Pre-clinical development of an ex-vivo lentiviral-based Hematopoietic Stem/Progenitor Cells (HSPC)-gene therapy (GT) approach for Mucopolysaccharidosis type IVA (MPSIVA)

被引：0
|
作者：
Crippa, S. [1 ]
Quaranta, P. [1 ]
Berti, M. [1 ,2 ]
Santi, L. [1 ]
De Ponti, G. [1 ]
Jofra-Hernandez, R. [1 ]
Basso-Ricci, L. [1 ]
Rilievo, A. A. [1 ]
Alberti, G. [1 ]
Forni, C. [4 ]
Visigalli, I. [3 ]
Albertini, P. [3 ]
Paoli, A. [5 ]
Morrone, A. [5 ]
Parini, R. [6 ]
Gentner, B. [1 ]
Scala, S. [1 ]
Aiuti, A. [1 ,2 ,7 ,8 ]
Bernardo, M. E. [1 ,2 ,7 ,8 ]
机构：
[1] San Raffaele Telethon Insitute Gene Therapy HSR T, Milan, Italy
[2] Univ Vita Salute San Raffaele, Milan, Italy
[3] GLP San Raffaele Telethon Inst Gene Therapy HSR T, Milan, Italy
[4] Telethon Fdn, Milan, Italy
[5] Meyer Childrens Hosp, Pediat Neurol Unit, Florence, Italy
[6] San Gerardo Hosp, Rare Metab Dis Unit, Monza, Italy
[7] Ist Sci San Raffaele, Pediat Immunohematol, Milan, Italy
[8] Ist Sci San Raffaele, Bone Marrow Transplantat Unit, Milan, Italy
来源：
HUMAN GENE THERAPY | 2022年 / 33卷 / 23-24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：
P547
引用
收藏
页码：A170 / A171
页数：2
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