Gene Therapy

Papers
(The TQCC of Gene Therapy is 8. 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 2021-05-01 to 2025-05-01.)
ArticleCitations
First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model102
Serotype-specific transduction of canine joint tissue explants and cultured monolayers by self-complementary adeno-associated viral vectors68
A shedding analysis after AAV8 CNS injection revealed fragmented viral DNA without evidence of functional AAV particles in mice54
Overexpressed ski efficiently promotes neurorestoration, increases neuronal regeneration, and reduces astrogliosis after traumatic brain injury50
AAV-vectored expression of monospecific or bispecific monoclonal antibodies protects mice from lethal Pseudomonas aeruginosa pneumonia39
Review of cardiac safety in onasemnogene abeparvovec gene replacement therapy: translation from preclinical to clinical findings39
Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease37
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system35
Clinical enrollment assay to detect preexisting neutralizing antibodies to AAV6 with demonstrated transgene expression in gene therapy trials33
Gene therapy for spinal muscular atrophy: the Qatari experience33
Rapid characterization of adeno-associated virus (AAV) gene therapy vectors by mass photometry31
Control of gene doping in human and horse sports30
Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene28
Correction: The disparate burden of infectious diseases28
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV226
Viral vector gene delivery of the novel chaperone protein SRCP1 to modify insoluble protein in in vitro and in vivo models of ALS25
Implications of maternal-fetal health on perinatal stem cell banking24
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa24
Correction: Site-specific modifications to AAV8 capsid yields enhanced brain transduction in the neonatal MPS IIIB mouse22
Unlocking patient access to gene therapy: five key practices22
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer22
Antibody-based protection against respiratory syncytial virus in mice and their offspring through vectored immunoprophylaxis22
Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression21
Analytical characterization of full, intermediate, and empty AAV capsids21
Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 119
Analysis and comparative evaluation of expedited programs for gene therapy products: insights from the United States, the European Union, Japan, and South Korea18
Injection pressure levels for creating blebs during subretinal gene therapy18
Correction: Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)18
Approval and therapeutic value of gene therapies in the US and Europe17
Optimization of adeno-associated viral (AAV) gene therapies vectors for balancing efficacy, longevity and safety for clinical application17
Epigenetic repression of gonadotropin gene expression via a GnRH-mediated DNA delivery system17
Prevalence of AAV2.5 neutralizing antibodies in synovial fluid and serum of patients with osteoarthritis16
Adeno-associated virus serotype 2 capsids with proteolytic cuts by trypsin remain intact and potent16
A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity15
CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing15
Retraction Note: Optimization of oncolytic effect of Newcastle disease virus Clone30 by selecting sensitive tumor host and constructing more oncolytic viruses15
Novel tool to quantify with single-cell resolution the number of incoming AAV genomes co-expressed in the mouse nervous system15
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia14
rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality14
A PCR-amplified transgene fragment flanked by a single copy of a truncated inverted terminal repeat for recombinant adeno-associated virus production prevents unnecessary plasmid DNA packaging14
An ectopic enhancer restores CFTR expression through de novo chromatin looping14
Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques13
Long-term effects of a fat-directed FGF21 gene therapy in aged female mice13
Correction: RapaCaspase-9-based suicide gene applied to the safety of IL-1RAP CAR-T cells13
Gene drives: an alternative approach to malaria control?12
Ghrelin mediated cardioprotection using in vitro models of oxidative stress12
Engineered compact pan-neuronal promoter from Alphaherpesvirus LAP2 enhances target gene expression in the mouse brain and reduces tropism in the liver12
Correction: Antitumoral gene-based strategy involving nitric oxide synthase type III overexpression in hepatocellular carcinoma11
Effects of intracardiac delivery of aldehyde dehydrogenase 2 gene in myocardial salvage11
Preclinical assessment of antigen-specific chimeric antigen receptor regulatory T cells for use in solid organ transplantation11
Schwann cell gene therapies in sight11
Correction: Preclinical safety and biodistribution of CRISPR targeting SIV in non-human primates11
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells10
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice10
Gene therapy corrects the neurological deficits of mice with sialidosis10
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses10
Rational engineering of a functional CpG-free ITR for AAV gene therapy10
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model10
Characterization of anti-AAV2 neutralizing antibody levels in sheep prior to and following intravitreal AAV2.7m8 injection9
AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis9
CCR1 enhances SUMOylation of DGCR8 by up-regulating ERK phosphorylation to promote spinal nerve ligation-induced neuropathic pain9
Onasemnogene abeparvovec gene replacement therapy for the treatment of spinal muscular atrophy: a real-world observational study9
Retraction Note: Long noncoding RNA RBMS3-AS3 acts as a microRNA-4534 sponge to inhibit the progression of prostate cancer by upregulating VASH19
Influence of donor age and comorbidities on transduced human adipose-derived stem cell in vitro osteogenic potential9
Exosomal PD-L1 confers chemoresistance and promotes tumorigenic properties in esophageal cancer cells via upregulating STAT3/miR-219
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications8
Towards access for all: 1st Working Group Report for the Global Gene Therapy Initiative (GGTI)8
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model8
Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy8
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE658
Assessment of AAV9 distribution and transduction in rats after administration through Intrastriatal, Intracisterna magna and Lumbar Intrathecal routes8
Prime editing: therapeutic advances and mechanistic insights8
Genes for bad backs8
0.035231828689575