 locus for liver-directed gene therapy	13
Highly branched poly β-amino ester/CpG-depleted CFTR plasmid nanoparticles for non-viral gene therapy in lung cystic fibrosis disease	13
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform	13
Persistent tailoring of MSC activation through genetic priming	13
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	13
In vivo genome editing at the albumin locus to treat methylmalonic acidemia	13
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1	13
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	13
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model	13
Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness	13

The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome	13
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice	13
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia	13
Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma	13
Optogenetic targeting of AII amacrine cells restores retinal computations performed by the inner retina	13
CRISPR-Cas9-based non-viral gene editing therapy for topical treatment of recessive dystrophic epidermolysis bullosa	12
Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing	12
Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina	12
Testing the efficacy of a human full-length OPG-Fc analog in a severe model of cardiotoxin-induced skeletal muscle injury and repair	12
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS	12
Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease	12
Improved safety of induced pluripotent stem cell-derived antigen-presenting cell-based cancer immunotherapy	12
Can mitochondria brown the lower-limb adipocytes?	12
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model	12
New dawn of cellular therapies in autoimmune diseases	12
Sendai F/HN pseudotyped lentiviral vector transduces human ciliated and non-ciliated airway cells using α 2,3 sialylated receptors	12
Archival skin biopsy specimens as a tool for miRNA-based diagnosis: Technical and post-analytical considerations	12
Measurement solutions and standards for advanced therapy	12
Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery	12
In vitro characterization of engineered red blood cells as viral traps against HIV-1 and SARS-CoV-2	12
The sodium/glucose cotransporters as potential therapeutic targets for CF lung diseases revealed by human lung organoid swelling assay	12
Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo	12
Directed evolution of AAV accounting for long-term and enhanced transduction of cardiovascular endothelial cells in vivo	11
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease	11
PAM-flexible dual base editor-mediated random mutagenesis and self-activation strategies to improve CRISPRa potency	11
Every little bit helps: A single-residue switch in a vascular AAV enables blood-brain barrier penetration	11
Immunological evaluation of an inactivated SARS-CoV-2 vaccine in rhesus macaques	11
Adipose-derived stem cells protect motor neurons and reduce glial activation in both in vitro and in vivo models of ALS	11
Peripheral leukemia burden at time of apheresis negatively affects the clinical efficacy of CART19 in refractory or relapsed B-ALL	11
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	11
Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution	11
From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia	11
The unknown impact of conditioning on HSC engraftment and clonal dynamics	11
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	11
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors	11
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution	11
Glycoengineering of AAV-delivered monoclonal antibodies yields increased ADCC activity	11
Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy	11
High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors	11
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver	11
An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction	11
Discovery of bone morphogenetic protein 7-derived peptide sequences that attenuate the human osteoarthritic chondrocyte phenotype	11
Self-amplifying mRNA SARS-CoV-2 vaccines raise cross-reactive immune response to variants and prevent infection in animal models	11
Light at the end of the tunnel of Corti	11
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	11
Systemic and local immune responses to intraocular AAV vector administration in non-human primates	11
Specific gene expression in unmyelinated dorsal root ganglion neurons in nonhuman primates by intra-nerve injection of AAV 6 vector	11
Characterization of AAV-mediated dorsal root ganglionopathy	11
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study	11