LentiGlobin BB305 Lentiviral Vector: LentiGlobin BB305 LVV is a replication defective, self-inactivating HIV-1 based vector pseudotyped with the vesicular stomatitis virus G envelope protein, carrying ...

To improve the efficacy of gene therapy when using anti-sickling beta globin gene transfer, they added cyclosporin (CsH) to increase transduction by inhibiting lentiviral restriction factors.

The team attempted to amplify the same fragment that Mullis targeted—a 110 base pair segment from the human β-globin gene. At first, they could not determine if they achieved specific target ...

Two nearly identical γ-globin genes are located next to the β-globin gene within the β-globin cluster. By birth, γ-globin production is normally switched off, but a significant fraction of ...

First up, the lentiviral vector that delivers the gene seems to be working as expected, with expression of the replacement β-globin gene HbAT87Q present at therapeutic levels (44% to 59% ...

Thalassemia and sickle cell anaemia are common genetic disorders in India. Early testing, prenatal screening, and improved ...

ARU-1801 leverages a proprietary lentiviral vector to deliver a gene encoding a modified γ-globin, called γ(G16D), into a patient’s own stem cells ex vivo. γ(G16D) was designed to have a ...

The following is a summary of “Evaluated NSUN3 in reticulocytes from HbH-CS disease that reflects cellular stress in ...

Beta Thalassemia occurs due to a mutation in the beta globin gene, which is essential for production of normal haemoglobin, the protein inside our red blood cells which carries oxygen from the ...