Exploiting chimeric GALC enzymes with improved bioavailability to refine gene therapy approaches for globoid cell leukodystrophy

被引：0

作者：

Cascino, F. ^{[1
]}

Ricca, A. ^{[1
]}

Picciotti, I. ^{[1
,2
]}

Morena, F. ^{[3
]}

Martino, S. ^{[3
]}

Gritti, A. ^{[1
]}

机构：

[1] San Raffaele Telethon Inst Gene Therapy HSR TIGET, Milan, Italy

[2] Univ Vita Salute San Raffaele, Milan, MI, Italy

[3] Univ Perugia, Dept Chem Biol & Biotechnol, Perugia, Italy

来源：

HUMAN GENE THERAPY | 2024年 / 35卷 / 3-4期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

P580

引用

页码：A248 / A248

页数：1

共 24 条

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[24] Bone Marrow Transplantation Augments the Effect of Brain- and Spinal Cord-Directed Adeno-Associated Virus 2/5 Gene Therapy by Altering Inflammation in the Murine Model of Globoid-Cell Leukodystrophy
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