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Modification of a Constitutive to Glucose-Responsive Liver-Specific Promoter Resulted in Increased Efficacy of Adeno-Associated Virus Serotype 8-Insulin Gene Therapy of Diabetic Mice.
We have previously used a hepatotropic adeno-associated viral (AAV) vector with a modified human insulin gene to treat diabetic mice. The HLP (hybrid liver-specific promoter) used was constitutively active and non-responsive to...
adeno-associated virus serotype 8 (AAV8) albumin enhancer (3′iALB) diabetes gene therapy glucose responsive element (GlRE) liver-specific glucose-responsive promoter long-term basal insulin expression Animals Dependovirus Diabetes mellitus Experimental Disease Models Animal Gene Expression Genetic Therapy Genetic Vectors Glucose Hepatocytes Humans Insulin Mice Promoter Regions Genetic Transgenes
Exploiting Single-Cell Tools in Gene and Cell Therapy.
Single-cell molecular tools have been developed at an incredible pace over the last five years as sequencing costs continue to drop and numerous molecular assays have been coupled to sequencing readouts. This rapid period of...
Retinal regeneration mechanisms linked to multiple cancer molecules
Over the last decade, a large number of research articles have been published demonstrating regeneration and/or neuroprotection of retinal ganglion cells following manipulation of specific genetic and molecular targets....
Linear mitochondrial DNA is rapidly degraded by components of the replication machinery.
Viktoriya Peeva, Daniel Blei, Genevieve Trombly, Sarah Corsi, Maciej J Szukszto, Pedro Rebelo-Guiomar, Payam A Gammage, Alexei P Kudin, Christian Becker, Janine Altmüller, Michal Minczuk, Gábor Zsurka, Wolfram S Kunz
Jul 17, 2018
Emerging gene therapy approaches that aim to eliminate pathogenic mutations of mitochondrial DNA (mtDNA) rely on efficient degradation of linearized mtDNA, but the enzymatic machinery performing this task is presently unknown....
Base Sequence CRISPR-Cas Systems DNA Breaks Double-Stranded DNA Cleavage DNA Helicases DNA Polymerase gamma DNA Replication DNA Mitochondrial Deoxyribonucleases Type II Site-Specific Electron Transport Complex IV Exodeoxyribonucleases Gene Editing Genetic Therapy HEK293 Cells Humans Mitochondria Recombinant Fusion Proteins
A track of the clones
Anne-Marie Lyne, David G Kent, Elisa Laurenti, Kerstin Cornils, Ingmar Glauche, Leïla Perié
Dec 17, 2018
Cell Lineage Cell Separation Cell Tracking Clinical Trials as Topic Clone Cells DNA barcoding Taxonomic DNA Transposable Elements Data Display Forecasting Genetic Markers Genetic Therapy Guidelines as Topic Hematopoiesis Models Biological Mosaicism Reproducibility of Results Research Design Stem Cells Virus Integration
Lack of Neuronal IFN-β-IFNAR Causes Lewy Body- and Parkinson's Disease-like Dementia.
Patrick Ejlerskov, Jeanette Göransdotter Hultberg, JunYang Wang, Robert Carlsson, Malene Ambjørn, Martin Kuss, Yawei Liu, Giovanna Porcu, Kateryna Kolkova, Carsten Friis Rundsten, Karsten Ruscher, Bente Pakkenberg, Tobias Goldmann, Desiree Loreth, Marco Prinz, David C Rubinsztein, Shohreh Issazadeh-Navikas
Aug 19, 2015
GDNF and Parkinson's Disease
Roger A Barker, Anders Björklund, Don M Gash, Alan Whone, Amber Van Laar, Jeffrey H Kordower, Krystof Bankiewicz, Karl Kieburtz, Mart Saarma, Sigrid Booms, Henri J Huttunen, Adrian P Kells, Massimo S Fiandaca, A Jon Stoessl, David Eidelberg, Howard Federoff, Merja H Voutilainen, David T Dexter, Jamie Eberling, Patrik Brundin, Lyndsey Isaacs, Leah Mursaleen, Eros Bresolin, Camille Carroll, Alasdair Coles, Brian Fiske, Helen Matthews, Codrin Lungu, Richard K Wyse, Simon Stott, Anthony E Lang
Jul 17, 2020
The concept of repairing the brain with growth factors has been pursued for many years in a variety of neurodegenerative diseases including primarily Parkinson's disease (PD) using glial cell line-derived neurotrophic factor...
Secretion of a mammalian chondroitinase ABC aids glial integration at PNS/CNS boundaries.
Philippa M Warren, Melissa R Andrews, Marc Smith, Katalin Bartus, Elizabeth J Bradbury, Joost Verhaagen, James W Fawcett, Jessica CF Kwok
Aug 19, 2020
Schwann cell grafts support axonal growth following spinal cord injury, but a boundary forms between the implanted cells and host astrocytes. Axons are reluctant to exit the graft tissue in large part due to the surrounding...
Animals Astrocytes Axons Cell Adhesion Cell Movement Cells Cultured Central Nervous System Chondroitin ABC Lyase Chondroitin Sulfate Proteoglycans Female Genetic Therapy Integrins Lentivirus Nerve Regeneration Neurites Neuroglia Neurons Peripheral Nervous System Rats Rats Sprague-Dawley Schwann Cells Spinal Cord Injuries
Human artificial chromosome with a conditional centromere for gene delivery and gene expression.
Yuichi Iida, Jung-Hyun Kim, Yasuhiro Kazuki, Hidetoshi Hoshiya, Masato Takiguchi, Masahiro Hayashi, Indri Erliandri, Hee-Sheung Lee, Alex Samoshkin, Hiroshi Masumoto, William C Earnshaw, Natalay Kouprina, Vladimir Larionov, Mitsuo Oshimura
Dec 18, 2020
Human artificial chromosomes (HACs), which carry a fully functional centromere and are maintained as a single-copy episome, are not associated with random mutagenesis and offer greater control over expression of ectopic genes on...
Animals CHO Cells Cell Line Centromere Chickens Chromosomes Artificial Human Cricetinae Cricetulus Gene Expression Gene Targeting Gene Transfer Techniques Genetic Therapy Genetic Vectors Green Fluorescent Proteins Humans In Situ Hybridization Fluorescence Operator Regions Genetic Plasmids Polymerase Chain Reaction Recombination Genetic Tetracyclines Transgenes
Efficacy and Safety of Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy Treated within 6 Months of Disease Onset.
Nancy J Newman, Patrick Yu-Wai-Man, Valerio Carelli, Mark L Moster, Valerie Biousse, Catherine Vignal-Clermont, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Piero Barboni, Adam A DeBusk, Jean François Girmens, Günther Rudolph, Rustum Karanjia, Magali Taiel, Laure Blouin, Gerard Smits, Barrett Katz, José-Alain Sahel, LHON Study Group
Apr 15, 2021
PURPOSE: To evaluate the efficacy of a single intravitreal injection of rAAV2/2-ND4 in subjects with visual loss from Leber hereditary optic neuropathy (LHON). DESIGN: RESCUE is a multicenter, randomized, double-masked...
Humphrey visual field perimetry Leber hereditary optic neuropathy Phase 3 randomized double-masked clinical trial Quality of life best-corrected visual acuity bilateral visual improvement Contrast Sensitivity EFFICACY intravitreal gene therapy retinal anatomic measures Safety Adolescent Adult Aged DNA Mitochondrial Dependovirus Double-Blind Method Electroretinography Female Follow-Up Studies Genetic Therapy Genetic Vectors Humans Intravitreal Injections Male Middle Aged Mutation Optic Atrophy Hereditary Leber Quality of life Time Factors Treatment Outcome Visual Acuity Visual Field Tests Visual Fields Young Adult
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Panicos Shangaris, Stavros P Loukogeorgakis, Sindhu Subramaniam, Christina Flouri, Laurence H Jackson, Wei Wang, Michael P Blundell, Shanrun Liu, Simon Eaton, Nahla Bakhamis, Durrgah Latchumi Ramachandra, Panayiotis Maghsoudlou, Luca Urbani, Simon N Waddington, Ayad Eddaoudi, Joy Archer, Michael N Antoniou, Daniel J Stuckey, Manfred Schmidt, Adrian J Thrasher, Thomas M Ryan, Paolo De Coppi, Anna L David
Oct 22, 2019
In utero gene therapy (IUGT) to the fetal hematopoietic compartment could be used to treat congenital blood disorders such as β-thalassemia. A humanised mouse model of β-thalassemia was used, in which heterozygous animals are...
Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease.
Stéphane Palfi, Jean Marc Gurruchaga, Hélène Lepetit, Katy Howard, G Scott Ralph, Sarah Mason, Gaëtane Gouello, Philippe Domenech, Philip C Buttery, Philippe Hantraye, Nicola J Tuckwell, Roger A Barker, Kyriacos A Mitrophanous
Nov 16, 2018
Parkinson's disease is typically treated with oral dopamine replacement therapies. However, long-term use is complicated by motor fluctuations from intermittent stimulation of dopamine receptors and off-target effects. ProSavin...
Design of a Novel Gene Therapy Construct to Achieve Sustained Brain-Derived Neurotrophic Factor Signaling in Neurons.
Brain-derived neurotrophic factor (BDNF) acting through the tropomyosin-related receptor-B (TrkB) is an important signaling system for the maintenance and survival of neurons. Gene therapy using either recombinant...
adeno-associated viral vector Brain-Derived Neurotrophic Factor gene therapy tropomyosin-related receptor kinase-B Animals Brain-Derived Neurotrophic Factor Dependovirus Genetic Therapy HEK293 Cells Humans Hydrogen Peroxide Ligands Membrane Glycoproteins Mice Neurons Oxidative Stress Phosphorylation Protein Sorting Signals Receptor trkB Retinal Ganglion Cells
Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells.
Anna K Dreismann, Michelle E McClements, Alun R Barnard, Elise Orhan, Jane P Hughes, Peter J Lachmann, Robert E MacLaren
May 26, 2021
AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy.
Alexia Kagiava, Christos Karaiskos, Jan Richter, Christina Tryfonos, Matthew J Jennings, Amanda J Heslegrave, Irene Sargiannidou, Marina Stavrou, Henrik Zetterberg, Mary M Reilly, Christina Christodoulou, Rita Horvath, Kleopas A Kleopa
Mar 15, 2021
Mutations in the GJB1 gene, encoding the gap junction (GJ) protein connexin32 (Cx32), cause X-linked Charcot-Marie-Tooth disease (CMT1X), an inherited demyelinating neuropathy. We developed a gene therapy approach for CMT1X...
Persistence of haemostatic response following gene therapy with valoctocogene roxaparvovec in severe haemophilia A.
K John Pasi, Michael Laffan, Savita Rangarajan, Tara M Robinson, Nina Mitchell, Will Lester, Emily Symington, Bella Madan, Xinqun Yang, Benjamin Kim, Glenn F Pierce, Wing Yen Wong
Nov 08, 2021
INTRODUCTION: Valoctocogene roxaparvovec is an investigational AAV5-based factor VIII (FVIII) gene therapy that has demonstrated sustained clinical benefit in people with severe haemophilia A. AIM: To report safety...
From Transcriptomics to Treatment in Inherited Optic Neuropathies.
Inherited optic neuropathies, including Leber Hereditary Optic Neuropathy (LHON) and Dominant Optic Atrophy (DOA), are monogenetic diseases with a final common pathway of mitochondrial dysfunction leading to retinal ganglion...
DOA LHON OPA1 RNA-seq gene-therapy Mitochondrial Neuroprotection optic neuropathies transcriptomics Alleles Animals Disease Management Gene Expression Profiling Genetic Association Studies Genetic Predisposition to Disease Genetic Therapy Genotype Humans Mutation Optic Atrophies Hereditary Phenotype Transcriptome
Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy.
Javier Torres-Torronteras, Raquel Cabrera-Pérez, Ferran Vila-Julià, Carlo Viscomi, Yolanda Cámara, Michio Hirano, Massimo Zeviani, Ramon Martí
Jul 04, 2018
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is caused by mutations in TYMP, the gene encoding the enzyme thymidine phosphorylase (TP). TP dysfunction results in systemic accumulation of the noxious TP...
AAV MNGIE liver/metabolic Mitochondria thymidine phosphorylase Animals Carcinogenesis Deoxyuridine Dependovirus Female Gene Dosage Genetic Therapy Genetic Vectors Intestinal Pseudo-Obstruction Kaplan-Meier Estimate Liver Male Mice Mitochondria Liver Muscular Dystrophy Oculopharyngeal Ophthalmoplegia Thymidine Thymidine Phosphorylase Time Factors Transgenes
In vivo mitochondrial base editing via adeno-associated viral delivery to mouse post-mitotic tissue.
Pedro Silva-Pinheiro, Pavel A Nash, Lindsey Van Haute, Christian D Mutti, Keira Turner, Michal Minczuk
Mar 13, 2022
Mitochondria host key metabolic processes vital for cellular energy provision and are central to cell fate decisions. They are subjected to unique genetic control by both nuclear DNA and their own multi-copy genome -...
Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye.
Gerard A Rodrigues, Evgenyi Shalaev, Thomas K Karami, James Cunningham, Nigel Kh Slater, Hongwen M Rivers
Jan 08, 2019
A resurgence of interest and investment in the field of gene therapy, driven in large part by advances in viral vector technology, has recently culminated in United States Food and Drug Administration approval of the first gene...
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