Correction of Methylmalonic AciduriaIn VivoUsing a Codon-Optimized Lentiviral Vector
نویسندگان
چکیده
منابع مشابه
Correction of methylmalonic aciduria in vivo using a codon-optimized lentiviral vector.
Methylmalonic aciduria is a rare disorder of organic acid metabolism with limited therapeutic options, resulting in high morbidity and mortality. Positive results from combined liver/kidney transplantation suggest, however, that metabolic sink therapy may be efficacious. Gene therapy offers a more accessible approach for the treatment of methylmalonic aciduria than organ transplantation. Accord...
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ژورنال
عنوان ژورنال: Human Gene Therapy
سال: 2014
ISSN: 1043-0342,1557-7422
DOI: 10.1089/hum.2013.111