Molecular Therapy-Methods & Clinical Development

Article	Citations
Molecular earplugs to protect the inner ear	88
Detection and quantification of integrated vector copy number by multiplex droplet digital PCR in dual-transduced CAR T cells	83
Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation	67
Breaching the blood-brain barrier: AAV triggers dose-dependent toxicity in the brain	59
Generation of hepatitis C virus–resistant liver cells by genome editing–mediated stable expression of RNA aptamer	59
Evaluation of parameters for efficient purification and long-term storage of herpes simplex virus-based vectors	56
AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency	53
Engineering new metabolic pathways in isolated cells for the degradation of guanidinoacetic acid and simultaneous production of creatine	53
Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease	51
Neurofilament light chain and dorsal root ganglia injury after adeno-associated virus 9 gene therapy in nonhuman primates	49
Large-scale manufacturing of base-edited chimeric antigen receptor T cells	47
Thank you to our 2024 reviewers	46
Insights in AAV-mediated antigen-specific immunity and a strategy for AAV vaccine dose reduction through AAV-extracellular vesicle association	46
Highly branched poly β-amino ester/CpG-depleted CFTR plasmid nanoparticles for non-viral gene therapy in lung cystic fibrosis disease	46
Characterization of drusen formation in a primary porcine tissue culture model of dry AMD	46
Preclinical efficacy and safety of adeno-associated virus 5 alpha-galactosidase: A gene therapy for Fabry disease	43
Cryopreserved anti-CD22 and bispecific anti-CD19/22 CAR T cells are as effective as freshly infused cells	41
Packaging cells for lentiviral vectors generated using the cumate and coumermycin gene induction systems and nanowell single-cell cloning	41
αFAP-specific nanobodies mediate a highly precise retargeting of modified AAV2 capsids thereby enabling specific transduction of tumor tissues	41
A third dose of the unmodified COVID-19 mRNA vaccine CVnCoV enhances quality and quantity of immune responses	40
Coagulation factor IX gene transfer to non-human primates using engineered AAV3 capsid and hepatic optimized expression cassette	39
DNA contamination within recombinant adeno-associated virus preparations correlates with decreased CD34+ cell clonogenic potential	39
Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series	38
Novel TCR-like CAR-T cells targeting an HLA∗0201-restricted SSX2 epitope display strong activity against acute myeloid leukemia	38
miRNA-mediated control of exogenous OCT4 during mesenchymal-epithelial transition increases measles vector reprogramming efficiency	37

Gene therapy for Friedreich ataxia: Too much, too little, or just right?	37
AUF1 gene transfer increases exercise performance and improves skeletal muscle deficit in adult mice	36
Safety and efficiency modifications of SIV-based integrase-defective lentiviral vectors for immunization	35
SAG therapy restores bone growth and reduces enchondroma incidence in a model of skeletal chondrodysplasias caused by Ihh deficiency	35
Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model	33
RNA-seq analysis of the human surfactant air-liquid interface culture reveals alveolar type II cell-like transcriptome	33
Follistatin-like 1 promotes proliferation of matured human hypoxic iPSC-cardiomyocytes and is secreted by cardiac fibroblasts	31
Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease	30
Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors	30
Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice	29
Mechanistic modeling explains the production dynamics of recombinant adeno-associated virus with the baculovirus expression vector system	29
Genome length determination in adeno-associated virus vectors with mass photometry	29
Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia	28
Preclinical specificity & activity of a fully human 41BB-expressing anti-CD19 CART- therapy for treatment-resistant autoimmune disease	28
Anti-tau intrabodies: From anti-tau immunoglobulins to the development of functional scFv intrabodies	28
First use of adeno-associated viruses in the human inner ear	28
Persistent tailoring of MSC activation through genetic priming	27
Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment	27
AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life	27
A randomized, double-blind phase 2b trial to evaluate efficacy of ChAd63-KH for treatment of post kala-azar dermal leishmaniasis	27
Glutaredoxin-1 modulates the NF-κB signaling pathway to activate inducible nitric oxide synthase in experimental necrotizing enterocolitis	27
Adeno-associated virus serotype 9 antibodies in neonates and young children: Seroprevalence and kinetics	27
AAVolve: Concatenated long-read deep sequencing enables whole capsid tracking during shuffled AAV library selection	26
Efficient generation of liver sinusoidal endothelial-like cells secreting coagulation factor VIII from human induced pluripotent stem cells	26
Seven-year follow-up of durability and safety of AAV CNS gene therapy for a lysosomal storage disorder in a large animal	26
Quantification of cell-free DNA for the analysis of CD19-CAR-T cells during lymphoma treatment	25
Perspectives of the Friedreich ataxia community on gene therapy clinical trials	25
Expression of NMNAT1 in the photoreceptors is sufficient to prevent NMNAT1-associated retinal degeneration	25
Nanodysferlins support membrane repair and binding to TRIM72/MG53 but do not localize to t-tubules or stabilize Ca2+ signaling	24
Durable transgene expression and efficient re-administration after rAAV2.5T-mediated fCFTRΔR gene delivery to adult ferret lungs	24
Impact of an autophagy-inducing peptide on immunogenicity and protection efficacy of an adenovirus-vectored SARS-CoV-2 vaccine	24
A C1qTNF3 collagen domain fusion chaperones diverse secreted proteins and anti-Aβ scFvs: Applications for gene therapies	24
Differential T cell immune responses to deamidated adeno-associated virus vector	22
Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes	22
Targeted knockdown of the adenosine A2A receptor by lipid NPs rescues the chemotaxis of head and neck cancer memory T cells	22
Induced dendritic cells co-expressing GM-CSF/IFN-α/tWT1 priming T and B cells and automated manufacturing to boost GvL	22
Size-exclusion chromatography as a multi-attribute method for process and product characterization of adeno-associated virus	22
Adeno-associated virus serotype 9 antibody seroprevalence for patients in the United States with spinal muscular atrophy	21
A clinically viable approach to restoring visual function using optogenetic gene therapy	21
Haplotype-specific insertion-deletion variations for allele-specific targeting in Huntington's disease	21
Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice	21
Intrathecal delivery of a bicistronic AAV9 vector expressing β-hexosaminidase A corrects Sandhoff disease in a murine model: A dosage study	21
Preclinical safety assessment of MV-s-NAP, a novel oncolytic measles virus strain armed with an H. pylori immunostimulatory bacterial transgene	21
Protein phosphatase 2A anchoring disruptor gene therapy for familial dilated cardiomyopathy	21
Intracellular RNase activity dampens zinc finger nuclease-mediated gene editing in hematopoietic stem and progenitor cells	20
TAZing down metabolic mayhem: siRNAs against liver inflammation and fibrosis in humanized mice	20
Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies	20
Combined clarification and affinity capture using magnetic resin enables efficient separation of rAAV5 from cell lysate	20
Liter-scale manufacturing of shelf-stable plasmid DNA/PEI transfection particles for viral vector production	20
Decrease in Angiotensin-Converting Enzyme activity but not concentration in plasma/lungs in COVID-19 patients offers clues for diagnosis/treatment	20

Hexon modification of human adenovirus type 5 vectors enables efficient transduction of human multipotent mesenchymal stromal cells	20
Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics	20
A positive take on negative selection for CAR-T manufacturing	20
Selective RNAi silencing of Schwann cell Piezo1 alleviates mechanical hypersensitization following peripheral nerve injury	19
Identification of the role of SNARE proteins in rAAV vector production through interaction with the viral MAAP	19
Recombinant AAV batch profiling by nanopore sequencing elucidates product-related DNA impurities and vector genome length distribution	19
Global regulatory progress in delivering on the promise of gene therapies for unmet medical needs	19
Nonclinical study of ixo-vec gene therapy for nAMD supports efficacy for a human dose of 6E10 vg/eye and staggered dosing of fellow eyes	19
Deciphering key parameters enhancing lentiviral vector producer cells yields: Vector components copy number and expression	19
A novel platform for engineered AAV-based vaccines	19
CRISPR-Cas9-mediated genome editing delivered by a single AAV9 vector inhibits HSV-1 reactivation in a latent rabbit keratitis model	19
Systemic delivery of AAV5, AAV8, and AAV9 packaging a C5-12-microdystrophin-FLAG expression cassette in non-human primates	18
Tumor antigen-loaded AAV vaccine drives protective immunity in a melanoma animal model	18
Peptide-encoding gene transfer to modulate intracellular protein-protein interactions	18
AAV vector production: Troublesome host innate responses in another setting	18
Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus	18
Preexisting antibody assays for gene therapy: Considerations on patient selection cutoffs and companion diagnostic requirements	18
PCR-based analytics of gene therapies using adeno-associated virus vectors: Considerations for cGMP method development	18
AAV-mediated expression of HLA-G for the prevention of experimental ocular graft vs. host disease	18
Characterization and effective expansion of CD4−CD8− TCRαβ+ T cells from individuals living with type 1 diabetes	18
Engineering a highly durable adeno-associated virus receptor for analytical applications	18
Cell-penetrating peptides enhance the transduction of adeno-associated virus serotype 9 in the central nervous system	18
Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis	18
Treating late-onset Tay Sachs disease: Brain delivery with a dual trojan horse protein	17
Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness	17
Fast HPLC-based affinity method to determine capsid titer and full/empty ratio of adeno-associated viral vectors	17
Monitoring cell-mediated immune responses in AAV gene therapy clinical trials using a validated IFN-γ ELISpot method	17
The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome	17
Adeno-associated viral vector serotype 9-based gene replacement therapy for SURF1-related Leigh syndrome	17
Producing high-quantity and high-quality recombinant adeno-associated virus by low-cis triple transfection	17
Genetic surgery for a cystic fibrosis-causing splicing mutation	17
Development of a gene edited next-generation hematopoietic cell transplant to enable acute myeloid leukemia treatment by solving off-tumor toxicity	17
Production of recombinant adeno-associated virus 5 using a novel self-attenuating adenovirus production platform	17
An amplification-free CRISPR-Cas12a assay for titer determination and composition analysis of the rAAV genome	17
AAV vector production: Troublesome host innate responses in another setting	17
Optimizing regulatory frameworks for gene therapies in rare diseases: Challenges and solutions	16
Hypothalamic AAV-BDNF gene therapy improves metabolic function and behavior in the Magel2-null mouse model of Prader-Willi syndrome	16
Nonclinical pharmacokinetics and biodistribution of VSV-GP using methods to decouple input drug disposition and viral replication	16
Medicaid coverage practices for approved gene and cell therapies: Existing barriers and proposed policy solutions	16
Dasatinib is a potent enhancer for CAR T cell generation by CD3-targeted lentiviral vectors	16
Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations	16
A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice	16
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration	16
Off-the-shelf allogeneic natural killer cells for the treatment of COVID-19	16
Exosomes and organ-specific metastasis	16
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy	16
Patient-specific responses to SMN2 splice-modifying treatments in spinal muscular atrophy fibroblasts	16
Enhancement of recombinant adeno-associated virus activity by improved stoichiometry and homogeneity of capsid protein assembly	15
A T cell-targeted multi-antigen vaccine generates robust cellular and humoral immunity against SARS-CoV-2 infection	15
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography	15
Studying how administration route and dose regulates antibody generation against LNPs for mRNA delivery with single-particle resolution	15
A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes	15
Channelrhodopsin fluorescent tag replacement for clinical translation of optogenetic hearing restoration	15
Micro-dystrophin gene therapy demonstrates long-term cardiac efficacy in a severe Duchenne muscular dystrophy model	15
Efficient autocrine and paracrine signaling explain the osteogenic superiority of transgenic BMP-2 over rhBMP-2	15
Lipid nanoparticle mRNA systems containing high levels of sphingomyelin engender higher protein expression in hepatic and extra-hepatic tissues	15
Delivery of nVEGFi using AAV8 for the treatment of neovascular age-related macular degeneration	15
A pro-inflammatory mediator USP11 enhances the stability of p53 and inhibits KLF2 in intracerebral hemorrhage	14
Production and characterization of an AAV1-VP3-only capsid: An analytical benchmark standard	14
Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1	14
Multiparametric domain insertional profiling of adeno-associated virus VP1	14
Efficient long-term multilineage engraftment of CD33-edited hematopoietic stem/progenitor cells in nonhuman primates	14
Lipid nanoparticles outperform electroporation in mRNA-based CAR T cell engineering	14
Assessing production variability in empty and filled adeno-associated viruses by single molecule mass analyses	14
AAV8 gene therapy reverses cardiac pathology and prevents early mortality in a mouse model of Friedreich’s ataxia	13
CRISPR-Cas9 correction of a nonsense mutation in LCA5 rescues lebercilin expression and localization in human retinal organoids	13
Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency	13
Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy	13
A Library-Based Screening Strategy for the Identification of DARPins as Ligands for Receptor-Targeted AAV and Lentiviral Vectors	13
Magnetic bead-sensitized optoporation coupled with antibodies-based activation for mRNA CAR-T cell manufacturing	13
Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I	13
Preclinical development of TAK-754, a high-performance AAV8-based vector expressing coagulation factor VIII	13
Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy	13
Can mitochondria brown the lower-limb adipocytes?	13
Peripheral leukemia burden at time of apheresis negatively affects the clinical efficacy of CART19 in refractory or relapsed B-ALL	13
Development of cell lines with increased susceptibility to diverse adeno-associated viral vectors to enable in vitro potency assays	13
Lipid nanoparticle encapsulation of a Delta spike-CD40L DNA vaccine improves effectiveness against Omicron challenge in Syrian hamsters	13
Automated manufacture of ΔNPM1 TCR-engineered T cells for AML therapy	13
Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model	13
Measurement solutions and standards for advanced therapy	13

Evaluation of two in vitro assays for tumorigenicity assessment of CRISPR-Cas9 genome-edited cells	13
Intravenous esketamine in pediatric Rett syndrome: An open-label, early phase 1 pilot study	13
Validation of a quantitative cell-based relative potency assay for LUXTURNA	13
Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution	12
An HPLC-SEC-based rapid quantification method for vesicular stomatitis virus particles to facilitate process development	12
Progress toward the challenging goal of HDR-based gene editing for hyper-IgM syndrome	12
Sendai F/HN pseudotyped lentiviral vector transduces human ciliated and non-ciliated airway cells using α 2,3 sialylated receptors	12
Exogenous expression of ATP8, a mitochondrial encoded protein, from the nucleus in vivo	12
Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution	12
An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs	12
CAR-NK cells derived from cord blood originate mainly from CD56−CD7+CD34−HLA-DR−Lin− NK progenitor cells	12
Deconvolution of spatial sequencing provides accurate characterization of hESC-derived DA transplants in vivo	12
An AAV capsid increases transduction of striatum and a ChAT promoter allows selective cholinergic neuron transduction	12
Efficacy of HSV-TK/GCV system suicide gene therapy using SHED expressing modified HSV-TK against lung cancer brain metastases	12
Immunological evaluation of an inactivated SARS-CoV-2 vaccine in rhesus macaques	12
Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS	12
Synergy between Lactobacillus murinus and anti-PcrV antibody delivered in the airways to boost protection against Pseudomonas aeruginosa	12
Optogenetic targeting of AII amacrine cells restores retinal computations performed by the inner retina	11
From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia	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
Case report of selumetinib as a novel therapy in a neurofibromatosis type 2-associated ependymoma	11
Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9	11
Efficacy and muscle safety assessment of fukutin-related protein gene therapy	11
The sodium/glucose cotransporters as potential therapeutic targets for CF lung diseases revealed by human lung organoid swelling assay	11
Complete intra-laboratory validation of a LAL assay for bacterial endotoxin determination in EBV-specific cytotoxic T lymphocytes	11
Circulating neurofilaments to track dorsal root ganglion toxicity risks with AAV-mediated gene therapy	11
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog	11
Efficient production of inhibitor-free foamy virus glycoprotein-containing retroviral vectors by proteoglycan-deficient packaging cells	11
A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease	11
Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors	11
High-titer manufacturing of SARS-CoV-2 Spike-pseudotyped VSV in stirred-tank bioreactors	11
Quantification of full and empty particles of adeno-associated virus vectors via a novel dual fluorescence-linked immunosorbent assay	11
Human retina-in-a-dish: Unlocking the potential to study mechanisms of inherited retinal disease	11
The unknown impact of conditioning on HSC engraftment and clonal dynamics	11
Every little bit helps: A single-residue switch in a vascular AAV enables blood-brain barrier penetration	11
Consensus-driven target product profiles for curative sickle cell disease gene therapies	11
Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency	11
Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice	11
A robust and flexible baculovirus-insect cell system for AAV vector production with improved yield, capsid ratios and potency	10
Temporal insights into molecular and cellular responses during rAAV production in HEK293T cells	10
Delivery of non-viral naked DNA vectors to liver in small weaned pigs by hydrodynamic retrograde intrabiliary injection	10
Improved safety of induced pluripotent stem cell-derived antigen-presenting cell-based cancer immunotherapy	10
Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs	10
Testing the efficacy of a human full-length OPG-Fc analog in a severe model of cardiotoxin-induced skeletal muscle injury and repair	10
Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model	10
Endovascular transplantation of mRNA-enhanced mesenchymal stromal cells results in superior therapeutic protein expression in swine heart	10
Rapid, accurate mapping of transgene integration in viable rhesus macaque embryos using enhanced-specificity tagmentation-assisted PCR	10
Non-encapsidated miRNA contaminants found in AAV preparations	10
Combining CRISPR-Cas-mediated terminal resolution with a novel genetic workflow to achieve high-diversity adenoviral libraries	10
Design and validation of cell-based potency assays for frataxin supplementation treatments	10
LATE–a novel sensitive cell-based assay for the study of CRISPR/Cas9-related long-term adverse treatment effects	10
Unlocking DOE potential by selecting the most appropriate design for rAAV optimization	10
High throughput screening of novel AAV capsids identifies variants for transduction of adult NSCs within the subventricular zone	10
Widespread correction of brain pathology in feline alpha-mannosidosis by dose escalation of intracisternal AAV vector injection	10
Genetic alteration of SJ293TS cells and modification of serum-free media enhances lentiviral vector production	10
Discovery of bone morphogenetic protein 7-derived peptide sequences that attenuate the human osteoarthritic chondrocyte phenotype	9
In vivo genome editing at the albumin locus to treat methylmalonic acidemia	9
Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection	9
Effect of epitope variant co-delivery on the depth of CD8 T cell responses induced by HIV-1 conserved mosaic vaccines	9
New dawn of cellular therapies in autoimmune diseases	9
CRISPR-Cas9-mediated gene disruption of HIV-1 co-receptors confers broad resistance to infection in human T cells and humanized mice	9
Quantitative proteomic analysis of residual host cell protein retention across adeno-associated virus affinity chromatography	9
Directed evolution of AAV accounting for long-term and enhanced transduction of cardiovascular endothelial cells in vivo	9
Immune inactivation of anti-simian immunodeficiency virus chimeric antigen receptor T cells in rhesus macaques	9
Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients	9
Single-dose AAV vector gene immunotherapy to treat food allergy	9
Modulation of AAV transduction and integration targeting by topoisomerase poisons	9
An investigation of the immune epitopes of adeno-associated virus capsid-derived peptides among hemophilia patients	9
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys	9
In vitro characterization of engineered red blood cells as viral traps against HIV-1 and SARS-CoV-2	9
Adipose-derived stem cells protect motor neurons and reduce glial activation in both in vitro and in vivo models of ALS	9
Outcomes of progranulin gene therapy in the retina are dependent on time and route of delivery	9
Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice	9
AAV-mediated BMP7 gene therapy counteracts insulin resistance and obesity	9
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease	9
miR-223-3p and miR-24-3p as novel serum-based biomarkers for myotonic dystrophy type 1	9
An autonucleolytic suspension HEK293F host cell line for high-titer serum-free AAV5 and AAV9 production with reduced levels of DNA impurity	9
PAM-flexible dual base editor-mediated random mutagenesis and self-activation strategies to improve CRISPRa potency	8
Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy	8
Resolving hidden subpopulations of filled AAVs by probing capsid integrity	8
Voluntary wheel running complements microdystrophin gene therapy to improve muscle function in mdx mice	8
Current landscape of clinical development and approval of advanced therapies	8
Disseminating transformative gene and cell therapy research	8
Probing recombinant AAV capsid integrity and genome release after thermal stress by mass photometry	8
Sustained long-term disease correction in a murine model of MPSII following stem cell gene therapy	8
5′ Transgenes drive leaky expression of 3′ transgenes in Cre-inducible bi-cistronic vectors	8
A chimeric virus-based probe unambiguously detects live circulating tumor cells with high specificity and sensitivity	8
A sensitive AAV transduction inhibition assay assists evaluation of critical factors for detection and concordance of pre-existing antibodies	8
Optimization of anti-CD19 CAR T cell production for treatment of patients with chronic lymphocytic leukemia	8
Development of new adeno-associated virus capsid variants for targeted gene delivery to human cardiomyocytes	8
Evidence generation and reproducibility in cell and gene therapy research: A call to action	8
Self-amplifying mRNA bicistronic influenza vaccines raise cross-reactive immune responses in mice and prevent infection in ferrets	8
Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids	8
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II	8