abstract |
The present disclosure generally provides codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the present disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for administering a polynucleotide encoding a Factor VIII polypeptide, eg, a codon-altered polynucleotide, to a hemophilia A patient. |