Professional Documents
Culture Documents
Recent Article on
Genetics in
Biologic and
Clinical Efficacy
of LentiGlobin
for Sickle Cell
Disease
Background
Sickle cell disease is characterized
by the painful recurrence of vaso-
occlusive events. Gene therapy with
the use of LentiGlobin for sickle cell
disease (bb1111:lovotibeglolene
autotemcel)consist of autologous
transplantation of hematopoietic
stem and progenitor cells
transduced with the BB305 lentiviral
vector encoding a modified B-globin
gene, which produces an antisickling
hemoglobin, HbAT87Q