Tailoring the AAV2 capsid vector for bone-targeting.
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ABSTRACT:
SUBMITTER: Almeciga-Diaz CJ
PROVIDER: S-EPMC6266866 | biostudies-literature | 2018 Oct
REPOSITORIES: biostudies-literature
Publications
Tailoring the AAV2 capsid vector for bone-targeting.
Alméciga-Díaz Carlos J CJ Montaño Adriana M AM Barrera Luis A LA Tomatsu Shunji S
Pediatric research 20181015 4
<h4>Background</h4>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.<h4>Methods</h4>To increase vector affinity to HA, an aspartic acid octapeptide (D8) was inserted immediately after the N-terminal region of the VP2 c ...[more]
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