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

Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries

欧洲及邻国异染性脑白质营养不良症造血干细胞移植现状调查

Schoenmakers, Daphne H; Mochel, Fanny; Adang, Laura A; Boelens, Jaap-Jan; Calbi, Valeria; Eklund, Erik A; Grønborg, Sabine W; Fumagalli, Francesca; Groeschel, Samuel; Lindemans, Caroline; Sevin, Caroline; Schöls, Ludger; Ram, Dipak; Zerem, Ayelet; Graessner, Holm; Wolf, Nicole I

Dose-response evaluation of intravenous gene therapy in a symptomatic mouse model of metachromatic leukodystrophy

异染性脑白质营养不良症症状小鼠模型中静脉基因治疗的剂量反应评估

Emilie Audouard, Nicolas Khefif, Charlotte Mansat, Océane Nelcha, Elena-Gaia Banchi, Camille Lupiet, Dominique Farabos, Antonin Lamaziere, Caroline Sevin, Françoise Piguet

Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

完全纠正异染性脑白质营养不良小鼠的大脑和脊髓病理

Emilie Audouard, Valentin Oger, Béatrix Meha, Nathalie Cartier, Caroline Sevin, Françoise Piguet

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice

AAVrh.10 脑内基因治疗对异染性脑白质营养不良小鼠脑少突胶质细胞的矫正

Françoise Piguet, Dolan Sondhi, Monique Piraud, Françoise Fouquet, Neil R Hackett, Ornella Ahouansou, Marie-Thérèse Vanier, Ivan Bieche, Patrick Aubourg, Ronald G Crystal, Nathalie Cartier, Caroline Sevin