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

页码：A170 / A171

页数：2

共 30 条

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