FH consists of two α-globin chains and two γ-globin chains. In utero, FH is the major constituent of fetal RBCs, because β-globin is not expressed until the third trimester. Two nearly ...

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 ...

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

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% ...

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 ...

Next, the investigators queried the identity of the pulled-down DNA by PCR, using primer pairs to amplify short sequences spanning the entire β-globin gene region. The DNA that was pulled down by ...

Ursache ist eine Punktmutation auf dem Chromosom 11: Die hydrophile Aminosäure Glutamin wird durch die hydrophobe Aminosäure Valin an Position 6 der β-Globin-Kette ausgetauscht. Die Folge ist eine ...