A novel gene editing system to treat both Tay-Sachs and Sandhoff diseases

por Ou, Li, Przybilla, Michael J, Tăbăran, Alexandru-Flaviu, Overn, Paula, O’Sullivan, M. Gerard, Jiang, Xuntian, Sidhu, Rohini, Kell, Pamela J., Ory, Daniel S., Whitley, Chester B.
Publicado 2020

A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1

por Prabhu, Anika V., Kang, Insung, De Pace, Raffaella, Wassif, Christopher A., Fujiwara, Hideji, Kell, Pamela, Jiang, Xuntian, Ory, Daniel S., Bonifacino, Juan S., Ward, Michael E., Porter, Forbes D.
Publicado 2021

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis

por Przybilla, Michael J., Stewart, Christine, Carlson, Timothy W., Ou, Li, Koniar, Brenda L., Sidhu, Rohini, Kell, Pamela J., Jiang, Xuntian, Jarnes, Jeanine R., O'Sullivan, M. Gerard, Whitley, Chester B.
Publicado 2021

A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis

por Kell, Pamela, Sidhu, Rohini, Qian, Mingxing, Mishra, Sonali, Nicoli, Elena-Raluca, D'Souza, Precilla, Tifft, Cynthia J., Gross, Amanda L., Gray-Edwards, Heather L., Martin, Douglas R., Sena- Esteves, Miguel, Dietzen, Dennis J., Singh, Manmilan, Luo, Jingqin, Schaffer, Jean E., Ory, Daniel S., Jiang, Xuntian
Publicado 2023

Eggs early in complementary feeding increase choline pathway biomarkers and DHA: a randomized controlled trial in Ecuador

por Iannotti, Lora L, Lutter, Chessa K, Waters, William F, Gallegos Riofrío, Carlos Andres, Malo, Carla, Reinhart, Gregory, Palacios, Ana, Karp, Celia, Chapnick, Melissa, Cox, Katherine, Aguirre, Santiago, Narvaez, Luis, López, Fernando, Sidhu, Rohini, Kell, Pamela, Jiang, Xuntian, Fujiwara, Hideji, Ory, Daniel S, Young, Rebecca, Stewart, Christine P
Publicado 2017