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

Optimal murine CD4(+) T cell priming by mRNA-lipid nanoparticle vaccines requires endogenous antigen processing.

mRNA-脂质纳米颗粒疫苗对小鼠CD4(+) T细胞的最佳启动需要内源性抗原加工。

Rood Julia E, Yoon Suh Kyung, Heard Mary K, Carro Stephen D, Hedgepeth Emma J, O'Mara Mary E, Hogan Michael J, Le Nhu, Muramatsu Hiromi, Lam Kieu, Schreiner Petra, Kasden Coral, Stedman Hansell H, Langlois Ryan A, Heyes James, Pardi Norbert, Eisenlohr Laurence C

AAV-microutrophin gene therapy confers durable cardioprotection against pharmacologic and exercise-induced injury in the mdx mouse.

AAV-microutrophin 基因疗法可对 mdx 小鼠的药物和运动引起的损伤提供持久的心脏保护作用。

Kasden Coral M, Corzo Maria P, Greer Christopher D, Singh Tanvi, Reyes Rufranshell, Castillo Alaine, Arany Zoltan, Kozyak Benjamin W, Stedman Hansell H

Confronting complexity in the design and conduct of high-risk rare disease clinical trials

应对高风险罕见病临床试验设计和实施中的复杂性

Stedman, Hansell H

Broader Implications of Progressive Liver Dysfunction and Lethal Sepsis in Two Boys following Systemic High-Dose AAV

两名男孩在接受全身性高剂量AAV治疗后出现进行性肝功能障碍和致命性脓毒症,其更广泛的意义值得关注。

Morales, Leon; Gambhir, Yuva; Bennett, Jean; Stedman, Hansell H

Mechanism of Deletion Removing All Dystrophin Exons in a Canine Model for DMD Implicates Concerted Evolution of X Chromosome Pseudogenes.

犬类 DMD 模型中所有肌营养不良蛋白外显子的删除机制表明 X 染色体假基因的协同进化

VanBelzen D Jake, Malik Alock S, Henthorn Paula S, Kornegay Joe N, Stedman Hansell H

Perspectives on best practices for gene therapy programs

基因治疗项目最佳实践展望

Cheever, Thomas R; Berkley, Dale; Braun, Serge; Brown, Robert H; Byrne, Barry J; Chamberlain, Jeffrey S; Cwik, Valerie; Duan, Dongsheng; Federoff, Howard J; High, Katherine A; Kaspar, Brian K; Klinger, Katherine W; Larkindale, Jane; Lincecum, John; Mavilio, Fulvio; McDonald, Cheryl L; McLaughlin, James; Weiss McLeod, Bonnie; Mendell, Jerry R; Nuckolls, Glen; Stedman, Hansell H; Tagle, Danilo A; Vandenberghe, Luk H; Wang, Hao; Wernett, Pamela J; Wilson, James M; Porter, John D; Gubitz, Amelie K

Cardiac surgical delivery of the sarcoplasmic reticulum calcium ATPase rescues myocytes in ischemic heart failure

通过心脏外科手术输送肌浆网钙ATP酶可挽救缺血性心力衰竭中的心肌细胞

Fargnoli, Anthony S; Katz, Michael G; Yarnall, Charles; Isidro, Alice; Petrov, Michael; Steuerwald, Nury; Ghosh, Sriparna; Richardville, Kyle C; Hillesheim, Richard; Williams, Richard D; Kohlbrenner, Erik; Stedman, Hansell H; Hajjar, Roger J; Bridges, Charles R

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

经外周静脉将腺相关病毒载体递送至骨骼肌,作为治疗B型血友病的新疗法

Arruda, Valder R; Stedman, Hansell H; Haurigot, Virginia; Buchlis, George; Baila, Stefano; Favaro, Patricia; Chen, Yifeng; Franck, Helen G; Zhou, Shangzhen; Wright, J Fraser; Couto, Linda B; Jiang, Haiyan; Pierce, Glenn F; Bellinger, Dwight A; Mingozzi, Federico; Nichols, Timothy C; High, Katherine A

Poster Session PPo4: Epidemiology and Risk Assessment

海报展示环节 PPo4:流行病学和风险评估

Nichols, Timothy C; Whitford, Margaret H; Arruda, Valder R; Stedman, Hansell H; Kay, Mark A; High, Katherine A

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

在大型动物模型中,通过区域性血管内途径向骨骼肌递送AAV-2-F.IX可实现B型血友病的长期矫正。

Arruda, Valder R; Stedman, Hansell H; Nichols, Timothy C; Haskins, Mark E; Nicholson, Matthew; Herzog, Roland W; Couto, Linda B; High, Katherine A