Ontology highlight
ABSTRACT:
SUBMITTER: Xie J
PROVIDER: S-EPMC3048189 | biostudies-literature | 2011 Mar
REPOSITORIES: biostudies-literature
Molecular therapy : the journal of the American Society of Gene Therapy 20101221 3
Recombinant adeno-associated viruses (rAAVs) that can cross the blood-brain-barrier and achieve efficient and stable transvascular gene transfer to the central nervous system (CNS) hold significant promise for treating CNS disorders. However, following intravascular delivery, these vectors also target liver, heart, skeletal muscle, and other tissues, which may cause untoward effects. To circumvent this, we used tissue-specific, endogenous microRNAs (miRNAs) to repress rAAV expression outside the ...[more]