Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice
نویسندگان
چکیده
منابع مشابه
Very-long-chain acyl-coenzyme a dehydrogenase deficiency in mice.
Fatty acid oxidation (FAO) defects are inborn errors of metabolism clinically associated with cardiomyopathy and sudden infant death syndrome (SIDS). FAO disorders often present in infancy with myocardial dysfunction and arrhythmias after exposure to stresses such as fasting, exercise, or intercurrent viral illness. It is uncertain whether the heart, in the absence of stress, is normal. We gene...
متن کاملGene Therapy for Very Long Chain Acyl-coA Dehydrogenase Deficiency Using Adeno-Associated Virus Vectors: A Dissertation
Very long chain acyl-coA dehydrogenase (VLCAD) is the rate-limiting step in mitochondrial fatty acid oxidation. VLCAD deficient mice and patients’ clinical symptoms stem from not only an energy deficiency but also long-chain metabolite accumulations. VLCAD deficient mice were treated systemically with 1x10 vector genomes of rAAV9-VLCAD. Expression was detected in the liver, heart and muscle. Al...
متن کاملKinetics of recombinant adeno-associated virus-mediated gene transfer.
Recombinant adeno-associated virus (rAAV) vectors have been shown to be useful for efficient gene delivery to a variety of dividing and nondividing cells. Mechanisms responsible for the long-term, persistent expression of the rAAV transgene are not well understood. In this study we investigated the kinetics of rAAV-mediated human factor IX (hFIX) gene transfer into human primary myoblasts and m...
متن کاملDepartment of Cardiac Function in LCAD Deficient Mice After a Single Bout of Endurance Exercise
Fa tty acids are the primary fuel source for cardiac tissue in both humans and animals. These become especially important during times of starvation and long bouts of exercise. Fatty acids are broken down into smaller, useable acyl-CoA subunits through a process called beta-oxidation. The first step in this process must be catalyzed by one of four acyl-CoA dehydrogenase enzymes depending on the...
متن کاملMutations of ACADS gene associated with short-chain acyl-coenzyme A dehydrogenase deficiency.
Short-chain acyl-coenzyme A dehydrogenase deficiency (SCADD) is an autosomal recessive disorder of mitochondrial fatty acid oxidation associated with mutations in the ACADS gene (Acyl-CoA Dehydrogenase, Short-chain, OMIM #606885). SCADD is a heterogeneous condition that has been associated with various clinical phenotypes ranging from fetal metabolic decompensation in infancy to asymptomatic in...
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ژورنال
عنوان ژورنال: The Journal of Gene Medicine
سال: 2008
ISSN: 1099-498X,1521-2254
DOI: 10.1002/jgm.1242