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

Dual-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase 1/2 trial

双载体rAAVrh8基因疗法治疗GM2神经节苷脂沉积症:一项1/2期试验

Eichler, Florian; Cataltepe, Oguz I; Daci, Rrita; Puri, Ajit S; Taghian, Toloo; Jiang, Xuntian; Shazeeb, Mohammed Salman; Kuhn, Anna; Hader, Asma; Celik, Hakki; Vardar, Zeynep; Lewis, Connor J; Artinian, Rebecca; Nagy, Amanda; Vachha, Behroze; Thompson, Robert; Gallagher, Thomas; Bateman, Scot; Parzych, Julia; Spanakis, Spiro G; Vaughn, Toby A; Pier, Kirsten; De Boever, Erika; Abbott, Mary-Alice; D Ambrosio, Eleonora; Kokoski, Danielle; Blackwood, Meghan; Drummond, Elise; Ratai, Eva-Maria; Townsend, Elise L; McLaughlin, Haley; Tifft, Cynthia J; Keeler, Allison M; Sena-Esteves, Miguel; Gray-Edwards, Heather L; Flotte, Terence R

Blood phenylalanine lowering partially reverses white matter changes in a mouse model of phenylketonuria

降低血液中苯丙氨酸水平可部分逆转苯丙酮尿症小鼠模型中的白质变化

Manek, Rachna; Huang, Weixiao; Huang, Yinyin; Guo, Lilu; Cornell, Cathleen S; Shazeeb, Mohammed Salman; Verbitsky, Alexander; Jackson, Robert; Johnson, Jennifer; Berthelette, Patricia; Yu, Dan; Pfister, Edith L; Bangari, Dinesh; Ying, Xiaoyou; Kumar, Dinesh; Mueller, Christian; Kyostio-Moore, Sirkka

Differential tractography: an imaging marker for tissue degeneration in neurodegenerative diseases

差异性纤维束成像:神经退行性疾病中组织退化的影像学标志物

Lewis, Connor J; Vardar, Zeynep; Kühn, Anna Luisa; Johnston, Jean M; D'Souza, Precilla; Yousef, Muhammad H; Gahl, William A; Shazeeb, Mohammed Salman; Tifft, Cynthia J; Acosta, Maria T

A Method for Imaging the Ischemic Penumbra with MRI Using Intravoxel Incoherent Motion

一种利用体素内不相干运动进行MRI缺血半暗带成像的方法

Liu, Mira M; Saadat, Niloufar; Roth, Steven P; Niekrasz, Marek A; Giurcanu, Mihai; Shazeeb, Mohammed Salman; Carroll, Timothy J; Christoforidis, Gregory A

Corrigendum to "Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients" [Molecular Genetics and Metabolism 2025 Mar;144(3):109025]

对“II 型晚期婴儿和青少年 GM1 神经节苷脂病患者的 MRI 脑体积自然史进展”的更正 [分子遗传学和代谢 2025 年 3 月;144(3):109025]

Kolstad, Josephine; Zoppo, Christopher; Johnston, Jean M; D'Souza, Precilla; Kühn, Anna Luisa; Vardar, Zeynep; Peker, Ahmet; Hader, Asma; Celik, Hakki; Lewis, Connor J; Lindsay, Clifford; Rentiya, Zubir S; Lebel, Catherine; Vedantham, Srinivasan; Vachha, Behroze; Gray-Edwards, Heather L; Acosta, Maria T; Tifft, Cynthia J; Shazeeb, Mohammed Salman

Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients

II型晚期婴儿期和青少年期GM1神经节苷脂沉积症患者的MRI脑容量自然史进展

Kolstad, Josephine; Zoppo, Christopher; Johnston, Jean M; D'Souza, Precilla; Kühn, Anna Luisa; Vardar, Zeynep; Peker, Ahmet; Hader, Asma; Celik, Hakki; Lewis, Connor J; Lindsay, Clifford; Rentiya, Zubir S; Lebel, Catherine; Vedantham, Srinivasan; Vachha, Behroze; Gray-Edwards, Heather L; Acosta, Maria T; Tifft, Cynthia J; Shazeeb, Mohammed Salman

Comprehensive Segmentation of Deep Grey Nuclei From Structural MRI Data

基于结构磁共振成像数据的深部灰质核团综合分割

Saranathan, Manojkumar; Cogliandro, Giuseppina; Hicks, Thomas; Patterson, Dianne; Vachha, Behroze; Hader, Asma; Shazeeb, Mohammed Salman; Cacciola, Alberto

Development of an in-vitro model based on patient vessel geometry for simulated use testing in neurointerventional surgery

基于患者血管几何形状的体外模型开发,用于神经介入手术的模拟使用测试

Epshtein, Mark; Shazeeb, Mohammed Salman; Kühn, Anna Luisa; Anagnostakou, Vania; Raskett, Christopher M; King, Robert M; Goyal, Mayank; Mendes Pereira, Vitor; Arthur, Adam S; Puri, Ajit S; Fiorella, David; Gounis, Matthew J

A Case for Automated Segmentation of MRI Data in Neurodegenerative Diseases: Type II GM1 Gangliosidosis

神经退行性疾病中MRI数据自动分割的必要性:以II型GM1神经节苷脂病为例

Lewis, Connor J; Johnston, Jean M; D'Souza, Precilla; Kolstad, Josephine; Zoppo, Christopher; Vardar, Zeynep; Kühn, Anna Luisa; Peker, Ahmet; Rentiya, Zubir S; Yousef, Muhammad H; Gahl, William A; Shazeeb, Mohammed Salman; Tifft, Cynthia J; Acosta, Maria T

AAV9 Gene Therapy in GM1 Gangliosidosis Type II: A Phase 1/2 Trial

AAV9基因疗法治疗GM1神经节苷脂沉积症II型:一项1/2期临床试验

Lewis, Connor J; D'Souza, Precilla; Johnston, Jean M; Acosta, Maria T; Farmer, Cristan; Baker, Eva H; Crowell, Anna; Mojica, Yoliann; Rahman, Sumaiya; Joseph, Lisa; Hartman, Adam; Vézina, Gilbert; Quezado, Zenaide; Yousef, Muhammad H; Luckett, Amelia; Vardar, Zeynep; Shazeeb, Mohammad Salman; Corti, Manuela; Blackwood, Meghan; Coleman, Kirsten; Thurm, Audrey; De Boever, Erika; Gahl, William A; Byrne, Barry J; Flotte, Terence R; Jiang, Xuntian; Gross, Amanda L; Keeler, Allison M; Gray-Edwards, Heather; Martin, Douglas R; Sena-Esteves, Miguel; Tifft, Cynthia J