[MedicalNews] Correcting the faulty gene
Next, Dr. Huang and team wanted to see whether it was the loss of APOE3 or the accumulation of APOE4 that caused the disease.
So, they compared neurons that did not produce either the E3 or the E4 variant of the protein with cells that had APOE4 added to them.
The former continued to behave normally, while adding APOE4 led to Alzheimer's-like pathologies. This confirmed the fact that it is the presence of the APOE4 that causes the disease.
As a final step, Dr. Huang and his team looked for ways in which to fix the faulty gene. To this end, they applied a previously developed APOE4 "structure corrector."
The so-called structure corrector has been shown in previous research, led by the same Dr. Huang, to change the structure of APOE4 so that it looks and behaves more like the inoffensive APOE3.
Applying this compound to human APOE4 neurons corrected the defects, thereby eliminating signs of the disease, restoring normal cell function, and helping the cells to live longer.
The researchers conclude:
"Treatment of APOE4-expressing neurons with a small-molecule structure corrector ameliorated the detrimental effects, thus showing that correcting the pathogenic conformation of APOE4 is a viable therapeutic approach for APOE4-related [Alzheimer's disease]."
Faster please. |