Targeted approaches to induce immune tolerance for Pompe disease therapy

por Doerfler, Phillip A, Nayak, Sushrusha, Corti, Manuela, Morel, Laurence, Herzog, Roland W, Byrne, Barry J
Publicado 2016

Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent

por Nayak, Sushrusha, Doerfler, Phillip A., Porvasnik, Stacy L., Cloutier, Denise D., Khanna, Richie, Valenzano, Ken J., Herzog, Roland W., Byrne, Barry J.
Publicado 2014

Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing

por Leibowitz, Mitchell L., Papathanasiou, Stamatis, Doerfler, Phillip A., Blaine, Logan J., Sun, Lili, Yao, Yu, Zhang, Cheng-Zhong, Weiss, Mitchell J., Pellman, David
Publicado 2021

Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations

por Woodard, Kaitly J., Doerfler, Phillip A., Mayberry, Kalin D., Sharma, Akshay, Levine, Rachel, Yen, Jonathan, Valentine, Virginia, Palmer, Lance E., Valentine, Marc, Weiss, Mitchell J.
Publicado 2022

Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin

por Doerfler, Phillip A., Feng, Ruopeng, Li, Yichao, Palmer, Lance E., Porter, Shaina N., Bell, Henry W., Crossley, Merlin, Pruett-Miller, Shondra M., Cheng, Yong, Weiss, Mitchell J.
Publicado 2021

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice

por Doyle, Brendan M., Turner, Sara M.F., Sunshine, Michael D., Doerfler, Phillip A., Poirier, Amy E., Vaught, Lauren A., Jorgensen, Marda L., Falk, Darin J., Byrne, Barry J., Fuller, David D.
Publicado 2019

Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells

por Qin, Kunhua, Huang, Peng, Feng, Ruopeng, Keller, Cheryl A., Peslak, Scott A., Khandros, Eugene, Saari, Megan, Lan, Xianjiang, Mayuranathan, Thiyagaraj, Doerfler, Phillip A., Abdulmalik, Osheiza, Giardine, Belinda, Chou, Stella T., Shi, Junwei, Hardison, Ross C., Weiss, Mitchell J., Blobel, Gerd A.
Publicado 2022