Ontology highlight
ABSTRACT:
SUBMITTER: Sabatino DE
PROVIDER: S-EPMC3048175 | biostudies-literature | 2011 Mar
REPOSITORIES: biostudies-literature
Sabatino Denise E DE Lange Amy M AM Altynova Ekaterina S ES Sarkar Rita R Zhou Shangzhen S Merricks Elizabeth P EP Franck Helen G HG Nichols Timothy C TC Arruda Valder R VR Kazazian Haig H HH
Molecular therapy : the journal of the American Society of Gene Therapy 20101116 3
Developing adeno-associated viral (AAV)-mediated gene therapy for hemophilia A (HA) has been challenging due to the large size of the factor VIII (FVIII) complementary DNA and the concern for the development of inhibitory antibodies to FVIII in HA patients. Here, we perform a systematic study in HA dogs by delivering a canine FVIII (cFVIII) transgene either as a single chain or two chains in an AAV vector. An optimized cFVIII single chain delivered using AAV serotype 8 (AAV8) by peripheral vein ...[more]