Ontology highlight
ABSTRACT:
SUBMITTER: Hausl MA
PROVIDER: S-EPMC2990515 | biostudies-literature | 2010 Nov
REPOSITORIES: biostudies-literature
Hausl Martin A MA Zhang Wenli W Müther Nadine N Rauschhuber Christina C Franck Helen G HG Merricks Elizabeth P EP Nichols Timothy C TC Kay Mark A MA Ehrhardt Anja A
Molecular therapy : the journal of the American Society of Gene Therapy 20100817 11
Sleeping Beauty (SB) transposase enables somatic integration of exogenous DNA in mammalian cells, but potency as a gene transfer vector especially in large mammals has been lacking. Herein, we show that hyperactive transposase system delivered by high-capacity adenoviral vectors (HC-AdVs) can result in somatic integration of a canine factor IX (cFIX) expression-cassette in canine liver, facilitating stabilized transgene expression and persistent haemostatic correction of canine hemophilia B with ...[more]