Muopolysaccharidosis Type I Skeletal Alterations and Cross Correction in an Ex Vivo Gene Therapy Approach

被引：0

作者：

Santi, Ludovica ^{[1
]}

Crippa, Stefania ^{[1
]}

Capo, Valentina ^{[1
]}

Penna, Sara ^{[1
]}

De Ponti, Giada ^{[1
]}

Alberti, Gaia ^{[1
]}

Berti, Margherita ^{[1
]}

Basini, Tommaso ^{[1
]}

Consiglieri, Giulia ^{[2
]}

Tucci, Francesca ^{[2
]}

Riminucci, Mara ^{[3
]}

Corsi, Alessandro ^{[3
]}

Barbero, Andrea ^{[4
]}

Lopa, Silvia ^{[5
]}

Martin, Ivan ^{[4
]}

Moretti, Matteo ^{[5
]}

Gentner, Bernhard ^{[1
]}

Aiuti, Alessandro ^{[1
]}

Villa, Anna ^{[1
]}

Bernardo, Maria Ester ^{[1
]}

机构：

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

[2] Pediat Immunohematol & Bone Marrow Transplantat U, Milan, Italy

[3] Sapienza Univ, Dept Mol Med, Rome, Italy

[4] Tissue Engn Dept Biomed, Basel, Switzerland

[5] Cell & Tissue Engn Lab, Milan, Italy

来源：

MOLECULAR THERAPY | 2023年 / 31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：

1514

引用

页码：712 / 713

页数：2

共 50 条

[41] Consortium nurtures ex vivo gene therapy firm
Laura DeFrancesco
Nature Biotechnology, 2016, 34 : 578 - 578
[42] Consortium nurtures ex vivo gene therapy firm
DeFrancesco, Laura
NATURE BIOTECHNOLOGY, 2016, 34 (06) : 578 - 578
[43] Gene therapy to human diseases: Ex vivo and in vivo studies (Review)
Boulikas, T
INTERNATIONAL JOURNAL OF ONCOLOGY, 1996, 9 (06) : 1239 - 1251
[44] Liver-directed ex vivo gene therapy
Nguyen, T
GENE THERAPY, 2004, 11 : S133 - S133
[45] Ex vivo gene therapy for the treatment of neurological disorders
Gowing, Genevieve
Svendsen, Soshana
Svendsen, Clive N.
FUNCTIONAL NEURAL TRANSPLANTATION IV: TRANSLATION TO CLINICAL APPLICATION, PT A, 2017, 230 : 99 - 132
[46] Targeted ex vivo gene therapy for epidermolytic hyperkeratosis
Chen, I.
Roop, D. R.
JOURNAL OF INVESTIGATIVE DERMATOLOGY, 2009, 129 : S82 - S82
[47] Modeling and Modulating Anti-Transgene Immune Response in Ex-Vivo Gene Therapy for Mucopolysaccharidosis Type-I (MPS-I)
Squeri, Giorgia
Ferro, Francesca
Aiuti, Alessandro
Bernardo, Maria Ester
Gentner, Bernhard
Annoni, Andrea
Biffi, Alessandra
Gregori, Silvia
MOLECULAR THERAPY, 2018, 26 (05) : 456 - 456
[48] AAV Ex Vivo Gene Therapy for Osteogenesis and Chondrogenesis
Mi, Michael Y.
Tang, Ying
Salay, Melessa N.
Li, Guangheng
Huard, Johnny
Fu, Freddie H.
Niyibizi, Chritopher
Wang, Bing
MOLECULAR THERAPY, 2009, 17 : S269 - S270
[49] Mesothelial cells: The panacea for ex vivo gene therapy?
Paillard, F
HUMAN GENE THERAPY, 1997, 8 (16) : 1839 - 1840
[50] Hematopoietic Stem Cell Gene Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal Cross-Correction Mechanisms
Tucci, Francesca
Consiglieri, Giulia
De Pellegrin, Maurizio
Cossutta, Matilde
Filisetti, Chiara
Sarzana, Marina
Scarparo, Stefano
Baldoli, Cristina
Mellone, Renata
Santi, Ludovica
Crippa, Stefania
Parini, Rossella
Aiuti, Alessandro
Bernardo, Maria Ester
MOLECULAR THERAPY, 2024, 32 (04) : 25 - 26

← 1 2 3 4 5 →