In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions
Nicolas, Clara T., VanLith, Caitlin J., Hickey, Raymond D., Du, Zeji, Hillin, Lori G., Guthman, Rebekah M., Cao, William J., Haugo, Benjamin, Lillegard, Annika, Roy, Diya, Bhagwate, Aditya, O’Brien, Daniel, Kocher, Jean-Pierre, Kaiser, Robert A., Russell, Stephen J., Lillegard, Joseph B.
Published in Nature communications (25.08.2022)
Published in Nature communications (25.08.2022)
Get full text
Journal Article
Development of a porcine model of phenylketonuria with a humanized R408W mutation for gene editing
Kaiser, Robert A, Carlson, Daniel F, Allen, Kari L, Webster, Dennis A, VanLith, Caitlin J, Nicolas, Clara T, Hillin, Lori G, Yu, Yue, Kaiser, Catherine W, Wahoff, William R, Hickey, Raymond D, Watson, Adrienne L, Winn, Shelley R, Thöny, Beat, Kern, Douglas R, Harding, Cary O, Lillegard, Joseph B
Published in PloS one (25.01.2021)
Published in PloS one (25.01.2021)
Get full text
Journal Article
Use of an adeno‐associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model
Kaiser, Robert A., Weber, Nicholas D., Trigueros‐Motos, Laia, Allen, Kari L., Martinez, Michael, Cao, William, VanLith, Caitlin J., Hillin, Lori G., Douar, Anne, González‐Aseguinolaza, Gloria, Aldabe, Rafael, Lillegard, Joseph B.
Published in Journal of inherited metabolic disease (01.11.2021)
Published in Journal of inherited metabolic disease (01.11.2021)
Get full text
Journal Article
In vivo lentiviral vector gene therapy to cure hereditary tyrosinemia type 1 and prevent development of precancerous and cancerous lesions
Nicolas, Clara T, Vanlith, Caitlin J, Allen, Kari L, Hickey, Raymond D, Du, Zeji, Hillin, Lori G, Guthman, Rebekah M, Cao, William J, Bhagwate, Aditya, Daniel O’brien, Kocher, Jean-Pierre, Kaiser, Robert A, Russell, Stephen J, Lillegard, Joseph B
Published in bioRxiv (16.03.2021)
Published in bioRxiv (16.03.2021)
Get full text
Paper