Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]
Allende, Maria L., Cook, Emily K., Larman, Bridget C., Nugent, Adrienne, Brady, Jacqueline M., Golebiowski, Diane, Sena-Esteves, Miguel, Tifft, Cynthia J., Proia, Richard L.
Published in Journal of lipid research (01.03.2018)
Published in Journal of lipid research (01.03.2018)
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The Central Nervous System-Restricted Transcription Factor Olig2 Opposes p53 Responses to Genotoxic Damage in Neural Progenitors and Malignant Glioma
Mehta, Shwetal, Huillard, Emmanuelle, Kesari, Santosh, Maire, Cecile L., Golebiowski, Diane, Harrington, Emily P., Alberta, John A., Kane, Michael F., Theisen, Matthew, Ligon, Keith L., Rowitch, David H., Stiles, Charles D.
Published in Cancer cell (08.03.2011)
Published in Cancer cell (08.03.2011)
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Requirement of the ATM/p53 Tumor Suppressor Pathway for Glucose Homeostasis
Armata, Heather L., Golebiowski, Diane, Jung, Dae Young, Ko, Hwi Jin, Kim, Jason K., Sluss, Hayla K.
Published in Molecular and Cellular Biology (01.12.2010)
Published in Molecular and Cellular Biology (01.12.2010)
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Anti-SOD1 Nanobodies That Stabilize Misfolded SOD1 Proteins Also Promote Neurite Outgrowth in Mutant SOD1 Human Neurons
Kumar, Meenakshi Sundaram, Fowler-Magaw, Megan E, Kulick, Daniel, Boopathy, Sivakumar, Gadd, Del Hayden, Rotunno, Melissa, Douthwright, Catherine, Golebiowski, Diane, Yusuf, Issa, Xu, Zuoshang, Brown, Jr, Robert H, Sena-Esteves, Miguel, O'Neil, Alison L, Bosco, Daryl A
Published in International journal of molecular sciences (16.12.2022)
Published in International journal of molecular sciences (16.12.2022)
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A novel mouse model of Duchenne muscular dystrophy carrying a multi-exonic Dmd deletion exhibits progressive muscular dystrophy and early-onset cardiomyopathy
Wong, Tatianna Wai Ying, Ahmed, Abdalla, Yang, Grace, Maino, Eleonora, Steiman, Sydney, Hyatt, Elzbieta, Chan, Parry, Lindsay, Kyle, Wong, Nicole, Golebiowski, Diane, Schneider, Joel, Delgado-Olguín, Paul, Ivakine, Evgueni A, Cohn, Ronald D
Published in Disease models & mechanisms (01.09.2020)
Published in Disease models & mechanisms (01.09.2020)
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A novel dual-plasmid platform provides scalable transfection yielding improved productivity and packaging across multiple AAV serotypes and genomes
van Lieshout, Laura P., Rubin, Miranda, Costa-Grant, Katrina, Ota, Stacy, Golebiowski, Diane, Panico, Troy, Wiberg, Eli, Szymczak, Klaudia, Gilmore, Richard, Stanvick, Marissa, Burnham, Brenda, Gagnon, Jeff, Iwuchukwu, Ifeyinwa, Yang, Guang, Ghazi, Iraj, Meola, Alex, Dickerson, Ryan, Thiers, Thomas, Mustich, Luke, Hayes, April, Rivas, Israel, Lotterhand, Jason, Avila, Nancy, McGivney, James, Yin, Jin, Kelly, Tim
Published in Molecular therapy. Methods & clinical development (08.06.2023)
Published in Molecular therapy. Methods & clinical development (08.06.2023)
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Micro-utrophin Improves Cardiac and Skeletal Muscle Function of Severely Affected D2/ mdx Mice
Kennedy, Tahnee L, Guiraud, Simon, Edwards, Ben, Squire, Sarah, Moir, Lee, Babbs, Arran, Odom, Guy, Golebiowski, Diane, Schneider, Joel, Chamberlain, Jeffrey S, Davies, Kay E
Published in Molecular therapy. Methods & clinical development (14.12.2018)
Published in Molecular therapy. Methods & clinical development (14.12.2018)
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Detailed Protocol for the Novel and Scalable Viral Vector Upstream Process for AAV Gene Therapy Manufacturing
Selvaraj, Nagarathinam, Wang, Chao-Kuei, Bowser, Brian, Broadt, Trevor, Shaban, Samir, Burns, Jenna, Saptharishi, Nirmala, Pechan, Peter, Golebiowski, Diane, Alimardanov, Asaf, Yang, Nora, Mitra, George, Vepachedu, Ramarao
Published in Human gene therapy (01.08.2021)
Published in Human gene therapy (01.08.2021)
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Journal Article
Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy
Birch, Sharla M, Lawlor, Michael W, Conlon, Thomas J, Guo, Lee-Jae, Crudele, Julie M, Hawkins, Eleanor C, Nghiem, Peter P, Ahn, Mihye, Meng, Hui, Beatka, Margaret J, Fickau, Brittany A, Prieto, Juan C, Styner, Martin A, Struharik, Michael J, Shanks, Courtney, Brown, Kristy J, Golebiowski, Diane, Bettis, Amanda K, Balog-Alvarez, Cynthia J, Clement, Nathalie, Coleman, Kirsten E, Corti, Manuela, Pan, Xiufang, Hauschka, Stephen D, Gonzalez, J Patrick, Morris, Carl A, Schneider, Joel S, Duan, Dongsheng, Chamberlain, Jeffrey S, Byrne, Barry J, Kornegay, Joe N
Published in Science translational medicine (04.01.2023)
Published in Science translational medicine (04.01.2023)
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Journal Article
Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice
Lahey, Hannah G., Webber, Chelsea J., Golebiowski, Diane, Izzo, Cassandra M., Horn, Erin, Taghian, Toloo, Rodriguez, Paola, Batista, Ana Rita, Ellis, Lauren E., Hwang, Misako, Martin, Douglas R., Gray-Edwards, Heather, Sena-Esteves, Miguel
Published in Molecular therapy (07.10.2020)
Published in Molecular therapy (07.10.2020)
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Journal Article
Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain
Golebiowski, Diane, van der Bom, Imramsjah M J, Kwon, Churl-Su, Miller, Andrew D, Petrosky, Keiko, Bradbury, Allison M, Maitland, Stacy, Kühn, Anna Luisa, Bishop, Nina, Curran, Elizabeth, Silva, Nilsa, GuhaSarkar, Dwijit, Westmoreland, Susan V, Martin, Douglas R, Gounis, Matthew J, Asaad, Wael F, Sena-Esteves, Miguel
Published in Human gene therapy (01.06.2017)
Published in Human gene therapy (01.06.2017)
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Journal Article
Efficient Gene Silencing in Brain Tumors with Hydrophobically Modified siRNAs
Osborn, Maire F, Coles, Andrew H, Golebiowski, Diane, Echeverria, Dimas, Moazami, Michael P, Watts, Jonathan K, Sena-Esteves, Miguel, Khvorova, Anastasia
Published in Molecular cancer therapeutics (01.06.2018)
Published in Molecular cancer therapeutics (01.06.2018)
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Journal Article
710. Optimization of AAV Vector Design for Safe Expression of β-N-Acetylhexosaminidase in the Brain for Tay-Sachs Disease Gene Therapy
Golebiowski, Diane, Petrosky, Keiko, Van der Marel, Kajo, Hutto, Elizabeth, Curran, Elizabeth, Balkanska-Sinclair, Elena, Bishop, Nina, Santos, Rosemary, Cummings Macri, Sheila, Martin, Douglas, Gounis, Matthew, Pilitsis, Julie, Asaad, Wael, Sena-Esteves, Miguel
Published in Molecular therapy (01.05.2015)
Published in Molecular therapy (01.05.2015)
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Journal Article
Modulation of AAV Vector Transgene Expression
Weismann, Cara, Sena-Esteves, Miguel, Stoica, Lorelei, Golebiowski, Diane
Year of Publication 23.12.2021
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Year of Publication 23.12.2021
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