scholarly journals Correction of Murine SCID-X1 by Lentiviral Gene Therapy Using a Codon-optimized IL2RG Gene and Minimal Pretransplant Conditioning

2011 ◽  
Vol 19 (10) ◽  
pp. 1867-1877 ◽  
Author(s):  
Marshall W Huston ◽  
Niek P van Til ◽  
Trudi P Visser ◽  
Shazia Arshad ◽  
Martijn H Brugman ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Il2rg Gene ◽  
Lentiviral Gene Therapy

scholarly journals Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease

PEDIATRICS ◽  
2020 ◽  
Vol 146 (Supplement 4) ◽  
pp. S380.2-S381
Author(s):  
Mamatha Mandava ◽  
Kelli W. Williams
Keyword(s):  
Gene Therapy ◽  
Chronic Granulomatous Disease ◽  
Granulomatous Disease ◽  
Lentiviral Gene Therapy

Hemoglobin disorders: lentiviral gene therapy in the starting blocks to enter clinical practice

2018 ◽  
Vol 64 ◽  
pp. 12-32 ◽  
Author(s):  
Karine Sii-Felice ◽  
Marie Giorgi ◽  
Philippe Leboulch ◽  
Emmanuel Payen
Keyword(s):  
Gene Therapy ◽  
Clinical Practice ◽  
Lentiviral Gene Therapy

Phase I study protocol for ex-vivo lentiviral gene therapy for the inherited skin disease, Netherton Syndrome

2013 ◽  
pp. 150127063140004
Author(s):  
Wei-Li Di ◽  
Jemima E Mellerio ◽  
Catina Bernadis ◽  
John Harper ◽  
Alya Abdul-Wahab ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Phase I ◽  
Study Protocol ◽  
Skin Disease ◽  
Phase I Study ◽  
Ex Vivo ◽  
Netherton Syndrome ◽  
Lentiviral Gene Therapy
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