. Author manuscript; available in PMC: 2017 Dec 6.

Published in final edited form as: Adv Exp Med Biol. 2017;1013:155–176. doi: 10.1007/978-1-4939-7299-9_6

Table 6.1.

Beta-globin vectors

Name	Author and year	Main characteristics
TNS9	May 2000 Rivella 2003	- Correction of a mouse model of thalassemia intermedia (2000) and prevention of lethality in a mouse model of thalassemia major (2003) - Variable expression
β^A(T87Q)	Pawliuk 2001	- Correction of two SCA mouse models - Anti-sickling (T87Q) form of beta-globin
BG-1	Puthenveetil 2004	- Full-length cHS4 enhancer used in 3′LTR (1.2 kb) - Amount of beta-globin approached normal levels - Low viral titers
T10	Lisowski 2007	- Addition of HS1 to HS2-4
Globe	Miccio 2008	- Higher titer with removal of HS4
β^A(T87Q) LentiGlobin	Cavazzana-Calvo 2010	- Used in first European clinical trial to achieve transfusion independence in a β⁰/β^E heterozygous patient - Transgenic β^(T87Q)-globin made up only 1/3 of total Hb, rest combination of HbE/HbF - Integration sites near potential oncogenes - Saw expansion of one clone with a HMGA2 integration site
G-Globe	Miccio 2011	- No HS4, incorporates HS2 enhancer of the GATA1 gene
AnkT9W T9AnkW	Breda 2012	- Incorporates the Ankyrin insulator - Shows improved β-globin expression over TNS9
CCL-βAS3-FB	Romero 2013	- “FB” insulator containing the minimal 77 bp binding site for CTCF - 3 mutations to beta-globin to confer anti-sickling properties