Molecular Therapy-Methods & Clinical Development

Article	Citations
Analytical methods for process and product characterization of recombinant adeno-associated virus-based gene therapies	107
Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning	90
CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain	89
Comparison of analytical techniques to quantitate the capsid content of adeno-associated viral vectors	72
CRISPR-Cas9 gene editing of hepatitis B virus in chronically infected humanized mice	70
Acerola exosome-like nanovesicles to systemically deliver nucleic acid medicine via oral administration	63
qPCR and qRT-PCR analysis: Regulatory points to consider when conducting biodistribution and vector shedding studies	60
Mechanistic model for production of recombinant adeno-associated virus via triple transfection of HEK293 cells	55
High Levels of Frataxin Overexpression Lead to Mitochondrial and Cardiac Toxicity in Mouse Models	47
piggyBac system to co-express NKG2D CAR and IL-15 to augment the in vivo persistence and anti-AML activity of human peripheral blood NK cells	47
Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry	46
Development of a scalable and robust AEX method for enriched rAAV preparations in genome-containing VCs of serotypes 5, 6, 8, and 9	45
Pharmacological interventions enhance virus-free generation of TRAC-replaced CAR T cells	44
Enhanced expression of immune checkpoint receptors during SARS-CoV-2 viral infection	43
Anion-exchange HPLC assay for separation and quantification of empty and full capsids in multiple adeno-associated virus serotypes	42
Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations	42
AAV-S: A versatile capsid variant for transduction of mouse and primate inner ear	41
The impact of lentiviral vector genome size and producer cell genomic to gag-pol mRNA ratios on packaging efficiency and titre	41
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy	40
Characterization of AAV-mediated dorsal root ganglionopathy	40
Characterization of AAV-Specific Affinity Ligands: Consequences for Vector Purification and Development Strategies	38
Transplantation of miPSC/mESC-derived retinal ganglion cells into healthy and glaucomatous retinas	36
Automated generation of gene-edited CAR T cells at clinical scale	36
Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD	35
A flexible, thermostable nanostructured lipid carrier platform for RNA vaccine delivery	34

Exosomes and organ-specific metastasis	34
Clinical Development of Gene Therapies: The First Three Decades and Counting	33
Immunogenicity assessment of AAV-based gene therapies: An IQ consortium industry white paper	33
Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients	31
Assessment of genome packaging in AAVs using Orbitrap-based charge-detection mass spectrometry	31
Systemic and local immune responses to intraocular AAV vector administration in non-human primates	30
Rapid Lentiviral Vector Producer Cell Line Generation Using a Single DNA Construct	30
Applying machine learning to predict viral assembly for adeno-associated virus capsid libraries	30
Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec	29
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors	29
Applying a clinical lens to animal models of CAR-T cell therapies	29
Generation of hypoimmunogenic induced pluripotent stem cells by CRISPR-Cas9 system and detailed evaluation for clinical application	29
Monolayer platform using human biopsy-derived duodenal organoids for pharmaceutical research	29
Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses	29
Enriching leukapheresis improves T cell activation and transduction efficiency during CAR T processing	29
In vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system	29
Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease	29
Promoter usage regulating the surface density of CAR molecules may modulate the kinetics of CAR-T cells in vivo	28
Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues	28
Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease	28
In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency	27
Impact of intracerebroventricular enzyme replacement therapy in patients with neuronopathic mucopolysaccharidosis type II	27
Lentiviral and adeno-associated vectors efficiently transduce mouse T lymphocytes when targeted to murine CD8	27
Structural basis for the neurotropic AAV9 and the engineered AAVPHP.eB recognition with cellular receptors	27
Chemically Defined, High-Density Insect Cell-Based Expression System for Scalable AAV Vector Production	27
Engineered extracellular vesicles directed to the spike protein inhibit SARS-CoV-2	26
Current landscape of clinical development and approval of advanced therapies	26
A qPCR Method for AAV Genome Titer with ddPCR-Level of Accuracy and Precision	26
iMATCH: an integrated modular assembly system for therapeutic combination high-capacity adenovirus gene therapy	25
Multiplex CRISPR/Cas9 genome editing in hematopoietic stem cells for fetal hemoglobin reinduction generates chromosomal translocations	25
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	25
A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation	25
A third dose of the unmodified COVID-19 mRNA vaccine CVnCoV enhances quality and quantity of immune responses	25
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	24
In Situ Detection of Adeno-associated Viral Vector Genomes with SABER-FISH	24
Cell therapy with hiPSC-derived RPE cells and RPCs prevents visual function loss in a rat model of retinal degeneration	24
In Vitro and In Vivo Amenability to Migalastat in Fabry Disease	24
Inhibition of KDM1A activity restores adult neurogenesis and improves hippocampal memory in a mouse model of Kabuki syndrome	24
Lentiviral Vector Production from a Stable Packaging Cell Line Using a Packed Bed Bioreactor	24
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina	24
Gene therapy strategies for idiopathic pulmonary fibrosis: recent advances, current challenges, and future directions	24
Laboratory-Scale Lentiviral Vector Production and Purification for Enhanced Ex Vivo and In Vivo Genetic Engineering	23
Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8	23
Improved diabetic wound healing by LFcinB is associated with relevant changes in the skin immune response and microbiota	23
Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors	23
Systematic improvements in lentiviral transduction of primary human natural killer cells undergoing ex vivo expansion	23
Engineering and In Vitro Selection of a Novel AAV3B Variant with High Hepatocyte Tropism and Reduced Seroreactivity	23
Antigen modifications improve nucleoside-modified mRNA-based influenza virus vaccines in mice	23
Analysis of thermally driven structural changes, genome release, disassembly, and aggregation of recombinant AAV by CDMS	23
Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates	22

Depletion of high-content CD14+ cells from apheresis products is critical for successful transduction and expansion of CAR T cells during large-scale cGMP manufacturing	22
Molecular subtyping and functional validation of TTK, TPX2, UBE2C, and LRP8 in sensitivity of TNBC to paclitaxel	22
Molecular analysis of AAV5-hFVIII-SQ vector-genome-processing kinetics in transduced mouse and nonhuman primate livers	21
Implications of circulating neurofilaments for spinal muscular atrophy treatment early in life: A case series	21
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	21
Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells	21
Optimization of AAV6 transduction enhances site-specific genome editing of primary human lymphocytes	21
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal	21
CRISPAltRations: A validated cloud-based approach for interrogation of double-strand break repair mediated by CRISPR genome editing	21
Adipose Tissue: An Emerging Target for Adeno-associated Viral Vectors	21
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping	20
Co-transducing B7H3 CAR-NK cells with the DNR preserves their cytolytic function against GBM in the presence of exogenous TGF-β	20
Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse	20
Circulating neurofilament light chain as a promising biomarker of AAV-induced dorsal root ganglia toxicity in nonclinical toxicology species	20
Advances and challenges in adeno-associated viral inner-ear gene therapy for sensorineural hearing loss	20
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone	20
Autologous antigen-presenting cells efficiently expand piggyBac transposon CAR-T cells with predominant memory phenotype	20
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia	20
Directed Evolution of AAV Serotype 5 for Increased Hepatocyte Transduction and Retained Low Humoral Seroreactivity	20
Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation	19
A pro-inflammatory mediator USP11 enhances the stability of p53 and inhibits KLF2 in intracerebral hemorrhage	19
AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice	18
Analytical band centrifugation for the separation and quantification of empty and full AAV particles	18
CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease	18
A hierarchical and collaborative BRD4/CEBPD partnership governs vascular smooth muscle cell inflammation	18
Clinically relevant T cell expansion media activate distinct metabolic programs uncoupled from cellular function	17
Accelerating clinical-scale production of BCMA CAR T cells with defined maturation stages	17
SARS-CoV-2 infection increases the gene expression profile for Alzheimer’s disease risk	17
Spatiotemporal in vivo tracking of polyclonal human regulatory T cells (Tregs) reveals a role for innate immune cells in Treg transplant recruitment	17
Engineered mesenchymal stromal cell therapy during human lung ex vivo lung perfusion is compromised by acidic lung microenvironment	17
Comprehensive and systemic optimization for improving the yield of SARS-CoV-2 spike pseudotyped virus	17
Discovery of key genes as novel biomarkers specifically associated with HPV-negative cervical cancer	17
Development and Optimization of a Hydrophobic Interaction Chromatography-Based Method of AAV Harvest, Capture, and Recovery	17
Plasmodium vivax latent liver infection is characterized by persistent hypnozoites, hypnozoite-derived schizonts, and time-dependent efficacy of primaquine	17
AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model	17
Manufacturing NKG2D CAR-T cells with piggyBac transposon vectors and K562 artificial antigen-presenting cells	17
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	17
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method	17
GJB2 gene therapy and conditional deletion reveal developmental stage-dependent effects on inner ear structure and function	16
Visceral adipose tissue-directed FGF21 gene therapy improves metabolic and immune health in BTBR mice	16
Adipose-derived stem cells protect motor neurons and reduce glial activation in both in vitro and in vivo models of ALS	16
Differential T cell immune responses to deamidated adeno-associated virus vector	16
Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis	16
A systematic comparison of optogenetic approaches to visual restoration	16
Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina	16
UTX/KDM6A deletion promotes the recovery of spinal cord injury by epigenetically triggering intrinsic neural regeneration	16
Human-specific GAPDH qRT-PCR is an accurate and sensitive method of xenograft metastasis quantification	16
Cardiotoxicity assessment using 3D vascularized cardiac tissue consisting of human iPSC-derived cardiomyocytes and fibroblasts	16
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids	16
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs	16
Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics	15
Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity	15
Repeat Dosing of AAV2.5T to Ferret Lungs Elicits an Antibody Response That Diminishes Transduction in an Age-Dependent Manner	15
CRISPR-Cas9-mediated gene disruption of HIV-1 co-receptors confers broad resistance to infection in human T cells and humanized mice	15
Decrease in Angiotensin-Converting Enzyme activity but not concentration in plasma/lungs in COVID-19 patients offers clues for diagnosis/treatment	15
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	15
A universal strategy for AAV delivery of base editors to correct genetic point mutations in neonatal PKU mice	15
IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy	15
In vivo optogenetic stimulation of the primate retina activates the visual cortex after long-term transduction	15
MTA1, a Target of Resveratrol, Promotes Epithelial-Mesenchymal Transition of Endometriosis via ZEB2	15
Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model	15
Hypoxia-directed tumor targeting of CRISPR-Cas9 and HSV-TK suicide gene therapy using lipid nanoparticles	15
CRISPR-Mediated Base Conversion Allows Discriminatory Depletion of Endogenous T Cell Receptors for Enhanced Synthetic Immunity	15
Alginate hydrogel polymers enable efficient delivery of a vascular-targeted AAV vector into aortic tissue	15
Targeted knockdown of the adenosine A2A receptor by lipid NPs rescues the chemotaxis of head and neck cancer memory T cells	15
Consensus guidelines for the validation of qRT-PCR assays in clinical research by the CardioRNA consortium	14
A human surfactant B deficiency air-liquid interface cell culture model suitable for gene therapy applications	14
Synapsin-caveolin-1 gene therapy preserves neuronal and synaptic morphology and prevents neurodegeneration in a mouse model of AD	14
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes	14
Efficient treatment of a preclinical inflammatory bowel disease model with engineered bacteria	14
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors	14
AAV9-mediated gene delivery of MCT1 to oligodendrocytes does not provide a therapeutic benefit in a mouse model of ALS	14
High concordance of ELISA and neutralization assays allows for the detection of antibodies to individual AAV serotypes	14
Pre-existing anti-drug antibodies in Fabry disease show less affinity for pegunigalsidase alfa	14
Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice	14
Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector	14
Tetravalent Bispecific Tandem Antibodies Improve Brain Exposure and Efficacy in an Amyloid Transgenic Mouse Model	14
Production of rAAV by plasmid transfection induces antiviral and inflammatory responses in suspension HEK293 cells	14
Discovery of bone morphogenetic protein 7-derived peptide sequences that attenuate the human osteoarthritic chondrocyte phenotype	14
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	14

Improved safety of induced pluripotent stem cell-derived antigen-presenting cell-based cancer immunotherapy	13
Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome	13
Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease	13
Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapy	13
Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice	13
A sensitive and reproducible cell-based assay via secNanoLuc to detect neutralizing antibody against adeno-associated virus vector capsid	13
Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector	13
Potent programmable antiviral against dengue virus in primary human cells by Cas13b RNP with short spacer and delivery by VLP	13
Meeting FDA Guidance recommendations for replication-competent virus and insertional oncogenesis testing	13
Modeling Transient Abnormal Myelopoiesis Using Induced Pluripotent Stem Cells and CRISPR/Cas9 Technology	13
In vivo CRISPR-Cas9 inhibition of hepatic LDH as treatment of primary hyperoxaluria	13
Cytokine and reactivity profiles in SLE patients following anti-CD19 CART therapy	13
The seroprevalence of neutralizing antibodies against the adeno-associated virus capsids in Japanese hemophiliacs	13
Comparison of highly pure rAAV9 vector stocks produced in suspension by PEI transfection or HSV infection reveals striking quantitative and qualitative differences	13
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	13
Directed evolution of AAV accounting for long-term and enhanced transduction of cardiovascular endothelial cells in vivo	13
Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard	13
Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device	13
Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping	13
Targeting the lung epithelium after intravenous delivery by directed evolution of underexplored sites on the AAV capsid	13
Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A	13
Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector	13
Effect of epitope variant co-delivery on the depth of CD8 T cell responses induced by HIV-1 conserved mosaic vaccines	13
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency	12
Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation	12
The p53 challenge of hematopoietic stem cell gene editing	12
Tacrolimus-resistant SARS-CoV-2-specific T cell products to prevent and treat severe COVID-19 in immunosuppressed patients	12
Self-amplifying mRNA SARS-CoV-2 vaccines raise cross-reactive immune response to variants and prevent infection in animal models	12
Regeneration of Tumor-Antigen-Specific Cytotoxic T Lymphocytes from iPSCs Transduced with Exogenous TCR Genes	12
Inducible caspase-9 suicide gene under control of endogenous oct4 to safeguard mouse and human pluripotent stem cell therapy	12
Unique somatic variants in DNA from urine exosomes of individuals with bladder cancer	12
Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective	12
Engineered Antigen-Specific T Cells Secreting Broadly Neutralizing Antibodies: Combining Innate and Adaptive Immune Response against HIV	12
Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector	12
Ad- and AAV8-mediated ABCA1 gene therapy in a murine model with retinal ischemia/reperfusion injuries	12
CReVIS-Seq: A highly accurate and multiplexable method for genome-wide mapping of lentiviral integration sites	12
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study	12
Low endotoxin E. coli strain-derived plasmids reduce rAAV vector-mediated immune responses both in vitro and in vivo	12
AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study	12
Genetically edited hepatic cells expressing the NTCP-S267F variant are resistant to hepatitis B virus infection	12
Targeting the Apoa1 locus for liver-directed gene therapy	12
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	12
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series	11
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	11
High value of 64Cu as a tool to evaluate the restoration of physiological copper excretion after gene therapy in Wilson’s disease	11
Synthetic Prostacyclin Agonist Attenuates Pressure-Overloaded Cardiac Fibrosis by Inhibiting FMT	11
Genetically blocking HPD via CRISPR-Cas9 protects against lethal liver injury in a pig model of tyrosinemia type I	11
Long-Term Metabolic Correction of Phenylketonuria by AAV-Delivered Phenylalanine Amino Lyase	11
NaCl and KCl mediate log increase in AAV vector particles and infectious titers in a specific/timely manner with the HSV platform	11
In vivo generation of CAR T cells in the presence of human myeloid cells	11
A combinatorial CRISPR-Cas12a attack on HIV DNA	11
The sodium/glucose cotransporters as potential therapeutic targets for CF lung diseases revealed by human lung organoid swelling assay	11
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1	11
Substantial restoration of night vision in adult mice with congenital stationary night blindness	11
Gene therapy restores adipose tissue and metabolic health in a pre-clinical mouse model of lipodystrophy	11
miR-1254 induced by NESG1 inactivates HDGF/DDX5-stimulated nuclear translocation of β-catenin and suppresses NPC metastasis	11
Fast and high-throughput LC-MS characterization, and peptide mapping of engineered AAV capsids using LC-MS/MS	11
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice	11
AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder	11
Structural basis of receptor usage by the engineered capsid AAV-PHP.eB	11
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs	11
AAV-mediated BMP7 gene therapy counteracts insulin resistance and obesity	11
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	11
High-titer AAV disrupts cerebrovascular integrity and induces lymphocyte infiltration in adult mouse brain	11
Machine learning prediction of methionine and tryptophan photooxidation susceptibility	11
Galactosialidosis: preclinical enzyme replacement therapy in a mouse model of the disease, a proof of concept	11
Tips and tricks for successfully culturing and adapting human induced pluripotent stem cells	11
Formatting and gene-based delivery of a human PD-L1 single domain antibody for immune checkpoint blockade	11
HDAd6/35++ - A new helper-dependent adenovirus vector platform for in vivo transduction of hematopoietic stem cells	11
siRNA targeting Schlemm’s canal endothelial tight junctions enhances outflow facility and reduces IOP in a steroid-induced OHT rodent model	11
In vivo genome editing at the albumin locus to treat methylmalonic acidemia	11
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution	10
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model	10
AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery	10
Genetic engineering of human and mouse CD4+ and CD8+ Tregs using lentiviral vectors encoding chimeric antigen receptors	10
HIV-based lentiviral vectors: Origin and sequence differences	10
Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients	10
Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation	10
Immunological evaluation of an inactivated SARS-CoV-2 vaccine in rhesus macaques	10
Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice	10
AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates	10
Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps	10
Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis	10
Clinical holds for cell and gene therapy trials: Risks, impact, and lessons learned	10
A Novel Proteomic Method Reveals NLS Tagging of T-DM1 Contravenes Classical Nuclear Transport in a Model of HER2-Positive Breast Cancer	10
Tumor antigen-loaded AAV vaccine drives protective immunity in a melanoma animal model	10
Allometric-like scaling of AAV gene therapy for systemic protein delivery	10
VEGFR-1/Flt-1 inhibition increases angiogenesis and improves muscle function in a mouse model of Duchenne muscular dystrophy	10
Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression	10
Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expression vector system	9
A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes	9
Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease	9
Optimization of S. aureus dCas9 and CRISPRi Elements for a Single Adeno-Associated Virus that Targets an Endogenous Gene	9
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	9
Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment	9