Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders von Buffa, Valentina, Alvarez Vargas, José Roberto, Galy, Anne, Spinozzi, Simone, Rocca, Céline J

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Trabectedin and its C subunit modified analogue PM01183 attenuate nucleotide excision repair and show activity toward platinum-resistant cells von Soares, Daniele G, Machado, Miriana S, Rocca, Céline J, Poindessous, Virginie, Ouaret, Djamila, Sarasin, Alain, Galmarini, Carlos M, Henriques, João A P, Escargueil, Alexandre E, Larsen, Annette K

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BRCA2 is needed for both repair and cell cycle arrest in mammalian cells exposed to S23906, an anticancer monofunctional DNA binder von Rocca, Céline J, Soares, Daniele G, Bouzid, Hana, Henriques, João A P, Larsen, Annette K, Escargueil, Alexandre E

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Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells von Corre, Guillaume, Seye, Ababacar, Frin, Sophie, Ferrand, Maxime, Winkler, Kathrin, Luc, Cyril, Dorange, Fabien, Rocca, Céline J., Galy, Anne

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Ataxia telangiectasia mutated- and Rad3-related kinase drives both the early and the late DNA-damage response to the monofunctional antitumour alkylator S23906 von Soares, Daniele G, Battistella, Aude, Rocca, Céline J, Matuo, Renata, Henriques, João A P, Larsen, Annette K, Escargueil, Alexandre E

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Potential use of stem cells as a therapy for cystinosis von Rocca, Celine J., Cherqui, Stephanie

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Brief Reports: Lysosomal Cross‐Correction by Hematopoietic Stem Cell‐Derived Macrophages Via Tunneling Nanotubes von Naphade, Swati, Sharma, Jay, Gaide Chevronnay, Héloïse P., Shook, Michael A., Yeagy, Brian A., Rocca, Celine J., Ur, Sarah N., Lau, Athena J., Courtoy, Pierre J., Cherqui, Stephanie

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Interaction between galectin-3 and cystinosin uncovers a pathogenic role of inflammation in kidney involvement of cystinosis von Lobry, Tatiana, Miller, Roy, Nevo, Nathalie, Rocca, Celine J., Zhang, Jinzhong, Catz, Sergio D., Moore, Fiona, Thomas, Lucie, Pouly, Daniel, Bailleux, Anne, Guerrera, Ida Chiara, Gubler, Marie-Claire, Cheung, Wai W., Mak, Robert H., Montier, Tristan, Antignac, Corinne, Cherqui, Stephanie

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Impairment of chaperone‐mediated autophagy leads to selective lysosomal degradation defects in the lysosomal storage disease cystinosis von Napolitano, Gennaro, Johnson, Jennifer L, He, Jing, Rocca, Celine J, Monfregola, Jlenia, Pestonjamasp, Kersi, Cherqui, Stephanie, Catz, Sergio D

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Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis von Harrison, Frank, Yeagy, Brian A, Rocca, Celine J, Kohn, Donald B, Salomon, Daniel R, Cherqui, Stephanie

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Cystinosin, the small GTPase Rab11, and the Rab7 effector RILP regulate intracellular trafficking of the chaperone-mediated autophagy receptor LAMP2A von Zhang, Jinzhong, Johnson, Jennifer L., He, Jing, Napolitano, Gennaro, Ramadass, Mahalakshmi, Rocca, Celine, Kiosses, William B., Bucci, Cecilia, Xin, Qisheng, Gavathiotis, Evripidis, Cuervo, Ana María, Cherqui, Stephanie, Catz, Sergio D.

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Upregulation of the Rab27a-Dependent Trafficking and Secretory Mechanisms Improves Lysosomal Transport, Alleviates Endoplasmic Reticulum Stress, and Reduces Lysosome Overload in Cy... von Johnson, Jennifer L., Napolitano, Gennaro, Monfregola, Jlenia, Rocca, Celine J., Cherqui, Stephanie, Catz, Sergio D.

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Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation von Rocca, Celine J, Kreymerman, Alexander, Ur, Sarah N, Frizzi, Katie E, Naphade, Swati, Lau, Athena, Tran, Tammy, Calcutt, Nigel A, Goldberg, Jeffrey L, Cherqui, Stephanie

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CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia von Rocca, Celine J., Rainaldi, Joseph N., Sharma, Jay, Shi, Yanmeng, Haquang, Joseph H., Luebeck, Jens, Mali, Prashant, Cherqui, Stephanie

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rAAV9 combined with renal vein injection is optimal for kidney-targeted gene delivery: conclusion of a comparative study von Rocca, C J, Ur, S N, Harrison, F, Cherqui, S

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The NER proteins XPC and CSB, but not ERCC1, regulate the sensitivity to the novel DNA binder S23906: Implications for recognition and repair of antitumor alkylators von Rocca, Céline J., Poindessous, Virginie, Soares, Daniele G., Ouadrani, Karima El, Sarasin, Alain, Guérin, Eric, de Gramont, Aimery, Henriques, João A.P., Escargueil, Alexandre E., Larsen, Annette K.

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163. Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model von Chevronnay, Heloise P. Gaide, Janssens, Virginie, Van Der Smissen, Patrick, Rocca, Celine J., Liao, Xiao-Hui, Refetoff, Samuel, Pierreux, Christophe E., Cherqui, Stephanie, Courtoy, Pierre J.

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Novel insights advancing the understanding of renal Fanconi syndrome in cystinosis von Khare, Veenita, Rocca, Celine J., Mishra, Priyanka, Badell-Grau, Rafael A., Sivakumar, Anusha, Sharma, Jay, Catz, Sergio D., Farre, Jean-Claude, Cherqui, Stephanie

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A Southern Blot Protocol to Detect Chimeric Nuclease-Mediated Gene Repair von Rocca, Céline J., Abdul-Razak, Hayder H., Holmes, Michael C., Gregory, Philip D., Yáñez-Muñoz, Rafael J.

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METHOD FOR PRODUCING THERAPEUTIC RNA PLATELET VEHICLES von HUMBERT, Magali, ROCCA, Céline, BURNIER, Laurent

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