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Gene Therapy: Possibilities and Promise [PDF]
Pediatric Research, 1993 I It is now nearly 50 years since DNA was discovered to be the repository of hereditary information and approximately 20 years I since the recombinant DNA era began. From the earliest times in this scientific saga. there have been hopes that it would be L possible to transfer therapeutically effective genes into human patients just as one can do with ...
Larry J. Shapiro
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The Impact of QSP Modeling on the Design and Optimization of Gene Therapy Approaches. [PDF]
CPT Pharmacometrics Syst PharmacolRayad N, Chowdhury EA, Meno-Tetang GML.
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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 +18 more
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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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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 +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 +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 +7 more
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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 +3 more
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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 +9 more
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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 +9 more
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