Results 1 to 10 of about 6,300,245 (266)

Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy

Nature Communications, 2021
Mucopolysaccharidosis type IVA (MPSIVA) is a lysosomal storage disorder causing severe skeletal and non-skeletal alterations in patients. Here, the authors generate a MPSIVA rat model that mimics the disabling human pathology and develop an AAV9-Galns ...
Joan Bertolin, Víctor Sánchez, Albert Ribera, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Xavier Sánchez, Sergio Muñoz, Sara Marcó, Jennifer Pérez, Gemma Elias, Xavier León, Carles Roca, Veronica Jimenez, Pedro Otaegui, Francisca Mulero, Marc Navarro, Jesús Ruberte, Fatima Bosch   +18 more
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A minimal bile salt excretory pump promoter allows bile acid-driven physiological regulation of transgene expression from a gene therapy vector

Cell & Bioscience, 2022
Background Bile acid (BA) homeostasis is mainly regulated by bile salt excretory pump (BSEP), a hepatocyte transporter that transfers BAs to the bile.
Javier Martínez-García, Manuela Molina, Leticia Odriozola, Angie Molina, Gloria González-Aseguinolaza, Nicholas D. Weber, Cristian Smerdou   +6 more
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Optimized riboswitch-regulated AAV vector for VEGF-B gene therapy

Frontiers in Medicine, 2022
Gene therapy would greatly benefit from a method to regulate therapeutic gene expression temporally. Riboswitches are small RNA elements that have been studied for their potential use in turning transgene expression on or off by ligand binding.
Reetta A. E. Eriksson, Reetta A. E. Eriksson, Tiina Nieminen, Lionel Galibert, Sanna K. Peltola, Petra Tikkanen, Piia Käyhty, Piia Käyhty, Hanna M. Leinonen, Igor Oruetxebarria, Saana Lepola, Anniina J. Valkama, Eevi M. Lipponen, Hanna P. Lesch, Seppo Ylä-Herttuala, Seppo Ylä-Herttuala, Kari J. Airenne   +16 more
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Exosome-mediated stable epigenetic repression of HIV-1

Nature Communications, 2021
A strategy to control HIV-1 infection is to stably repress HIV-1 and induce “deep latency”. Here the authors show that a recombinant anti-HIV-1-1 protein can be packaged as mRNA into exosomes and delivered systemically to repress HIV-1-1 within the ...
Surya Shrivastava, Roslyn M. Ray, Leo Holguin, Lilliana Echavarria, Nicole Grepo, Tristan A. Scott, John Burnett, Kevin V. Morris   +7 more
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The impact of minimally oversized adeno-associated viral vectors encoding human factor VIII on vector potency in vivo

Molecular Therapy: Methods & Clinical Development, 2016
Recombinant adeno-associated viral (rAAV) vectors containing oversized genomes provide transgene expression despite low efficiency packaging of complete genomes.
Sirkka Kyostio-Moore, Patricia Berthelette, Susan Piraino, Cathleen Sookdeo, Bindu Nambiar, Robert Jackson, Brenda Burnham, Catherine R O'Riordan, Seng H Cheng, Donna Armentano   +9 more
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Integrated gene expression profiles reveal a transcriptomic network underlying the thermogenic response in adipose tissue

Scientific Reports, 2023
Obesity and type 2 diabetes are two closely related diseases representing a serious threat worldwide. An increase in metabolic rate through enhancement of non-shivering thermogenesis in adipose tissue may represent a potential therapeutic strategy ...
Jordi Rodó, Miquel Garcia, Estefania Casana, Sergio Muñoz, Claudia Jambrina, Victor Sacristan, Sylvie Franckhauser, Ignasi Grass, Veronica Jimenez, Fatima Bosch   +9 more
doaj   +1 more source

AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice

Molecular Metabolism, 2017
Objective: Type 1 diabetes is characterized by autoimmune destruction of β-cells leading to severe insulin deficiency. Although many improvements have been made in recent years, exogenous insulin therapy is still imperfect; new therapeutic approaches ...
Cristina Mallol, Estefania Casana, Veronica Jimenez, Alba Casellas, Virginia Haurigot, Claudia Jambrina, Victor Sacristan, Meritxell Morró, Judith Agudo, Laia Vilà, Fatima Bosch   +10 more
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A comprehensive atlas of Aggrecan, Versican, Neurocan and Phosphacan expression across time in wildtype retina and in retinal degeneration

Scientific Reports, 2022
As photoreceptor cells die during retinal degeneration, the surrounding microenvironment undergoes significant changes that are increasingly recognized to play a prominent role in determining the efficacy of therapeutic interventions.
A. Matsuyama, A. A. Kalargyrou, A. J. Smith, R. R. Ali, R. A. Pearson   +4 more
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Antibody-based CCR5 blockade protects Macaques from mucosal SHIV transmission

Nature Communications, 2021
CCR5 is a co-receptor for many transmitted HIV strains. Here, the authors show that biweekly injection of the CCR5-specific antibody Leronlimab protects rhesus macaques against infection following repeated intrarectal challenges of a CCR5-tropic SHIV.
Xiao L. Chang, Gabriela M. Webb, Helen L. Wu, Justin M. Greene, Shaheed Abdulhaqq, Katherine B. Bateman, Jason S. Reed, Cleiton Pessoa, Whitney C. Weber, Nicholas Maier, Glen M. Chew, Roxanne M. Gilbride, Lina Gao, Rebecca Agnor, Travis Giobbi, Jeffrey Torgerson, Don Siess, Nicole Burnett, Miranda Fischer, Oriene Shiel, Cassandra Moats, Bruce Patterson, Kush Dhody, Scott Kelly, Nader Pourhassan, Diogo M. Magnani, Jeremy Smedley, Benjamin N. Bimber, Nancy L. Haigwood, Scott G. Hansen, Timothy R. Brown, Lishomwa C. Ndhlovu, Jonah B. Sacha   +32 more
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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Nature Communications, 2020
Adeno-associated viruses (AAVs) are vehicles for gene therapy in humans, but currently only a limited amount of AAV serotypes is available. Here, the authors identify a novel AAV, AAVv66, and demonstrate enhanced production yields, virion stability, and ...
Hung-Lun Hsu, Alexander Brown, Anna B. Loveland, Anoushka Lotun, Meiyu Xu, Li Luo, Guangchao Xu, Jia Li, Lingzhi Ren, Qin Su, Dominic J. Gessler, Yuquan Wei, Phillip W. L. Tai, Andrei A. Korostelev, Guangping Gao   +14 more
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