[godmode2019]

Can someone explain who this works?

""" I am a member of a team of researchers from UMass Chan Medical School and Auburn University who developed a gene therapy that may help get around this barrier. Our treatment uses two harmless viral vectors to deliver DNA instructions to brain cells that teach them how to produce the missing enzyme. Similar techniques have been used to treat a number of related diseases and other conditions. In the case of Tay-Sachs, these DNA instructions enter the nucleus of these cells and stay there, allowing for long-term production of HexA. """

They use a monkey virus to deliver two mRNA codes to brain cells, the mRNA after being transcribed, tells the brain cells to create HexA to deal with the target enzyme.

Okay, I get that much.

But how on earth does this work.

""" these DNA instructions enter the nucleus of these cells and stay there """

I thought mRNA could not alter DNA and it was by definition broken down after use by the cell, ie short term by definition?

[JunkDNA]

They’re not using mRNA. They are using a viral vector that contains DNA.

[dahfizz]

Plenty of viruses use DNA and not RNA. The virus injects it's DNA sequence into the host DNA.

This is why people have "flare ups" of herpes/cold sores. That is a DNA virus, so once the cells are infected they carry that DNA sequence forever, even after the cells divide.