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Targeting serum response factor (SRF) deactivates ΔFosB and mitigates Levodopa-induced dyskinesia in a mouse model of Parkinson’s disease
Targeting serum response factor (SRF) deactivates ΔFosB and mitigates Levodopa-induced dyskinesia in a mouse model of Parkinson’s disease
L-3,4-dihydroxyphenylalanine (L-DOPA) is currently the preferred treatment for Parkinson’s Disease (PD) and is consi...
Suppression of matrigel-induced choroidal neovascularization by AAV delivery of a novel anti-Scg3 antibody
Suppression of matrigel-induced choroidal neovascularization by AAV delivery of a novel anti-Scg3 antibody
Efforts to develop gene therapy for long-term treatment of neovascular disease are hampered by ongoing concerns that biolo...
Precision medicine: toward restoring fat with gene therapy in inherited lipodystrophy
Mann JP, Savage DB. What lipodystrophies teach us about the metabolic syndrome. J Clin Invest. 2019;129:4009–21. https://d...
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects
Gene therapy of Dent disease type 1 in newborn ClC-5 null mice for sustained transgene expression and gene therapy effects
Dent disease type 1 is caused by changes in the chloride voltage-gated channel 5 (CLCN5) gene on chromosome X, resulting i...
BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target
BRD9 promotes the progression of gallbladder cancer via CST1 upregulation and interaction with FOXP1 through the PI3K/AKT pathway and represents a therapeutic target
Gallbladder cancer (GBC) is highly aggressive and has poor prognosis, with most patients only diagnosed at an advanced sta...
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Correction: Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Open Access This article is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 ...
Retraction Note: Inhibition of microRNA-495 suppresses chondrocyte apoptosis through activation of the NF-κB signaling pathway by regulating CCL4 in osteoarthritis
Authors and AffiliationsDepartment of Orthopaedics, The Fourth Affiliated Hospital of Harbin Medical University, 150001, H...
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice
Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice
In hemophilia, deficiency of factor VIII or IX prevents the activation of the common coagulation pathway, and inhibits the...
Retraction Note: miR-503-5p inhibits colon cancer tumorigenesis, angiogenesis, and lymphangiogenesis by directly downregulating VEGF-A
Authors and AffiliationsDepartment of Radiotherapy, Cancer Hospital of China Medical University, Liaoning Cancer Hospital ...
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model
CRISPR/Cas9-mediated exon skipping to restore premature translation termination in a DFNB4 mouse model
SLC26A4 encodes pendrin, a crucial anion exchanger essential for maintaining hearing function. Mutations in SLC26A4, inclu...
AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration
AAV dose-dependent transduction efficiency in retinal ganglion cells and functional efficacy of optogenetic vision restoration
Optogenetics is a promising approach for restoring vision to the blind after photoreceptor degeneration. The ability to re...
Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress
Intravesical nerve growth factor antisense therapy for bladder hypersensitivity induced by psychological stress
This study assessed the relationship between NGF expression in the bladder and bladder hypersensitivity caused by psycholo...
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Mutation-agnostic treatments such as airway gene therapy have the potential to treat any individual with cystic fibrosis (...
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses
Correction: AAV2-VEGF-B gene therapy failed to induce angiogenesis in ischemic porcine myocardium due to inflammatory responses
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model
Almost all attempts to date at gene therapy approaches for monogenetic disease have used the amino acid sequences of the n...
Retraction Note: Oncolysis of pancreatic tumour cells by a γ34.5-deleted HSV-1 does not rely upon Ras-activation, but on the PI 3-kinase pathway
Authors and AffiliationsDepartment of Histology, Microbiology and Medical Biotechnologies, Section of Microbiology and Vir...
Correction: Alternative oxidase encoded by sequence-optimized and chemically-modified RNA transfected into mammalian cells is catalytically active
Open Access This article is licensed under a Creative Commons Attribution 4.0 International License, which...
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2
The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2
Recombinant adeno-associated virus (rAAV) vectors are currently the only proven vehicles for treating ophthalmological dis...
Correction: Andrew C. G. Porter (1955–2023)
Authors and AffiliationsAGCTlab.org, Centre of Gene and Cell Therapy, Department of Biological Sciences, School of Life Sc...
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
Correction: Limited potential of AAV-mediated gene therapy in transducing human mesenchymal stem cells for bone repair applications
Correction to: Gene Therapy https://doi.org/10.1038/s41434-020-0182-4, published online 17 August 202...
Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles
Characterization of brain transduction capability of a BBB-penetrant AAV vector in mice, rats and macaques reveals differences in expression profiles
Different screening methods are being developed to generate adeno-associated viral vectors (AAV) with the ability to bypas...
Gene drives: an alternative approach to malaria control?
Gene drives: an alternative approach to malaria control?
Genetic modification for the control of mosquitoes is frequently touted as a solution for a variety of vector-borne diseas...
Amelioration of airway and GI disease in G551D-CF ferrets by AAV1 and AAV6
Amelioration of airway and GI disease in G551D-CF ferrets by AAV1 and AAV6
Gene therapy for CF has concentrated on targeting the lung. Here we took a different approach by injecting into the cephal...
Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy
Preclinical evaluation of tissue-selective gene therapies for congenital generalised lipodystrophy
Lipodystrophy is a rare disorder which can be life-threatening. Here individuals fail to develop or maintain appropriate a...
Andrew C. G. Porter (1955–2023)
Andrew C. G. Porter (1955–2023)
Andrew Christopher George Porter, known to everyone as Andy, was a dedicated, gifted, and generous sc...
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system
Highly efficient and specific regulation of gene expression using enhanced CRISPR-Cas12f system
The recently developed CRISPR activator (CRISPRa) system uses a CRISPR-Cas effector-based transcriptional activator to eff...
Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules
Stable inhibition of choroidal neovascularization by adeno-associated virus 2/8-vectored bispecific molecules
Neovascular age-related macular degeneration (nAMD) causes severe visual impairment. Pigment epithelium-derived factor (PE...
Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques
Administration and detection of a multi-target rAAV gene doping vector in horses using multiple matrices and molecular techniques
Gene doping, which includes the non-therapeutic use of genes or genetic elements that have the capacity to enhance athleti...
Correction: Dual-targeted NIS polyplexes—a theranostic strategy toward tumors with heterogeneous receptor expression
Correction: Dual-targeted NIS polyplexes—a theranostic strategy toward tumors with heterogeneous receptor expression
Correction to: Gene Therapy https://doi.org/10.1038/s41434-019-0059-6, published online 25 January 20...
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65
Correction: Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65
Authors and AffiliationsDepartment of Genetics, UCL Institute of Ophthalmology, London, EC1V 9EL, UKA. Georgiadis, Y. Dura...
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