Search Results - "Kuniholm, Ashley"

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease by Kim, Jinkuk, Hu, Chunguang, Moufawad El Achkar, Christelle, Black, Lauren E, Douville, Julie, Larson, Austin, Pendergast, Mary K, Goldkind, Sara F, Lee, Eunjung A, Kuniholm, Ashley, Soucy, Aubrie, Vaze, Jai, Belur, Nandkishore R, Fredriksen, Kristina, Stojkovska, Iva, Tsytsykova, Alla, Armant, Myriam, DiDonato, Renata L, Choi, Jaejoon, Cornelissen, Laura, Pereira, Luis M, Augustine, Erika F, Genetti, Casie A, Dies, Kira, Barton, Brenda, Williams, Lucinda, Goodlett, Benjamin D, Riley, Bobbie L, Pasternak, Amy, Berry, Emily R, Pflock, Kelly A, Chu, Stephen, Reed, Chantal, Tyndall, Kimberly, Agrawal, Pankaj B, Beggs, Alan H, Grant, P. Ellen, Urion, David K, Snyder, Richard O, Waisbren, Susan E, Poduri, Annapurna, Park, Peter J, Patterson, Al, Biffi, Alessandra, Mazzulli, Joseph R, Bodamer, Olaf, Berde, Charles B, Yu, Timothy W

“…A child with a neuronal ceroid lipofuscinosis was found to carry loss-of-function mutations in the gene MFSD8 ( CLN7 ). A year after genetic diagnosis, the…”
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A framework for individualized splice-switching oligonucleotide therapy by Kim, Jinkuk, Woo, Sijae, de Gusmao, Claudio M., Zhao, Boxun, Chin, Diana H., DiDonato, Renata L., Nguyen, Minh A., Nakayama, Tojo, Hu, Chunguang April, Soucy, Aubrie, Kuniholm, Ashley, Thornton, Jennifer Karlin, Riccardi, Olivia, Friedman, Danielle A., El Achkar, Christelle Moufawad, Dash, Zane, Cornelissen, Laura, Donado, Carolina, Faour, Kamli N. W., Bush, Lynn W., Suslovitch, Victoria, Lentucci, Claudia, Park, Peter J., Lee, Eunjung Alice, Patterson, Al, Philippakis, Anthony A., Margus, Brad, Berde, Charles B., Yu, Timothy W.

“…Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of individuals with genetic diseases 1 , but the systematic identification…”
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Metformin for Treatment of Cytopenias in Children and Young Adults with Fanconi Anemia by Pollard, Jessica A., Furutani, Elissa M., Liu, Shanshan, Esrick, Erica B., Cohen, Laurie, Bledsoe, Jacob, Lu, Kun, Ramirez, Maria Jose, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S., Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria V., Boulad, Farid, Carroll, Clinton, Hartung, Helge, Hont, Amy, Nakano, Taizo A., Olson, Timothy S., Sze, Sei-Gyung K., Thompson, Alexis A, Wlodarski, Marcin W., Libermann, Towia A, D'Andrea, Alan D., Grompe, Markus, Weller, Edie A., Shimamura, Akiko

“…Fanconi anemia (FA), a genetic disorder affecting DNA repair, is characterized by bone marrow failure and cancer susceptibility. In FA mouse models, metformin…”
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Metformin for treatment of cytopenias in children and young adults with Fanconi anemia by Pollard, Jessica A, Furutani, Elissa, Liu, Shanshan, Esrick, Erica, Cohen, Laurie E, Bledsoe, Jacob, Liu, Chih-Wei, Lu, Kun, de Haro, Maria Jose Ramirez, Surrallés, Jordi, Malsch, Maggie, Kuniholm, Ashley, Galvin, Ashley, Armant, Myriam, Kim, Annette S, Ballotti, Kaitlyn, Moreau, Lisa, Zhou, Yu, Babushok, Daria, Boulad, Farid, Carroll, Clint, Hartung, Helge, Hont, Amy, Nakano, Taizo, Olson, Tim, Sze, Sei-Gyung, Thompson, Alexis A, Wlodarski, Marcin W, Gu, Xuesong, Libermann, Towia A, D'Andrea, Alan, Grompe, Markus, Weller, Edie, Shimamura, Akiko

“…Fanconi anemia (FA), a genetic DNA repair disorder characterized by marrow failure and cancer susceptibility. In FA mice, metformin improves blood counts and…”
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