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

Use of plasmapheresis to lower anti-AAV antibodies in nonhuman primates with pre-existing immunity to AAVrh74

利用血浆置换术降低对 AAVrh74 具有预先免疫力的非人灵长类动物体内的抗 AAV 抗体

Potter, Rachael A; Peterson, Ellyn L; Griffin, Danielle; Cooper Olson, Grace; Lewis, Sarah; Cochran, Kyle; Mendell, Jerry R; Rodino-Klapac, Louise R

Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

系统性 γ-肌聚糖 AAV 基因转移可导致 LGMD 2C/R5 小鼠模型中肌肉缺陷的剂量依赖性纠正

Young-Eun Seo, Stephen H Baine, Amber N Kempton, Oliver C Rogers, Sarah Lewis, Kaitlin Adegboye, Alex Haile, Danielle A Griffin, Ellyn L Peterson, Eric R Pozsgai, Rachael A Potter, Louise R Rodino-Klapac

Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

在杜氏肌营养不良症 mdx 小鼠模型中,全身给药 rAAVrh74.MHCK7.micro-dystrophin 的剂量递增研究

Potter, Rachael A; Griffin, Danielle A; Heller, Kristin N; Peterson, Ellyn L; Clark, Emma K; Mendell, Jerry R; Rodino-Klapac, Louise R

Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

腺相关 α-肌聚糖基因转移治疗肢带型肌营养不良症的临床前系统性递送

Danielle A Griffin, Eric R Pozsgai, Kristin N Heller, Rachael A Potter, Ellyn L Peterson, Louise R Rodino-Klapac

Systemic Delivery of Dysferlin Overlap Vectors Provides Long-Term Gene Expression and Functional Improvement for Dysferlinopathy

系统性递送 Dysferlin 重叠载体可实现 Dysferlin 病患者的长期基因表达和功能改善。

Potter, Rachael A; Griffin, Danielle A; Sondergaard, Patricia C; Johnson, Ryan W; Pozsgai, Eric R; Heller, Kristin N; Peterson, Ellyn L; Lehtimäki, Kimmo K; Windish, Hillarie P; Mittal, Plavi J; Albrecht, Douglas E; Mendell, Jerry R; Rodino-Klapac, Louise R

Parenteral nutrition as an unexpected and preventable source of mercury exposure in preterm infants

肠外营养是早产儿汞暴露的一个意想不到且可预防的来源

Jering, Karola; Aschner, Michael; Beller, Amy; Hamm, Ellyn L; Langdon, Margaret; Maitre, Nathalie L

Feeding outcomes and parent perceptions after the pacifier-activated music player with mother's voice trial

使用安抚奶嘴激活音乐播放器并播放母亲语音的试验后,喂养结果和父母的感知

Hamm, Ellyn L; Chorna, Olena D; Stark, Ann R; Maitre, Nathalie L