Ontology highlight
ABSTRACT:
SUBMITTER: High KA
PROVIDER: S-EPMC4802375 | biostudies-literature | 2016 Apr
REPOSITORIES: biostudies-literature
Human molecular genetics 20151127 R1
Gene transfer studies for the treatment of hemophilia began more than two decades ago. A large body of pre-clinical work evaluated a variety of vectors and target tissues, but by the start of the new millennium it became evident that adeno-associated viral (AAV)-mediated gene transfer to the liver held great promise as a therapeutic tool. The transition to the clinical arena uncovered a number of unforeseen challenges, mainly in the form of a human-specific immune response against the vector tha ...[more]