Gene therapy has long held promise as a method for targeted alteration of neuronal function in Parkinson's disease. Different gene-therapy approaches aim to correct dysfunctional circuits or have ...

Finally, the sequencing of a fragment of the beta-globin gene was carried out to identify the Hb variant. Figure 1C shows the sequencing electropherograms of the infant (Figure 1C.1), who presented ...

The ABC model is based on the mechanistic notion that an enhancer’s effect on gene expression depends on the intrinsic strength of the enhancer (activity) and the frequency with which it comes into ...

Currently, more than 900 mutations in the beta-globin chain gene have been described. 1 Among them, the mutation leading to the formation of hemoglobin (Hb) S is the most frequent in the general ...

TDT is a severe genetic blood disorder where mutations in the beta-globin gene leads to reduced or absence of hemoglobin, a condition where regular blood transfusion is required to manage anemia and ...

Labie, D. and Elion, J. (1996) Sequence polymorphisms of potential functional relevance in the beta-globin gene locus. Hemoglobin, 20, 85-101. doi10.3109/03630269609027914 ...

Zynteglo, a lentiviral vector-based gene therapy that's created ex vivo using patients' own CD34-positive hematopoietic stem and progenitor cells, is designed to deliver a functional copy of a ...

ZYNTEGLO works by adding functional copies of a modified form of the beta-globin gene (β A-T87Q- globin gene) into a patient’s own hematopoietic (blood) stem and progenitor cells to enable the ...

However, previous studies of mouse skeletal resistance arteries found acetylcholine-stimulated vasodilation to be increased after knockdown of alpha globin gene expression. 63 We demonstrated the ...