OOIR: Observatory of International Research

Papers

(The TQCC of Gene Therapy is 9. 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 2020-11-01 to 2024-11-01.)

Article	Citations
Current status and perspective of CAR-T and CAR-NK cell therapy trials in Germany	168
CARAMBA: a first-in-human clinical trial with SLAMF7 CAR-T cells prepared by virus-free Sleeping Beauty gene transfer to treat multiple myeloma	91
Prime editing – an update on the field	89
Non-viral delivery of CRISPR–Cas9 complexes for targeted gene editing via a polymer delivery system	47
Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions	47
Correction of X-CGD patient HSPCs by targeted CYBB cDNA insertion using CRISPR/Cas9 with 53BP1 inhibition for enhanced homology-directed repair	45
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells	38
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry	37
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy	36
Cas9-directed immune tolerance in humans—a model to evaluate regulatory T cells in gene therapy?	33
Gene therapy for spinal muscular atrophy: the Qatari experience	33
Fabrication of anionic dextran-coated micelles for aptamer targeted delivery of camptothecin and survivin-shRNA to colon adenocarcinoma	32
Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice	32
Genome editing for Duchenne muscular dystrophy: a glimpse of the future?	29
Gene editing and RNAi approaches for COVID-19 diagnostics and therapeutics	28
Successes and challenges in clinical gene therapy	25
Rational engineering of a functional CpG-free ITR for AAV gene therapy	25
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1	25
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation	25
Curing SMA: Are we there yet?	25
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9	24
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1	24
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study	23
Mesenchymal stem cells overexpressing BMP-9 by CRISPR-Cas9 present high in vitro osteogenic potential and enhance in vivo bone formation	22
Antitumor effects of IL-12 and GM-CSF co-expressed in an engineered oncolytic HSV-1	21

Rescue of retinal ganglion cells in optic nerve injury using cell-selective AAV mediated delivery of SIRT1	21
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies	21
The estimated annual financial impact of gene therapy in the United States	20
Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters	20
Restoration of RPGR expression in vivo using CRISPR/Cas9 gene editing	20
Control of gene doping in human and horse sports	20
Advantages of robotic assistance over a manual approach in simulated subretinal injections and its relevance for gene therapy	18
XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma	18
Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates	18
Genetic insights, disease mechanisms, and biological therapeutics for Waardenburg syndrome	18
Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells	18
Retroviral gene therapy in Germany with a view on previous experience and future perspectives	17
Effective viral-mediated lung gene therapy: is airway surface preparation necessary?	17
Long-term safety and efficacy of intramyocardial adenovirus-mediated VEGF-DΔNΔC gene therapy eight-year follow-up of phase I KAT301 study	17
Chitosan-coated Zn-metal-organic framework nanocomposites for effective targeted delivery of LNA-antisense miR-224 to colon tumor: in vitro studies	17
Exosomal microRNA-133b-3p from bone marrow mesenchymal stem cells inhibits angiogenesis and oxidative stress via FBN1 repression in diabetic retinopathy	16
Novel tool to quantify with single-cell resolution the number of incoming AAV genomes co-expressed in the mouse nervous system	16
Implications of hematopoietic stem cells heterogeneity for gene therapies	14
Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor	14
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-21	14
Molecular targeting of vulnerable RNA sequences in SARS CoV-2: identifying clinical feasibility	14
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)	14
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice	13
Screening for gene doping transgenes in horses via the use of massively parallel sequencing	13
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene	13
The transformative potential of HSC gene therapy as a genetic medicine	13
Effects of oncolytic viruses and viral vectors on immunity in glioblastoma	13
From macrophage biology to macrophage-based cellular immunotherapies	13
Optimisation of a TALE nuclease targeting the HIV co-receptor CCR5 for clinical application	13
Ptbp1 knockdown failed to induce astrocytes to neurons in vivo	13
Splicing mutations in the CFTR gene as therapeutic targets	12
Dystrophin and mini-dystrophin quantification by mass spectrometry in skeletal muscle for gene therapy development in Duchenne muscular dystrophy	12
Exon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells	12
E3 ubiquitin ligase SMURF2 prevents colorectal cancer by reducing the stability of the YY1 protein and inhibiting the SENP1/c-myc axis	11
Large-scale purification of functional AAV particles packaging the full genome using short-term ultracentrifugation with a zonal rotor	11
Upper motor neurons are a target for gene therapy and UCHL1 is necessary and sufficient to improve cellular integrity of diseased upper motor neurons	11
Genome editing using CRISPR/Cas9 to treat hereditary hematological disorders	11
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	11
Single-cell mapping of focused ultrasound-transfected brain	10
AAV-mediated delivery of actoxumab and bezlotoxumab results in serum and mucosal antibody concentrations that provide protection from C. difficile toxin challenge	10
Gene delivery to the hypoglossal motor system: preclinical studies and translational potential	10
Double viral vector technology for selective manipulation of neural pathways with higher level of efficiency and safety	10
Evaluation of the dystrophin carboxy-terminal domain for micro-dystrophin gene therapy in cardiac and skeletal muscles in the DMDmdx rat model	10
Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells	10
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVA	10
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease	10
AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses	9
Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?	9
Restored microRNA-519a enhances the radiosensitivity of non-small cell lung cancer via suppressing EphA2	9
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy	9

Adjuvant properties of IFN-γ and GM-CSF in the scFv6.C4 DNA vaccine against CEA-expressing tumors	9
Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa	9
Insidious Insights: Implications of viral vector engineering for pathogen enhancement	9