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Lipid Nanoparticles for In Vivo Lung Delivery of CRISPR-Cas9 Ribonucleoproteins Allow Gene Editing of Clinical Targets Haley, RM; Padilla, MS; El-Mayta, RD; Joseph, RA; Weber, JA; Figueroa-Espada, CG; Mukalel, AJ; Ricciardi, AS; Palanki, R; Geisler, HC; Jester, MT; Davidson, BL; Mitchell, MJ Share Save
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CASP8 intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer's disease risk Nguyen, Lien; Ajredini, Ramadan; Guo, Shu; Romano, Lisa E. L.; Tomas, Rodrigo F.; Bell, Logan R.; Ranum, Paul T.; Zu, Tao; Coronel, Monica Banez; Kelley, Chase P.; Redding-Ochoa, Javier; Nizamis, Evangelos; Melloni, Alexandra; Connors, Theresa R.; Gaona, Angelica; Thangaraju, Kiruphagaran; Pletnikova, Olga; Clark, H. Brent; Davidson, Beverly L.; Yachnis, Anthony T.; Golde, Todd E.; Lou, Xiangyang; Wang, Eric T.; Renton, Alan E.; Goate, Alison; Valdmanis, Paul N.; Prokop, Stefan; Troncoso, Juan C.; Hyman, Bradley T.; Ranum, Laura P. W. Share Save
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APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease Jackson, Rosemary J.; Keiser, Megan S.; Meltzer, Jonah C.; Fykstra, Dustin P.; Dierksmeier, Steven E.; Hajizadeh, Soroush; Kreuzer, Johannes; Morris, Robert; Melloni, Alexandra; Nakajima, Tsuneo; Tecedor, Luis; Ranum, Paul T.; Carrell, Ellie; Chen, Yonghong; Nishtar, Maryam A.; Holtzman, David M.; Haas, Wilhelm; Davidson, Beverly L.; Hyman, Bradley T. Share Save
A novel iPSC model reveals selective vulnerability of neurons in multiple sulfatase deficiency Pham, Vi; Finoti, Livia Sertori; Cassidy, Margaret M.; Maguire, Jean Ann; Gagne, Alyssa L.; Waxman, Elisa A.; French, Deborah L.; King, Kaitlyn; Zhou, Zitao; Gelb, Michael H.; Wongkittichote, Parith; Hong, Xinying; Schlotawa, Lars; Davidson, Beverly L.; Ahrens-Nicklas, Rebecca C. Share Save
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF Carrell, Ellie M.; Chen, Yong Hong; Ranum, Paul T.; Coffin, Stephanie L.; Singh, Larry N.; Tecedor, Luis; Keiser, Megan S.; Hudry, Eloise; Hyman, Bradley T.; Davidson, Beverly L. Share Save
Haplotyping SNPs for allele-specific gene editing of the expanded huntingtin allele using long-read sequencing (vol 4, 100146, 2023) Fang, Li; Monteys, Alex Mas; Durr, Alexandra; Keiser, Megan; Cheng, Congsheng; Harapanahalli, Akhil; Gonzalez-Alegre, Pedro; Davidson, Beverly L.; Wang, Kai Share Save
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching Dawicki-McKenna, Jennine M.; Felix, Alex J.; Waxman, Elisa A.; Cheng, Congsheng; Amado, Defne A.; Ranum, Paul T.; Bogush, Alexey; Dungan, Lea V.; Maguire, Jean Ann; Gagne, Alyssa L.; Heller, Elizabeth A.; French, Deborah L.; Davidson, Beverly L.; Prosser, Benjamin L. Share Save
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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective Sabatino, Denise E.; Bushman, Frederic D.; Chandler, Randy J.; Crystal, Ronald G.; Davidson, Beverly L.; Dolmetsch, Ricardo; Eggan, Kevin C.; Gao, Guangping; Gil-Farina, Irene; Kay, Mark A.; McCarty, Douglas M.; Montini, Eugenio; Ndu, Adora; Yuan, Jing Share Save
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