Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK
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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: Leukemia
سال: 2010
ISSN: 0887-6924,1476-5551
DOI: 10.1038/leu.2010.140