Table 1. Comparisons of different... | American Society of Hematology

Table 1.

Comparisons of different lentiviral vectors used for gene therapy of β-thalassemia.

	Transgene	Rise in Hb/Vector Copy	Summary of Findings
May et al²⁸	β-globin	3.8 gm	Correction of anemia in thalassemia intermedia mice
Imren et al²⁹	β⁸⁷-globin	1.5 gm	Correction of anemia in thalassemia intermedia mice
Rivella et al³²	β-globin	2.3 gm	Rescue of lethality in a thalassemia major mouse model
Persons et al³¹	γ-globin	1.1 gm	Correction of anemia in thalassemia intermedia mice
Hanawa et al³³	γ-globin	2.0 gm	Correction of anemia in thalassemia intermedia mice   Reduced position effects
Imren et al⁴²	β⁸⁷-globin	—	High level expression of an antisickling ß⁸⁷ globin in human erythroid cells derived from cord blood progenitors in immune deficient mice   Integration of vector near potential oncogenes
Puthenveetil et al³⁴	β-globin	—	Correction of Human β-thalassemia major phenotype in vitro and in immune deficient mice   Reduced position effects

	Transgene	Rise in Hb/Vector Copy	Summary of Findings
May et al²⁸	β-globin	3.8 gm	Correction of anemia in thalassemia intermedia mice
Imren et al²⁹	β⁸⁷-globin	1.5 gm	Correction of anemia in thalassemia intermedia mice
Rivella et al³²	β-globin	2.3 gm	Rescue of lethality in a thalassemia major mouse model
Persons et al³¹	γ-globin	1.1 gm	Correction of anemia in thalassemia intermedia mice
Hanawa et al³³	γ-globin	2.0 gm	Correction of anemia in thalassemia intermedia mice   Reduced position effects
Imren et al⁴²	β⁸⁷-globin	—	High level expression of an antisickling ß⁸⁷ globin in human erythroid cells derived from cord blood progenitors in immune deficient mice   Integration of vector near potential oncogenes
Puthenveetil et al³⁴	β-globin	—	Correction of Human β-thalassemia major phenotype in vitro and in immune deficient mice   Reduced position effects