Highly efficient neuronal gene knockout in vivo by CRISPR-Cas9 via neonatal intracerebroventricular injection of AAV in mice
Hana, Sam, Peterson, Michael, McLaughlin, Helen, Marshall, Eric, Fabian, Attila J., McKissick, Olivia, Koszka, Kathryn, Marsh, Galina, Craft, Michael, Xu, Shanqin, Sorets, Alexander, Torregrosa, Tess, Sun, Chao, Henderson, Chris E., Lo, Shih-Ching
Published in Gene therapy (01.11.2021)
Published in Gene therapy (01.11.2021)
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Journal Article
Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice
Torregrosa, Tess, Lehman, Sydney, Hana, Sam, Marsh, Galina, Xu, Shanqin, Koszka, Kathryn, Mastrangelo, Nicole, McCampbell, Alexander, Henderson, Christopher E., Lo, Shih-Ching
Published in Gene therapy (01.08.2021)
Published in Gene therapy (01.08.2021)
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Journal Article
Efficacy and safety of a SOD1-targeting artificial miRNA delivered by AAV9 in mice are impacted by miRNA scaffold selection
Lau, Shukkwan K., Hawley, Zachary C.E., Zavodszky, Maria I., Hana, Sam, Ferretti, Daniel, Grubor, Branka, Hawes, Michael, Xu, Shanqin, Hamann, Stefan, Marsh, Galina, Cullen, Patrick, Challa, Ravi, Carlile, Thomas M., Zhang, Hang, Lee, Wan-Hung, Peralta, Andrea, Clarner, Pete, Wei, Cong, Koszka, Kathryn, Gao, Feng, Lo, Shih-Ching
Published in Molecular therapy. Nucleic acids (12.12.2023)
Published in Molecular therapy. Nucleic acids (12.12.2023)
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Journal Article
In vivo genome editing using novel AAV-PHP variants rescues motor function deficits and extends survival in a SOD1-ALS mouse model
Chen, Yi A., Kankel, Mark W., Hana, Sam, Lau, Shukkwan Kelly, Zavodszky, Maria I., McKissick, Olivia, Mastrangelo, Nicole, Dion, Jessica, Wang, Bin, Ferretti, Daniel, Koske, David, Lehman, Sydney, Koszka, Kathryn, McLaughlin, Helen, Liu, Mei, Marshall, Eric, Fabian, Attila J., Cullen, Patrick, Marsh, Galina, Hamann, Stefan, Craft, Michael, Sebalusky, Jennifer, Arnold, H. Moore, Driscoll, Rachelle, Sheehy, Adam, Luo, Yi, Manca, Sonia, Carlile, Thomas, Sun, Chao, Sigrist, Kirsten, McCampbell, Alexander, Henderson, Christopher E., Lo, Shih-Ching
Published in Gene therapy (01.05.2023)
Published in Gene therapy (01.05.2023)
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