OOIR: Observatory of International Research

Papers

(The median citation count of Gene Therapy is 3. 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
Curing SMA: Are we there yet?	25
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
AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1	24
Enhanced CNS transduction from AAV.PHP.eB infusion into the cisterna magna of older adult rats compared to AAV9	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
Changing trends in the development of AAV-based gene therapies: a meta-analysis of past and present therapies	21

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
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
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
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
Exon skipping induced by CRISPR-directed gene editing regulates the response to chemotherapy in non-small cell lung carcinoma cells	12
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
Rs1h−/y exon 3-del rat model of X-linked retinoschisis with early onset and rapid phenotype is rescued by RS1 supplementation	11
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
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
Oncolytic Newcastle disease virus expressing the co-stimulator OX40L as immunopotentiator for colorectal cancer therapy	8
Adeno-associated virus mediated expression of monoclonal antibody MR191 protects mice against Marburg virus and provides long-term expression in sheep	8
Automated production of CCR5-negative CD4+-T cells in a GMP-compatible, clinical scale for treatment of HIV-positive patients	8
Equitable access to cell and gene therapies in South Africa: opportunities and hurdles	8
RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells	8
Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates	8
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury	8
Injection pressure levels for creating blebs during subretinal gene therapy	7
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain	7
Thrombopoietin-based CAR-T cells demonstrate in vitro and in vivo cytotoxicity to MPL positive acute myelogenous leukemia and hematopoietic stem cells	7
Survey of perspectives of people with inherited retinal diseases on ocular gene therapy in Australia	7
Cerebral organoids as an in vitro model to study autism spectrum disorders	7
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials	7
Approval and therapeutic value of gene therapies in the US and Europe	7
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model	7
Towards affordable CRISPR genomic therapies: a task force convened by the Innovative Genomics Institute	7
Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse	6
CRISPR/Cas9-based genome editing for the modification of multiple duplications that cause Duchenne muscular dystrophy	6
A new protocol for whole-brain biodistribution analysis of AAVs by tissue clearing, light-sheet microscopy and semi-automated spatial quantification	6
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS	6
Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models	6
Progress on siRNA-based gene therapy targeting secondary injury after intracerebral hemorrhage	6
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells	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
Adeno-associated virus serotype 9 antibody titers in patients with SMA pre-screened for treatment with onasemnogene abeparvovec –routine care evidence	6
Novel design of nucleic acid standards for hydrolysis probe-based PCR with melting analysis	5
Cost of gene therapy	5
Targeted shock-and-kill HIV-1 gene therapy approach combining CRISPR activation, suicide gene tBid and retargeted adenovirus delivery	5
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings	5
Access to affordable medicines: obligations of universities and academic medical centers	5
Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets	5
Characterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapy	5
Induced pluripotent stem cell-derived dendritic cell vaccine therapy genetically modified on the ubiquitin-proteasome system	5
Development of a cGMP-compliant process to manufacture donor-derived, CD45RA-depleted memory CD19-CAR T cells	5
Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy	5
Homology-directed repair of an MYBPC3 gene mutation in a rat model of hypertrophic cardiomyopathy	5
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential	5
Characterization of integration frequency and insertion sites of adenovirus DNA into mouse liver genomic DNA following intravenous injection	5
Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model	4
Acoustically targeted noninvasive gene therapy in large brain volumes	4
Distinct functions of CAR-T cells possessing a dectin-1 intracellular signaling domain	4
Combinatorial gene therapy for epilepsy: Gene sequence positioning and AAV serotype influence expression and inhibitory effect on seizures	4
Adeno-associated virus vectors and neurotoxicity—lessons from preclinical and human studies	4
Terminal hairpins improve protein expression in IRES-initiated mRNA in the absence of a cap and polyadenylated tail	4
Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?	4
Biodistribution and immunity of adenovirus 5/35 and modified vaccinia Ankara vector vaccines against human immunodeficiency virus 1 clade C	4
FEN1 inhibitor synergizes with low-dose camptothecin to induce increased cell killing via the mitochondria mediated apoptotic pathway	4
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing	4
Repeat or single-dose lentiviral vector administration to mouse lungs? It’s all about the timing	4
Targeting mAKAPβ expression as a therapeutic approach for ischemic cardiomyopathy	4
Insights and future directions of potential genetic therapy for Apert syndrome: A systematic review	4
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model	4
Outer retinal transduction by AAV2-7m8 following intravitreal injection in a sheep model of CNGA3 achromatopsia	4
Loss of RAGE prevents chronic intermittent hypoxia-induced nonalcoholic fatty liver disease via blockade of NF-кB pathway	3
Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases	3
Identification of RPGR ORF15 mutation for X-linked retinitis pigmentosa in a large Chinese family and in vitro correction with prime editor	3
Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active	3
Proteosomal degradation impairs transcytosis of AAV vectors from suprachoroidal space to retina	3
Glyco-binding domain chimeric antigen receptors as a new option for cancer immunotherapy	3
Correction of a knock-in mouse model of acrodysostosis with gene therapy using a rAAV9-CAG-human PRKAR1A vector	3
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes	3
Correction: Intracellular trafficking of adeno-associated virus (AAV) vectors: challenges and future directions	3
Toward gene therapy of Laron syndrome	3
Taylor Dispersion Analysis to support lipid-nanoparticle formulations for mRNA vaccines	3
HIV- 1 lentivirus tethering to the genome is associated with transcription factor binding sites found in genes that favour virus survival	3
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer	3
Removal of the GAA repeat in the heart of a Friedreich’s ataxia mouse model using CjCas9	3
Artificial microRNA suppresses C9ORF72 variants and decreases toxic dipeptide repeat proteins in vivo	3
CRISPR/sgRNA-directed synergistic activation mediator (SAM) as a therapeutic tool for Parkinson´s disease	3