日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

AAV-mediated gene therapy in a model of SLC13A5 citrate transporter disorder rescues epileptic and metabolic phenotypes.

在 SLC13A5 柠檬酸转运蛋白疾病模型中,AAV 介导的基因治疗可挽救癫痫和代谢表型。

Bailey Lauren E, Adams Raegan M, Schackmuth Morgan K, Garza Irvin T, Knight Krishanna, Holmes Sydni K, Eller Meghan M, Lee MinJae, Bailey Rachel M

Nitrous oxide enhances MR-guided focused ultrasound delivery of gene therapy to the murine hippocampus

一氧化二氮增强磁共振引导聚焦超声向小鼠海马体输送基因疗法的效果

Bhardwaj, Deepshikha; Youssef, Ibrahim; Imphean, Darren; Holmes, Sydni K; Krishnan, Venugopal; Estill-Terpack, Sandi Jo; Diamond, Marc; Chopra, Rajiv; Bailey, Rachel M; Shah, Bhavya R

Neurological, metabolic and inflammatory phenotypes in a mouse model of ECHS1 deficiency

ECHS1缺陷小鼠模型中的神经系统、代谢和炎症表型

Eller, Meghan M; Zuberi, Aamir R; Fu, Xiaorong; Montalbano, Alina P; Nitschke, Felix; Burgess, Shawn C; Lutz, Cat; Bailey, Rachel M

Expression and distribution of rAAV9 intrathecally administered in juvenile to adolescent mice.

rAAV9鞘内注射入幼年至青春期小鼠体内的表达和分布

Garza Irvin T, Eller Meghan M, Holmes Sydni K, Schackmuth Morgan K, Bailey Rachel M

T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)

巨轴索神经病(GAN)中颈髓薄束的T2高信号

Armao, Diane; Bouldin, Thomas W; Bharucha-Goebel, Diana X; Hartman, Terry S; Gray, Steven J; Bailey, Rachel M; Saade, Dimah; Todd, Joshua J; Jain, Minal; Waite, Melissa; Bönnemann, Carsten G; Smith, J Keith

Preclinical Development of a Vectorized Artificial miRNA Gene Therapy for Tauopathies

针对tau蛋白病的载体化人工miRNA基因疗法的临床前开发

Garza, Irvin T; Snyder, Brina; Holmes, Sydni K; Pearce, Katherine M; Knight, Krishanna; Bailey, Rachel M

Intrathecal Gene Therapy for Giant Axonal Neuropathy

鞘内基因疗法治疗巨轴突神经病

Bharucha-Goebel, Diana X; Todd, Joshua J; Saade, Dimah; Norato, Gina; Jain, Minal; Lehky, Tanya; Bailey, Rachel M; Chichester, Jessica A; Calcedo, Roberto; Armao, Diane; Foley, A Reghan; Mohassel, Payam; Tesfaye, Eshetu; Carlin, Bradley P; Seremula, Beth; Waite, Melissa; Zein, Wadih M; Huryn, Laryssa A; Crawford, Thomas O; Sumner, Charlotte J; Hoke, Ahmet; Heiss, John D; Charnas, Lawrence; Hooper, Jody E; Bouldin, Thomas W; Kang, Elizabeth M; Rybin, Denis; Gray, Steven J; Bönnemann, Carsten G

Integrative multi‐omics epigenome‐wide association study identifies dysregulated DNA methylation near oligodendrocyte genes that associate with tau levels in the AD brain

整合多组学表观基因组关联研究发现,少突胶质细胞基因附近的DNA甲基化异常与AD脑中的tau蛋白水平相关。

Bailey, Rachel M; Howard, John; Knight, Joshua; Sahara, Naruhiko; Dickson, Dennis W; Lewis, Jada; Qi, Qing; Boyle, Rory; Cao, Bolin; Muniz‐Terrera, Graciela; Koychev, Ivan; Malhotra, Paresh; O'Brien, John T; Ritchie, Craig; Lawlor, Brian; Naci, Lorina; Khan, Suleiman; Soliman, Muhammad T; Murray, Sean; Szabo, Jakub; Llanos, Michael; Kim, Hakyoung; Mir, Ema; Santoyo‐Alanis, Daniela; Kingsley, Charles; Swain, Jody; Mulholland, Michele Marie; Hopkins, William D; Wisniewski, Thomas; Veraart, Jelle; Zaim‐Wadghiri, Youssef; Scholtzova, Henrieta; Egerton, Rebecca J; Sweeney, Aoife; McGuinness, Bernadette; Ross, Owen A; Beverland, David; Passmore, Anthony Peter; Heslegrave, Amanda J; Zetterberg, Henrik; Bowman, Emily; Cunningham, Emma L; Sewell, Kelsey R; Solis‐Urra, Patricio; Huang, Haiqing; Grove, George; Kramer, Arthur F; McAuley, Edward; Burns, Jeffrey M; Hillman, Charles; Vidoni, Eric D; Marsland, Anna; Kang, Chaeryon; Wan, Lu; Oberlin, Lauren; Erickson, Kirk I; Evans, Elizabeth E; Fisher, Terrence L Jr; Mishra, Vikas; Balch, Leslie; Gersz, Elaine E; Howell, Alan; Kirk, Renee; Gil‐Moore, Malgorzata; Mallow, Crystal L; Foster, Amber; Porsteinsson, Anton P; Feigin, Andrew; Zauderer, Maurice; Curl, Patti; Walker, Rod L; Donald, Christine L Mac; Andre, Jalal B; McEvoy, Linda K; Ehrlich, Kelly; Crane, Paul K; Watermeyer, Tamlyn J; Butler, Joe; Danso, Samuel O; Russell, John‐Joseph; Parra‐Rodriguez, Mario A; Nir, Talia M; Somu, Sunanda; Low, Kevin; Narula, Siddharth; Villalon‐Reina, Julio E; Gleave, Emma J; Thomopoulos, Sophia I; Lee, Noelle; Braskie, Meredith N; Thompson, Paul M; Jahanshad, Neda; Symanski, Leah E; Breidenbach, Brianne M; Spahic, Alma; Fernandes‐Taylor, Sara; Edmunds, Kyle J; Eisenmenger, Laura B; Rivera‐Rivera, Leonardo A; Driscoll, Ira; Asthana, Sanjay; Johnson, Sterling C; Wieben, Oliver; Okonkwo, Ozioma C; Shi, Hui; Archer, Derek B; Perry, Arden; Pechman, Kimberly R; Shashikumar, Niranjana; Landman, Bennett A; Hohman, Timothy J; Jefferson, Angela L; Full, Kelsie M; Chatterjee, Aadrita; Sirkis, Daniel W; Johnson, Taylor; Pham, Thu; Miller, Bruce L; Rabinovici, Gil D; Wilkins, Heather M; Yaffe, Kristine; Swerdlow, Russell H; Yokoyama, Jennifer S; Andrews, Shea J; Bernabe, Cristian S; Kotredes, Kevin P; Pandey, Ravi S; Carter, Gregory W; Sasner, Michael; Oblak, Adrian L; Howell, Gareth R; Lamb, Bruce T; Territo, Paul R; Register, Thomas C; Frye, Brett M; Negrey, Jacob D; Johnson, Corbin SC; Snyder‐Mackler, Noah; Craft, Suzanne; Montine, Thomas J; Shively, Carol A; Oatman, Stephanie R; Reddy, Joseph S; Wang, Xue; Quicksall, Zachary; Atashgaran, Amin; Vanelderen, Floor; Bergman, Jeremiah; Is, Ozkan; Carrasquillo, Minerva M; Liu, Chia‐Chen; Yamazaki, Yu; Nguyen, Thuy; Heckman, Michael G; Zhao, Na; DeTure, Michael; Murray, Melissa E; Bu, Guojun; Kanekiyo, Takahisa; Dickson, Dennis W; Allen, Mariet; Ertekin‐Taner, Nilüfer

Toward a translational gene therapy for mucolipidosis IV

迈向粘脂病IV型转化基因治疗

Snyder, Brina; Bailey, Rachel M

Sleep Abnormalities in SLC13A5 Citrate Transporter Disorder

SLC13A5柠檬酸转运蛋白紊乱症患者的睡眠异常

Adams, Raegan M; Ozlu, Can; Bailey, Lauren E; Solidum, Rayann M; Cooper, Sydney; Best, Carrie R; Elacio, Jennifer; Kavanaugh, Brian C; Brown, Tanya L; Nye, Kimberly; Liu, Judy; Porter, Brenda E; Goodspeed, Kimberly; Bailey, Rachel M