A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells
Young, Courtney S., Hicks, Michael R., Ermolova, Natalia V., Nakano, Haruko, Jan, Majib, Younesi, Shahab, Karumbayaram, Saravanan, Kumagai-Cresse, Chino, Wang, Derek, Zack, Jerome A., Kohn, Donald B., Nakano, Atsushi, Nelson, Stanley F., Miceli, M. Carrie, Spencer, Melissa J., Pyle, April D.
Published in Cell stem cell (07.04.2016)
Published in Cell stem cell (07.04.2016)
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Journal Article
ERBB3 and NGFR mark a distinct skeletal muscle progenitor cell in human development and hPSCs
Hicks, Michael R., Hiserodt, Julia, Paras, Katrina, Fujiwara, Wakana, Eskin, Ascia, Jan, Majib, Xi, Haibin, Young, Courtney S., Evseenko, Denis, Nelson, Stanley F., Spencer, Melissa J., Handel, Ben Van, Pyle, April D.
Published in Nature cell biology (01.01.2018)
Published in Nature cell biology (01.01.2018)
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Journal Article
CRISPR/Cas9-Mediated Correction of the Sickle Mutation in Human CD34+ cells
Hoban, Megan D, Lumaquin, Dianne, Kuo, Caroline Y, Romero, Zulema, Long, Joseph, Ho, Michelle, Young, Courtney S, Mojadidi, Michelle, Fitz-Gibbon, Sorel, Cooper, Aaron R, Lill, Georgia R, Urbinati, Fabrizia, Campo-Fernandez, Beatriz, Bjurstrom, Carmen F, Pellegrini, Matteo, Hollis, Roger P, Kohn, Donald B
Published in Molecular therapy (01.09.2016)
Published in Molecular therapy (01.09.2016)
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Journal Article
Nanoparticles systemically biodistribute to regenerating skeletal muscle in DMD
Hicks, Michael R, Liu, Xiangsheng, Young, Courtney S, Saleh, Kholoud, Ji, Ying, Jiang, Jinhong, Emami, Michael R, Mokhonova, Ekaterina, Spencer, Melissa J, Meng, Huan, Pyle, April D
Published in Journal of nanobiotechnology (29.08.2023)
Published in Journal of nanobiotechnology (29.08.2023)
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Journal Article
Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy
Young, Courtney S., Mokhonova, Ekaterina, Quinonez, Marbella, Pyle, April D., Spencer, Melissa J.
Published in Journal of neuromuscular diseases (2017)
Published in Journal of neuromuscular diseases (2017)
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Journal Article
Innate and adaptive AAV-mediated immune responses in a mouse model of Duchenne muscular dystrophy
Emami, Michael R., Espinoza, Alejandro, Young, Courtney S., Ma, Feiyang, Farahat, Philip K., Felgner, Philip L., Chamberlain, Jeffrey S., Xu, Xiangmin, Pyle, April D., Pellegrini, Matteo, Villalta, S. Armando, Spencer, Melissa J.
Published in Molecular therapy. Methods & clinical development (14.09.2023)
Published in Molecular therapy. Methods & clinical development (14.09.2023)
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Journal Article
A Human Skeletal Muscle Atlas Identifies the Trajectories of Stem and Progenitor Cells across Development and from Human Pluripotent Stem Cells
Xi, Haibin, Langerman, Justin, Sabri, Shan, Chien, Peggie, Young, Courtney S., Younesi, Shahab, Hicks, Michael, Gonzalez, Karen, Fujiwara, Wakana, Marzi, Julia, Liebscher, Simone, Spencer, Melissa, Van Handel, Ben, Evseenko, Denis, Schenke-Layland, Katja, Plath, Kathrin, Pyle, April D.
Published in Cell stem cell (02.07.2020)
Published in Cell stem cell (02.07.2020)
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Journal Article
CRISPR for Neuromuscular Disorders: Gene Editing and Beyond
Young, Courtney S, Pyle, April D, Spencer, Melissa J
Published in Physiology (Bethesda, Md.) (01.09.2019)
Published in Physiology (Bethesda, Md.) (01.09.2019)
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Journal Article
A Human Skeletal Muscle Atlas Identifies the Trajectories of Stem and Progenitor Cells across Development and from Human Pluripotent Stem Cells
Xi, Haibin, Langerman, Justin, Sabri, Shan, Chien, Peggie, Young, Courtney S., Younesi, Shahab, Hicks, Michael, Gonzalez, Karen, Fujiwara, Wakana, Marzi, Julia, Liebscher, Simone, Spencer, Melissa, Van Handel, Ben, Evseenko, Denis, Schenke-Layland, Katja, Plath, Kathrin, Pyle, April D.
Published in Cell stem cell (02.07.2020)
Published in Cell stem cell (02.07.2020)
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Journal Article
Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients
Emami, Michael R., Brimble, Mark A., Espinoza, Alejandro, Owens, Jane, Whiteley, Laurence O., Casinghino, Sandra, Lanz, Thomas A., Farahat, Philip K., Pellegrini, Matteo, Young, Courtney S., Thomas, Paul G., McNally, Elizabeth M., Villalta, S. Armando, Schattgen, Stefan A., Spencer, Melissa J.
Published in Molecular therapy. Methods & clinical development (01.12.2024)
Published in Molecular therapy. Methods & clinical development (01.12.2024)
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Journal Article
Release of Plasmid DNA-Encoding IL-10 from PLGA Microparticles Facilitates Long-Term Reversal of Neuropathic Pain Following a Single Intrathecal Administration
Soderquist, Ryan Gene, Sloane, Evan M, Loram, Lisa C, Harrison, Jacqueline A, Dengler, Ellen C, Johnson, Scott M, Amer, Luke D, Young, Courtney S, Lewis, Makenzie T, Poole, Stephen, Frank, Matthew G, Watkins, Linda R, Milligan, Erin D, Mahoney, Melissa J
Published in Pharmaceutical research (01.05.2010)
Published in Pharmaceutical research (01.05.2010)
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Journal Article
121. Targeted Gene Therapy in the Treatment of X-Linked Hyper IgM Syndrome
Kuo, Caroline Y., Hoban, Megan D., Joglekar, Alok V., Young, Courtney S., Kleinman, John, Abele, Alison N., Kohn, Donald B.
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article
735. Functional Restoration of Dystrophin Protein in HiPSC-Derived Skeletal Myotubes and Cardiomyocytes After CRISPR/Cas9-Mediated Deletion of 530-725kb of DMD
Young, Courtney S., Hicks, Michael R., Ermolova, Natalia V., Nakano, Haruko, Jan, Majib, Younesi, Shahab, Nakano, Atsushi, Nelson, Stanley F., Miceli, M. Carrie, Spencer, Melissa J., Pyle, April D.
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Journal Article
"Of Mice and Measures": A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic
Gordish-Dressman, Heather, Willmann, Raffaella, Dalle Pazze, Laura, Kreibich, Arati, van Putten, Maaike, Heydemann, Ahlke, Bogdanik, Laurent, Lutz, Cathleen, Davies, Kay, Demonbreun, Alexis R, Duan, Dongsheng, Elsey, David, Fukada, So-Ichiro, Girgenrath, Mahasweta, Patrick Gonzalez, J, Grounds, Miranda D, Nichols, Andy, Partridge, Terry, Passini, Marco, Sanarica, Francesca, Schnell, Frederick J, Wells, Dominic J, Yokota, Toshifumi, Young, Courtney S, Zhong, Zhong, Spurney, Christopher, Spencer, Melissa, De Luca, Annamaria, Nagaraju, Kanneboyina, Aartsma-Rus, Annemieke
Published in Journal of neuromuscular diseases (2018)
Published in Journal of neuromuscular diseases (2018)
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Journal Article
Conference Proceeding
“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10
Gordish-Dressman, Heather, Willmann, Raffaella, Dalle Pazze, Laura, Kreibich, Arati, van Putten, Maaike, Heydemann, Ahlke, Bogdanik, Laurent, Lutz, Cathleen, Davies, Kay, Demonbreun, Alexis R., Duan, Dongsheng, Elsey, David, Fukada, So-ichiro, Girgenrath, Mahasweta, Patrick Gonzalez, J., Grounds, Miranda D., Nichols, Andy, Partridge, Terry, Passini, Marco, Sanarica, Francesca, Schnell, Frederick J., Wells, Dominic J., Yokota, Toshifumi, Young, Courtney S., Zhong, Zhong, Spurney, Christopher, Spencer, Melissa, De Luca, Annamaria, Nagaraju, Kanneboyina, Aartsma-Rus, Annemieke
Published in Journal of neuromuscular diseases (23.10.2018)
Published in Journal of neuromuscular diseases (23.10.2018)
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Journal Article
“Of Mice and Measures”: A Project to Improve How We Advance Duchenne Muscular Dystrophy Therapies to the Clinic: First Workshop Report: Examining current findings and opportunities around the emerging D2.B10-Dmdmdx/J (D2/mdx) model in context of the classic C57BL/10ScSn-Dmdmdx/J (Bl10/mdx)
Gordish-Dressman, Heather, Willmann, Raffaella, Dalle Pazze, Laura, Kreibich, Arati, van Putten, Maaike, Heydemann, Ahlke, Bogdanik, Laurent, Lutz, Cathleen, Davies, Kay, Demonbreun, Alexis R., Duan, Dongsheng, Elsey, David, Fukada, So-ichiro, Girgenrath, Mahasweta, Patrick Gonzalez, J., Grounds, Miranda D., Nichols, Andy, Partridge, Terry, Passini, Marco, Sanarica, Francesca, Schnell, Frederick J., Wells, Dominic J., Yokota, Toshifumi, Young, Courtney S., Zhong, Zhong, Spurney, Christopher, Spencer, Melissa, De Luca, Annamaria, Nagaraju, Kanneboyina, Aartsma-Rus, Annemieke
Published in Journal of neuromuscular diseases (23.10.2018)
Published in Journal of neuromuscular diseases (23.10.2018)
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Journal Article