Tailoring the AAV2 capsid vector for bone-targeting.

ABSTRACT:

Background

Targeting specific tissues remains a major challenge to the promise of gene therapy. For example, several strategies have failed to target adeno-associated virus 2 (AAV2) vectors, to bone. We have evaluated in vitro and in vivo the affinity of an AAV2 vector to bone matrix, hydroxyapatite (HA) to treat Mucopolysacccharidosis IVA.

Methods

To increase vector affinity to HA, an aspartic acid octapeptide (D8) was inserted immediately after the N-terminal region of the VP2 capsid protein. The modified vector had physical titers and transduction efficiencies comparable to the unmodified vector.

Results

The bone-targeting vector had significantly higher HA affinity and vector genome copies in bone than the unmodified vector. The modified vector was also released f

SUBMITTER: Almeciga-Diaz CJ 

PROVIDER: S-EPMC6266866 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature