AAV Intein Retinal Gene Therapy for Stargardt Disease is Effective and Safe in Large Animal Models

被引：0

作者：

Trapani, Ivana ^{[1
,2
]}

Ferla, Rita ^{[3
]}

Pugni, Eugenio ^{[1
]}

Lupo, Mariangela ^{[1
]}

Perota, Andrea ^{[4
]}

Gesualdo, Carlo ^{[5
]}

Rossi, Settimio ^{[5
]}

Simonelli, Francesca ^{[5
]}

Bacci, Maria Laura ^{[6
]}

Galli, Cesare ^{[4
]}

Memon, Ali ^{[3
]}

Shams, Naveed ^{[3
]}

Auricchio, Alberto ^{[1
,2
,3
]}

机构：

[1] Telethon Inst Genet & Med, Pozzuoli, Italy

[2] Univ Naples Federico II, Dept Adv Biomed Sci, Naples, Italy

[3] AAVantgarde Bio Srl, Milan, Italy

[4] Avantea, Lab Reprod Technol, Cremona, Italy

[5] Univ Campania Luigi Vanvitelli, Multidisciplinary Dept Med Surg & Dent Sci, Eye Clin, Naples, Italy

[6] Univ Bologna, Dept Vet Med Sci, Ozzano Dellemilia, Italy

来源：

MOLECULAR THERAPY | 2024年 / 32卷 / 04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：

297

引用

页码：158 / 159

页数：2

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