Results 1 to 10 of about 1,183,829 (263)

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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Gene therapy [PDF]

Proceedings of the National Academy of Sciences, 1997
In recent years, there have been a number of technological breakthroughs that have allowed for clinical trials in gene therapy to be initiated. In combination with the genome initiative, the potential for new therapeutics is limitless. Although an enormous amount of information has been obtained in a relatively short period of time, gene therapy ...
M A, Kay, D, Liu, P M, Hoogerbrugge
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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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Gene Therapy

Hematology/Oncology Clinics of North America, 2017
Gene therapy refers to a rapidly growing field of medicine in which genes are introduced into the body to treat or prevent diseases. Although a variety of methods can be used to deliver the genetic materials into the target cells and tissues, modified viral vectors represent one of the more common delivery routes because of its transduction efficiency ...
Barb, Thorne, Ryan, Takeya, Francesca, Vitelli, Xin, Swanson   +3 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
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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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