With the human genome product and continuing advances in molecular biology many therapeutic genes have been discovered. In the cardiovascular system, gene therapy has the potential to improve myocardial vascularization and ameliorate congestive heart failure. For successful development of clinical gene therapy, however, effective gene delivery vectors are needed. Ultrasound contrast agents can be used to develop new, more effective vectors for gene delivery. Ultrasound contrast agents lower the threshold for cavitation by ultrasound energy. Using physical properties of microbubbles and coating materials, genetic drugs have been incorporated into ultrasound contrast agents. Gene-bearing microbubbles can be injected IV and ultrasound energy applied to the target region. As the microbubbles enter the region of insonation, the microbubbles cavitate, locally releasing DNA. Cavitation also likely causes a local shockwave that improves cellular uptake of DNA. With transthoracic ultrasound, using commercially available diagnostic ultrasound system and an IV injection of gene-bearing microbubbles, high levels of transgene expression are observed in the insonated region of the myocardium. This new technology using microbubbles and ultrasound for gene delivery merits further study and development.
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http://dx.doi.org/10.1046/j.1540-8175.2001.00355.x | DOI Listing |
BMC Pregnancy Childbirth
January 2025
Department of Obstetrics and Gynecology, National Clinical Research Center for Obstetrics and Gynecology, Peking University Third Hospital, Peking University Third Hospital), National Center for Healthcare Quality Management in Obstetrics, Beijing, 100191, China.
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Olivia Newton-John Cancer Research Institute, Heidelberg, Melbourne, Australia.
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Chongqing Key Laboratory of High Active Traditional Chinese Drug Delivery System, Chongqing Engineering Research Center of Pharmaceutical Sciences, Chongqing Medical and Pharmaceutical College, Chongqing 401331, PR China. Electronic address:
Small-molecule pectin (SMP) extracted from the leaves of Premna ligustroides Hemsl, with a molecular weight range of 5000-35,000 Da, has demonstrated anti-inflammatory and lipid-lowering properties in vitro. This study explored the effects of SMP on hypercholesterolemia in mice, with a focus on inflammation, lipid profiles, and cholesterol metabolism. Mice received SMP at doses of 607, 303, and 152 mg/kg body weight.
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Formerly with Spark Therapeutics, Inc., 3025 Market Street, Philadelphia, PA, 19104.
Transmission electron microscopy has become a standard characterization tool for adeno-associated virus-based gene therapy products. However, cost and expertise requirements place in-house traditional transmission electron microscope systems out of reach for many companies in the field. Recently developed low voltage electron microscopes can fulfill many of the needs for adeno-associated virus characterization at a fraction of the cost.
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January 2025
Cancer and Stem Cell Biology Laboratory, Department of Genetic Engineering, SRM Institute of Science and Technology, Kattankulathur, Chengalpattu District, TamilNadu 603203, India. Electronic address:
Cancer is considered the second most common disease globally. In the past few decades, many approaches have been proposed for cancer treatment. One among those is targeted therapy using CRISPR-Cas system which plays an irreplaceable role in translational research through gene editing.
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