OOIR: Observatory of International Research

Papers

(The median citation count of Gene Therapy is 4. The table below lists those papers that are above that threshold based on CrossRef citation counts [max. 250 papers]. The publications cover those that have been published in the past four years, i.e., from 2020-05-01 to 2024-05-01.)

Article	Citations
Self-amplifying RNA vaccines for infectious diseases	217
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany	149
Prime editing – an update on the field	75
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma	70
Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy	66
Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters	62
Gene therapy and gene correction: targets, progress, and challenges for treating human diseases	57
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair	41
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions	39
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	38
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system	33
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy	33
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	30
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	29
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells	28
Natural polyphenol assisted delivery of single-strand oligonucleotides by cationic polymers	27
Gene therapy for spinal muscular atrophy: the Qatari experience	27
miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A	26
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma	26
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics	26
An overview of development in gene therapeutics in China	25
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?	25
The stability of envelope-pseudotyped lentiviral vectors	24
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1	23

Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	22
Rational engineering of a functional CpG-free ITR for AAV gene therapy	22
LncRNA SNHG16 promotes colorectal cancer cell proliferation, migration, and epithelial–mesenchymal transition through miR-124-3p/MCP-1	22
Antiviral immunity and nucleic acid sensing in haematopoietic stem cell gene engineering	22
Retinal gene therapy: an eye-opener of the 21st century	21
Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation	21
Sensitization of ON-bipolar cells with ambient light activatable multi-characteristic opsin rescues vision in mice	20
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing	19
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	19
Antitumor effects of IL-12 and GM-CSF co-expressed in an engineered oncolytic HSV-1	18
Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells	18
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9	18
Sigma-1 receptor activity in primary sensory neurons is a critical driver of neuropathic pain	18
Large gene delivery to the retina with AAV vectors: are we there yet?	16
Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates	16
Maximizing lentiviral vector gene transfer in the CNS	16
Control of gene doping in human and horse sports	16
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma	16
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters	16
Treating Bietti crystalline dystrophy in a high-fat diet-exacerbated murine model using gene therapy	15
Exosomal microRNA-133b-3p from bone marrow mesenchymal stem cells inhibits angiogenesis and oxidative stress via FBN1 repression in diabetic retinopathy	15
Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1	15
Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery	15
Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses	14
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	14
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
Effective viral-mediated lung gene therapy: is airway surface preparation necessary?	14
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study	14
Novel tool to quantify with single-cell resolution the number of incoming AAV genomes co-expressed in the mouse nervous system	13
Advantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy	13
Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility	13
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome	13
Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application	13
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice	13
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies	13
Effects of oncolytic viruses and viral vectors on immunity in glioblastoma	13
Implications of hematopoietic stem cells heterogeneity for gene therapies	12
Curing SMA: Are we there yet?	12
Chitosan-coated Zn-metal-organic framework nanocomposites for effective targeted delivery of LNA-antisense miR-224 to colon tumor: in vitro studies	12
From macrophage biology to macrophage-based cellular immunotherapies	12
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
The transformative potential of HSC gene therapy as a genetic medicine	11
Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice	11
Easy and robust electrotransfection protocol for efficient ectopic gene expression and genome editing in human B cells	11
Long noncoding RNA CA3-AS1 suppresses gastric cancer migration and invasion by sponging miR-93-5p and targeting BTG3	11
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	11
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy	10
Screening for gene doping transgenes in horses via the use of massively parallel sequencing	10

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor	10
Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells	10
Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety	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
Recent development of AAV-based gene therapies for inner ear disorders	10
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	10
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
Successes and challenges in clinical gene therapy	9
Single-cell mapping of focused ultrasound-transfected brain	9
Lentiviral gene therapy vectors encoding VIP suppressed diabetes-related inflammation and augmented pancreatic beta-cell proliferation	9
Overexpression of miR-144-3p alleviates polycystic ovaries syndrome through targeting expression of HSP-70	9
Exon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells	9
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	9
Effect of connective tissue growth factor gene editing using adeno-associated virus–mediated CRISPR–Cas9 on rabbit glaucoma filtering surgery outcomes	9
Splicing mutations in the CFTR gene as therapeutic targets	8
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo	8
Gene therapy development in hearing research in China	8
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	8
Automated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients	8
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy	8
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders	8
Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?	8
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential	8
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	8
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	8
AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge	8
Sufficiency for inducible Caspase-9 safety switch in human pluripotent stem cells and disease cells	8
Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	8
Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2	7
Intra-cisterna magna delivery of an AAV vector with the GLUT1 promoter in a pig recapitulates the physiological expression of SLC2A1	7
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	7
Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep	7
Neutralisation of adeno-associated virus transduction by human vitreous humour	7
Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease	7
E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis	7
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	7
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles	7
Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells	7
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	7
Preconditioning of surgical pedicle flaps with DNA plasmid expressing hypoxia-inducible factor-1α (HIF-1α) promotes tissue viability	7
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	6
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	6
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	6
Approval and therapeutic value of gene therapies in the US and Europe	6
Insidious Insights: Implications of viral vector engineering for pathogen enhancement	6
The estimated annual financial impact of gene therapy in the United States	6
Enzymatically amplified linear dbDNATM as a rapid and scalable solution to industrial lentiviral vector manufacturing	6
Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo	6
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis	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
Promoting role of circ-Jarid2/miR-129-5p/Celf1 axis in cardiac hypertrophy	5
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage	5
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	5
Role and the molecular mechanism of lncRNA PTENP1 in regulating the proliferation and invasion of cervical cancer cells	5
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa	5
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence	5
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
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	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
CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy	4
Access to affordable medicines: obligations of universities and academic medical centers	4
Injection pressure levels for creating blebs during subretinal gene therapy	4
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain	4
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells	4
Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection	4
Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications	4
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy	4
Cerebral organoids as an in vitro model to study autism spectrum disorders	4
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	4
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway	4