Genome editing for inborn errors of metabolism: advancing towards the clinic

por Schneller, Jessica L., Lee, Ciaran M., Bao, Gang, Venditti, Charles P.
Publicado 2017

Enrichment of transgene integrations by transient CRISPR activation of a silent reporter gene

por Mikkelsen, Nanna S., Hernandez, Sabina S., Jensen, Trine I., Schneller, Jessica L., Bak, Rasmus O.
Publicado 2023

In vivo genome editing at the albumin locus to treat methylmalonic acidemia

por Schneller, Jessica L., Lee, Ciaran M., Venturoni, Leah E., Chandler, Randy J., Li, Ang, Myung, Sangho, Cradick, Thomas J., Hurley, Ayrea E., Lagor, William R., Bao, Gang, Venditti, Charles P.
Publicado 2021

Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia

por Chandler, Randy J., Venturoni, Leah E., Liao, Jing, Hubbard, Brandon T., Schneller, Jessica L., Hoffmann, Victoria, Gordo, Susana, Zang, Shengwen, Ko, Chih‐Wei, Chau, Nelson, Chiang, Kyle, Kay, Mark A., Barzel, Adi, Venditti, Charles P.
Publicado 2021

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

por An, Ding, Schneller, Jessica L., Frassetto, Andrea, Liang, Shi, Zhu, Xuling, Park, Ji-Sun, Theisen, Matt, Hong, Sue-Jean, Zhou, Jenny, Rajendran, Raj, Levy, Becca, Howell, Rebecca, Besin, Gilles, Presnyak, Vladimir, Sabnis, Staci, Murphy-Benenato, Kerry E., Kumarasinghe, E. Sathyajith, Salerno, Timothy, Mihai, Cosmin, Lukacs, Christine M., Chandler, Randy J., Guey, Lin T., Venditti, Charles P., Martini, Paolo G.V.
Publicado 2017