Treating Cardiovascular Disease with Liver Genome Engineering

por Hurley, Ayrea, Lagor, William R.
Publicado 2022

Adeno-Associated virus 8 delivers an immunomodulatory peptide to mouse liver more efficiently than to rat liver

por Wang, Yuqing, Hurley, Ayrea, De Giorgi, Marco, Tanner, Mark R., Hu, Rong-Chi, Pennington, Michael W., Lagor, William R., Beeton, Christine
Publicado 2023

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing

por Li, Ang, Lee, Ciaran M., Hurley, Ayrea E., Jarrett, Kelsey E., De Giorgi, Marco, Lu, Weiqi, Balderrama, Karol S., Doerfler, Alexandria M., Deshmukh, Harshavardhan, Ray, Anirban, Bao, Gang, Lagor, William R.
Publicado 2018

AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

por Li, Ang, Tanner, Mark R., Lee, Ciaran M., Hurley, Ayrea E., De Giorgi, Marco, Jarrett, Kelsey E., Davis, Timothy H., Doerfler, Alexandria M., Bao, Gang, Beeton, Christine, Lagor, William R.
Publicado 2020

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

Targeting the Apoa1 locus for liver-directed gene therapy

por De Giorgi, Marco, Li, Ang, Hurley, Ayrea, Barzi, Mercedes, Doerfler, Alexandria M., Cherayil, Nikitha A., Smith, Harrison E., Brown, Jonathan D., Lin, Charles Y., Bissig, Karl-Dimiter, Bao, Gang, Lagor, William R.
Publicado 2021

LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study

por Doerfler, Alexandria M., Park, So Hyun, Assini, Julia M., Youssef, Amer, Saxena, Lavanya, Yaseen, Adam B., De Giorgi, Marco, Chuecos, Marcel, Hurley, Ayrea E., Li, Ang, Marcovina, Santica M., Bao, Gang, Boffa, Michael B., Koschinsky, Marlys L., Lagor, William R.
Publicado 2022

TFEB regulates murine liver cell fate during development and regeneration

por Pastore, Nunzia, Huynh, Tuong, Herz, Niculin J., Calcagni’, Alessia, Klisch, Tiemo J., Brunetti, Lorenzo, Kim, Kangho Ho, De Giorgi, Marco, Hurley, Ayrea, Carissimo, Annamaria, Mutarelli, Margherita, Aleksieva, Niya, D’Orsi, Luca, Lagor, William R., Moore, David D., Settembre, Carmine, Finegold, Milton J., Forbes, Stuart J., Ballabio, Andrea
Publicado 2020

Speg interactions that regulate the stability of excitation-contraction coupling protein complexes in triads and dyads

por Lee, Chang Seok, Jung, Sung Yun, Yee, Rachel Sue Zhen, Agha, Nadia H., Hong, Jin, Chang, Ting, Babcock, Lyle W., Fleischman, Jorie D., Clayton, Benjamin, Hanna, Amy D., Ward, Christopher S., Lanza, Denise, Hurley, Ayrea E., Zhang, Pumin, Wehrens, Xander H. T., Lagor, William R., Rodney, George G., Hamilton, Susan L.
Publicado 2023

In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene

por De Giorgi, Marco, Park, So Hyun, Castoreno, Adam, Cao, Mingming, Hurley, Ayrea, Saxena, Lavanya, Chuecos, Marcel A., Walkey, Christopher J., Doerfler, Alexandria M., Furgurson, Mia N., Ljungberg, M. Cecilia, Patel, Kalyani R., Hyde, Sarah, Chickering, Tyler, Lefebvre, Stephanie, Wassarman, Kelly, Miller, Patrick, Qin, June, Schlegel, Mark K., Zlatev, Ivan, Li, Rich Gang, Kim, Jong, Martin, James F., Bissig, Karl-Dimiter, Jadhav, Vasant, Bao, Gang, Lagor, William R.
Publicado 2023