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

Lentiviral vectors for hematopoietic stem cell gene therapy restore α-globin expression in α-thalassemia red blood cells

用于造血干细胞基因治疗的慢病毒载体可恢复α-地中海贫血红细胞中α-珠蛋白的表达

Eva E R Segura ,Kevyn Hart ,Beatriz Campo Fernandez ,Devin Brown ,Kevin Tam ,Andrea Gutierrez Garcia ,Eva Seigneurbieux ,Karen Li ,Carol Mulumba ,Emma Blakely ,Katelyn Masiuk ,Roshani Sinha ,Devesh Sharma ,John Everett ,Matthew Hogenauer ,M Kyle Cromer ,Frederic Bushman ,Tippi C MacKenzie ,Donald B Kohn

Effective gene therapy for metachromatic leukodystrophy achieved with minimal lentiviral genomic integrations

利用最小的慢病毒基因组整合实现对异染性脑白质营养不良的有效基因治疗

Lucas Tricoli ,Sunetra Sase ,Julia L Hacker ,Vi Pham ,Maxwell Chappell ,Laura Breda ,Stephanie N Hurwitz ,Naoto Tanaka ,Carlo Castruccio Castracani ,Amaliris Guerra ,Zhongqi Hou ,Lars Schlotawa ,Karthikeyan Radhakrishnan ,Matthew Hogenauer ,Aoife Roche ,John Everett ,Frederic Bushman ,Peter Kurre ,Rebecca Ahrens-Nicklas ,Laura A Adang ,Adeline L Vanderver ,Stefano Rivella