Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside

por Cherqui, Stephanie
Publicado 2021

Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia

por Sivakumar, Anusha, Cherqui, Stephanie
Publicado 2022

Identification and Characterisation of the Murine Homologue of the Gene Responsible for Cystinosis, Ctns

por Cherqui, Stéphanie, Kalatzis, Vasiliki, Forestier, Lionel, Poras, Isabelle, Antignac, Corinne
Publicado 2000

rAAV9 combined with renal vein injection is optimal for kidney-targeted gene delivery: conclusion of a comparative study

por Rocca, Céline J., Ur, Sarah N., Harrison, Frank, Cherqui, Stephanie
Publicado 2014

Macrophage polarization impacts tunneling nanotube formation and intercellular organelle trafficking

por Goodman, Spencer, Naphade, Swati, Khan, Meisha, Sharma, Jay, Cherqui, Stephanie
Publicado 2019

Quantitative in vivo and ex vivo confocal microscopy analysis of corneal cystine crystals in the Ctns(−/−) knockout mouse

por Simpson, Jennifer, Nien, Chyong Jy, Flynn, Kevin, Jester, Brian, Cherqui, Stephanie, Jester, James
Publicado 2011

DYNC1LI2 regulates localization of the chaperone-mediated autophagy receptor LAMP2A and improves cellular homeostasis in cystinosis

por Rahman, Farhana, Johnson, Jennifer L., Zhang, Jinzhong, He, Jing, Pestonjamasp, Kersi, Cherqui, Stephanie, Catz, Sergio D.
Publicado 2021

Rescue of Alzheimer’s disease phenotype in a mouse model by transplantation of wild-type hematopoietic stem and progenitor cells

por Mishra, Priyanka, Silva, Alexander, Sharma, Jay, Nguyen, Jacqueline, Pizzo, Donald P., Hinz, Denise, Sahoo, Debashis, Cherqui, Stephanie
Publicado 2023

Impairment of chaperone-mediated autophagy leads to selective lysosomal degradation defects in the lysosomal storage disease cystinosis

por Napolitano, Gennaro, Johnson, Jennifer L, He, Jing, Rocca, Celine J, Monfregola, Jlenia, Pestonjamasp, Kersi, Cherqui, Stephanie, Catz, Sergio D
Publicado 2015

Muscle wasting and adipose tissue browning in infantile nephropathic cystinosis

por Cheung, Wai W., Cherqui, Stephanie, Ding, Wei, Esparza, Mary, Zhou, Ping, Shao, Jianhua, Lieber, Richard L., Mak, Robert H.
Publicado 2015

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia

por Rocca, Celine J., Rainaldi, Joseph N., Sharma, Jay, Shi, Yanmeng, Haquang, Joseph H., Luebeck, Jens, Mali, Prashant, Cherqui, Stephanie
Publicado 2020

Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model

por Goodman, Spencer, Khan, Meisha, Sharma, Jay, Li, Zijie, Cano, Jose, Castellanos, Carlos, Estrada, Monica V., Gertsman, Ilya, Cherqui, Stephanie
Publicado 2021

Non-invasive intradermal imaging of cystine crystals in cystinosis

por Bengali, Marya, Goodman, Spencer, Sun, Xiaoying, Dohil, Magdalene A., Dohil, Ranjan, Newbury, Robert, Lobry, Tatiana, Hernandez, Laura, Antignac, Corinne, Jain, Sonia, Cherqui, Stephanie
Publicado 2021

Hematopoietic Stem Cell Gene Therapy for Brain Metastases Using Myeloid Cell–Specific Gene Promoters

por Andreou, Tereza, Rippaus, Nora, Wronski, Krzysztof, Williams, Jennifer, Taggart, David, Cherqui, Stephanie, Sunderland, Ashley, Kartika, Yolanda D, Egnuni, Teklu, Brownlie, Rebecca J, Mathew, Ryan K, Holmen, Sheri L, Fife, Christopher, Droop, Alastair, Lorger, Mihaela
Publicado 2019

Tunneling Nanotubes and Gap Junctions–Their Role in Long-Range Intercellular Communication during Development, Health, and Disease Conditions

por Ariazi, Jennifer, Benowitz, Andrew, De Biasi, Vern, Den Boer, Monique L., Cherqui, Stephanie, Cui, Haifeng, Douillet, Nathalie, Eugenin, Eliseo A., Favre, David, Goodman, Spencer, Gousset, Karine, Hanein, Dorit, Israel, David I., Kimura, Shunsuke, Kirkpatrick, Robert B., Kuhn, Nastaran, Jeong, Claire, Lou, Emil, Mailliard, Robbie, Maio, Stephen, Okafo, George, Osswald, Matthias, Pasquier, Jennifer, Polak, Roel, Pradel, Gabriele, de Rooij, Bob, Schaeffer, Peter, Skeberdis, Vytenis A., Smith, Ian F., Tanveer, Ahmad, Volkmann, Niels, Wu, Zhenhua, Zurzolo, Chiara
Publicado 2017