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Heather Gray-Edwards

u mass chan medical school
H指数:25
被引数:2,191

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Long-term comparative analysis of AAV9-mediated gene replacement therapies for spinal muscular atrophy in mice

AAV9介导的脊髓性肌萎缩症基因替代疗法在 mice 中的长期比较分析

2026-05-230
OAAI
Five-year analysis of efficacy and safety ofa bidirectional AAV gene therapy in Tay-Sachs sheep

Tay-Sachs绵羊中双向AAV基因疗法的疗效与安全性五年分析

2025-12-010
OA
Treatment of a severe vascular disease using a bespoke CRISPR–Cas9 base editor in mice

使用定制的CRISPR–Cas9碱基编辑器治疗小鼠中的严重血管疾病

2025-09-110
PRE
Cerebrospinal Fluid Flow Enhancement (CFE) Increases the Spatial Distribution of Methotrexate after Intracerebroventricular Administration in a Sheep Model

脑脊液流动增强(CFE)可增加鞘内脑室给药后甲氨蝶呤在绵羊模型中的空间分布。

2025-06-100
PRE
Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients (vol 144, 109025, 2025)

II型晚婴期和青少年型GM1神经节苷脂贮积症患者MRI脑容积测量的自然病史进展(卷 144,109025,2025)

2025-05-010
PRE
Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients
2025-03-010
PRE
A minimally invasive endovascular approach to the cerebellopontine angle cistern enables broad CNS biodistribution of scAAV9-CB-GFP

一种针对小脑脑桥角池的微创经血管途径,可实现scAAV9-CB-GFP在CNS中的广泛生物分布。

2024-10-011
OAAI
Life-Limiting Peripheral Organ Dysfunction in Feline Sandhoff Disease Emerges after Effective CNS Gene Therapy
2023-08-163
OA
From mouse to sheep: Generating a sheep model and developing a gene therapy for sialidosis
2023-02-010
PRE
AAV gene therapy for Tay-Sachs disease
2022-02-1067
OA
Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide
2021-12-2399
OA