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

Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform

FDA新的合理机制框架对开发个性化体内先导基因编辑平台的影响

Feierman, Emily R; Whittaker, Madelynn N; Quigley, Aidan; Brooks, Dominique L; McVeigh, Paige; Nan, Angela X; Hsu, Alvin; Said, Hooda; Soliman, Ousamah Younoss; Giovenco, Ryan; Davidson, Beverly L; Alameh, Mohamad-Gabriel; Liu, David R; Wang, Xiao; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C

Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing

利用腺嘌呤碱基编辑技术纠正甲基丙二酸血症中的复发性致病变异

Kahn, Elena M; Said, Hooda; Qu, Ping; Alameh, Mohamad-Gabriel; Wang, Xiao; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C

Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum.

针对肝脏的 ABCC6 碱基编辑可防止假性黄色瘤人源化小鼠模型中的异位钙化。

Testa Lauren C, Obiri-Yeboah Dora, Said Hooda, Qu Ping, Levine Michael A, Alameh Mohamad-Gabriel, Musunuru Kiran, Li Qiaoli, Wang Xiao

Systemic mRNA-LNP administration in fetuses improves survival in a mouse model of spinal muscular atrophy.

在脊髓性肌萎缩症小鼠模型中,对胎儿进行全身性 mRNA-LNP 给药可提高存活率。

Carpenter Marco D, Kus Nicole, Choi Rosa B, Said Hooda, Dumitru Ana Maria, Bauer Jackson H, Wang Samantha, Luks Valerie, Teerdhala Sriya, Shah Aarsha, Zoltick Philip W, Alameh Mohamad G, Peranteau William

Pre-Vaccination Immune Profiles and Responsiveness to Innate Stimuli Predict Reactogenicity and Antibody Magnitude Following mRNA Vaccination.

接种疫苗前的免疫特征和对先天刺激的反应性可预测 mRNA 疫苗接种后的反应原性和抗体水平

Zelkoski Amanda E, Goguet Emilie, Samuels Darcey Emily, Alameh Mohamad-Gabriel, Said Hooda, Pollett Simon, Powers John H 3rd, Laing Eric D, Olsen Cara, Mitre Edward, Malloy Allison M W

A base editing strategy using mRNA-LNPs for in vivo correction of the most frequent phenylketonuria variant

利用mRNA-LNP进行碱基编辑的策略,用于体内纠正最常见的苯丙酮尿症变异体

Brooks, Dominique L; Whittaker, Madelynn N; Said, Hooda; Dwivedi, Garima; Qu, Ping; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C; Alameh, Mohamad-Gabriel; Wang, Xiao

Ionization and structural properties of mRNA lipid nanoparticles influence expression in intramuscular and intravascular administration

mRNA脂质纳米粒子的电离和结构特性会影响肌内注射和血管内给药后的表达。

Carrasco, Manuel J; Alishetty, Suman; Alameh, Mohamad-Gabriel; Said, Hooda; Wright, Lacey; Paige, Mikell; Soliman, Ousamah; Weissman, Drew; Cleveland, Thomas E 4th; Grishaev, Alexander; Buschmann, Michael D