Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates.

by Jeff L Ellsworth, Jacinthe Gingras, Laura J Smith, Hillard Rubin, Tania A Seabrook, Kruti Patel, Nicole Zapata, Kevin Olivieri, Michael O'Callaghan, Elizabeth Chlipala, Pablo Morales, Albert Seymour
Published 2019-01-01

Molecular characterization of precise in vivo targeted gene integration in human cells using AAVHSC15.

by Huei-Mei Chen, Rachel Resendes, Azita Ghodssi, Danielle Sookiasian, Michael Tian, Serena Dollive, Laura Adamson-Small, Nancy Avila, Cagdas Tazearslan, John F Thompson, Jeff L Ellsworth, Omar Francone, Albert Seymour, Jason B Wright
Published 2020-01-01

Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

by Seemin S. Ahmed, Hillard Rubin, Minglun Wang, Deiby Faulkner, Arnold Sengooba, Serena N. Dollive, Nancy Avila, Jeff L. Ellsworth, Diana Lamppu, Maria Lobikin, Jason Lotterhand, Laura Adamson-Small, Teresa Wright, Albert Seymour, Omar L. Francone
Published 2020-06-01