Gene Therapy

Article	Citations
Self-amplifying RNA vaccines for infectious diseases	196
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany	143
Prime editing – an update on the field	71
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma	68
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy	65
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters	59
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases	52
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair	38
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions	34
Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device – the SERCA-LVAD TRIAL	34
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system	32
Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa	32
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy	31
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?	29
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice	28
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells	27
Gene therapy for spinal muscular atrophy: the Qatari experience	27
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	26
An overview of development in gene therapeutics in China	25
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?	24
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics	24
The stability of envelope-pseudotyped lentiviral vectors	24
Natural polyphenol assisted delivery of single-strand oligonucleotides by cationic polymers	23
LncRNA SNHG16 promotes colorectal cancer cell proliferation, migration, and epithelial–mesenchymal transition through miR-124-3p/MCP-1	22
miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A	22

AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1	22
Rational engineering of a functional CpG-free ITR for AAV gene therapy	21
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma	21
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	20
Retinal gene therapy: an eye-opener of the 21st century	20
Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice	19
Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation	19
Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells	18
Sigma-1 receptor activity in primary sensory neurons is a critical driver of neuropathic pain	18
Antiviral immunity and nucleic acid sensing in haematopoietic stem cell gene engineering	18
Knockdown of LINC00467 contributed to Axitinib sensitivity in hepatocellular carcinoma through miR-509-3p/PDGFRA axis	18
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing	18
MiR-140-5p inhibits oxidized low-density lipoprotein-induced oxidative stress and cell apoptosis via targeting toll-like receptor 4	16
Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates	16
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9	16
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	15
Antitumor effects of IL-12 and GM-CSF co-expressed in an engineered oncolytic HSV-1	15
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma	15
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery	15
Maximizing lentiviral vector gene transfer in the CNS	15
Large gene delivery to the retina with AAV vectors: are we there yet?	15
LncRNA SNHG16 induces proliferation and fibrogenesis via modulating miR-141-3p and CCND1 in diabetic nephropathy	14
Retroviral gene therapy in Germany with a view on previous experience and future perspectives	14
Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy	14
Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy	14
Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	14
AAV3-miRNA vectors for growth suppression of human hepatocellular carcinoma cells in vitro and human liver tumors in a murine xenograft model in vivo	14
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice	13
Exosomal microRNA-133b-3p from bone marrow mesenchymal stem cells inhibits angiogenesis and oxidative stress via FBN1 repression in diabetic retinopathy	13
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome	13
Control of gene doping in human and horse sports	13
Reactivation of FMR1 gene expression is a promising strategy for fragile X syndrome therapy	13
Advantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy	13
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1	13
Detection of non-targeted transgenes by whole-genome resequencing for gene-doping control	12
Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples	12
Effects of oncolytic viruses and viral vectors on immunity in glioblastoma	12
Treating osteoarthritis via gene therapy with rejuvenation factors	12
Implications of hematopoietic stem cells heterogeneity for gene therapies	12
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters	12
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	12
Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility	12
Novel tool to quantify with single-cell resolution the number of incoming AAV genomes co-expressed in the mouse nervous system	12
Effective viral-mediated lung gene therapy: is airway surface preparation necessary?	12
Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application	12
From macrophage biology to macrophage-based cellular immunotherapies	12
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study	11
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies	11
Long noncoding RNA CA3-AS1 suppresses gastric cancer migration and invasion by sponging miR-93-5p and targeting BTG3	11
Chitosan-coated Zn-metal-organic framework nanocomposites for effective targeted delivery of LNA-antisense miR-224 to colon tumor: in vitro studies	11

Easy and robust electrotransfection protocol for efficient ectopic gene expression and genome editing in human B cells	11
Gene therapy for cardiovascular diseases in China: basic research	11
Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety	10
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice	10
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy	10
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons	10
Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment	10
Curing SMA: Are we there yet?	10
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	9
Single-cell mapping of focused ultrasound-transfected brain	9
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	9
Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells	9
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	9
Silencing of long non-coding RNA FOXD2-AS1 inhibits the progression of gallbladder cancer by mediating methylation of MLH1	9
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA	9
Overexpression of miR-144-3p alleviates polycystic ovaries syndrome through targeting expression of HSP-70	9
Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor	9
Screening for gene doping transgenes in horses via the use of massively parallel sequencing	9
Recent development of AAV-based gene therapies for inner ear disorders	8
Toward a new framework for the development of individualized therapies	8
Lentiviral gene therapy vectors encoding VIP suppressed diabetes-related inflammation and augmented pancreatic beta-cell proliferation	8
Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	8
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy	8
Exon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells	8
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	8
Effect of connective tissue growth factor gene editing using adeno-associated virus–mediated CRISPR–Cas9 on rabbit glaucoma filtering surgery outcomes	8
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders	8
Automated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients	8
Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells	7
Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?	7
Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2	7
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	7
E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis	7
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential	7
‘Mini’ U6 Pol III promoter exhibits nucleosome redundancy and supports multiplexed coupling of CRISPR/Cas9 effects	7
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease	7
Gene therapy development in hearing research in China	7
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	7
Potent CAR-T cells engineered with Sleeping Beauty transposon vectors display a central memory phenotype	7
Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1	7
The transformative potential of HSC gene therapy as a genetic medicine	6
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles	6
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo	6
Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep	6
Neutralisation of adeno-associated virus transduction by human vitreous humour	6
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	6
Sufficiency for inducible Caspase-9 safety switch in human pluripotent stem cells and disease cells	6
Preconditioning of surgical pedicle flaps with DNA plasmid expressing hypoxia-inducible factor-1α (HIF-1α) promotes tissue viability	6
Successes and challenges in clinical gene therapy	6
Gene conversion following CRISPR/Cas9 DNA cleavage: an overlooked effect	6
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	6
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	6
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia	5
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models	5
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS	5
The estimated annual financial impact of gene therapy in the United States	5
Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice	5
Promoting role of circ-Jarid2/miR-129-5p/Celf1 axis in cardiac hypertrophy	5
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa	5
Development of a laboratory scalable process for enhancing lentivirus production by transient transfection of HEK293 adherent cultures	5
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	5
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	5
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	5
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis	5
Approval and therapeutic value of gene therapies in the US and Europe	5
Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo	5
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage	5
Insidious Insights: Implications of viral vector engineering for pathogen enhancement	5
AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge	5
AAV-mediated cardiac gene transfer of wild-type desmin in mouse models for recessive desminopathies	5
Enzymatically amplified linear dbDNATM as a rapid and scalable solution to industrial lentiviral vector manufacturing	4
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing	4
Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?	4
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	4
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	4
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway	4
Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	4
Splicing mutations in the CFTR gene as therapeutic targets	4
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	4
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	4

Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection	4
Access to affordable medicines: obligations of universities and academic medical centers	4
Role and the molecular mechanism of lncRNA PTENP1 in regulating the proliferation and invasion of cervical cancer cells	4
Cerebral organoids as an in vitro model to study autism spectrum disorders	4
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	4
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells	4