Long-Term Sex-Biased Correction of Circulating Propionic Acidemia Disease Markers by Adeno-Associated Virus Vectors
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چکیده
منابع مشابه
Long QTc Syndrome and Propionic Acidemia.
A 9-year-old boy diagnosed as Propionic acidemia at 2 months of age, complained of palpitations and nearsyncope episodes related with exercise. His metabolic condition was well controlled on sodium benzoate, carnitine, protein-free diet and biotin treatment. A prolonged QTc interval was noted on ECG-Holter (Fig. 1). Echocardiography showed no anomalies. He fulfilled criteria for diagnosis of Lo...
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Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2015
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2014.126