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

The curious case of AAV immunology

AAV免疫学的奇特案例

Keeler, Allison M; Zhan, Wei; Ram, Sanjay; Fitzgerald, Katherine A; Gao, Guangping

Efficient Cas9 nuclease-based editing in skeletal muscle via lipid nanoparticle delivery

通过脂质纳米颗粒递送在骨骼肌中高效进行基于Cas9核酸酶的基因编辑

Iyer, Sukanya; Daman, Katelyn; Sun, Yehui; Tutto, Amanda; Holbrook, Sarah E; Joynt, Anya T; Yan, Jing; Ambegaokar, Prajakta; Guo, Dongsheng; Liu, Pengpeng; Stauffer, Jennifer; Maitland, Stacy A; Lee, Sang M; Gallagher, Thomas L; Cox, Gregory A; Keeler, Allison M; Siegwart, Daniel J; Emerson, Charles P; Wolfe, Scot A

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

Immune responses to central nervous system directed adeno-associated virus gene therapy: Does direct CNS delivery make a difference?

针对中枢神经系统靶向腺相关病毒基因治疗的免疫反应:直接递送至中枢神经系统是否会产生影响?

Harkins, Ashley L; Ambegaokar, Prajakta P; Keeler, Allison M

Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy

在非人灵长类动物中,通过肢体灌注递送rAAV1 α-1抗胰蛋白酶载体是安全的,但不足以用于治疗。

Pires-Ferreira, Debora; Reil, Darcy; Tang, Qiushi; Blackwood, Meghan; Gallagher, Thomas; Keeler, Allison M; Chichester, Jessica A; Vyhnal, Kristin K; Lindborg, Jane A; Benson, Janet; Fu, Dongtao; Flotte, Terence R; Gruntman, Alisha M

Immunogenicity of Recombinant Adeno-Associated Virus (AAV) Vectors for Gene Transfer

重组腺相关病毒(AAV)载体的免疫原性

Arjomandnejad, Motahareh; Dasgupta, Ishani; Flotte, Terence R; Keeler, Allison M

CAR-T Regulatory (CAR-Treg) Cells: Engineering and Applications

CAR-T调节细胞(CAR-Treg):工程改造及应用

Arjomandnejad, Motahareh; Kopec, Acadia L; Keeler, Allison M

Regulatory T Cell Therapeutics for Neuroinflammatory Disorders

调节性T细胞疗法在神经炎症性疾病中的应用

Harkins, Ashley L; Kopec, Acadia L; Keeler, Allison M

CRISPR/Cas-Dependent and Nuclease-Free In Vivo Therapeutic Gene Editing

CRISPR/Cas依赖性且无需核酸酶的体内治疗性基因编辑

Dasgupta, Ishani; Flotte, Terence R; Keeler, Allison M