Actual Gene Editing in a Patient with Metabolic Disease

[anciendaze]

This story turned up in an unusual place, Wall Street Pit, because of interest by venture capital firms. It states that a patient with an incurable inherited metabolic disease, Hunter syndrome, has been successfully treated using CRISPR gene editing. There is obviously far more to know about this report, but I'm starting this topic to have a place for information on this subject.

For the first time in history, scientists make permanent human genome edits inside a living patient.

 

[Marky90]

Well let`s see if hes cured before calling it a breakthrough? Genetic repair is the future tho

 

[drob31]

I'm curious how it would edit the genes in all 100 trillion cells?

 

[anciendaze]

I'm curious how it would edit the genes in all 100 trillion cells?

If you did that, you would likely change germ-line cells, producing a heritable change. Doing so would cross an ethical boundary, since unborn children could be affected by an experiment performed before they were conceived. This is a much more modest change. You also need to remember that there are hundreds of types of cells, and most genes are only active in a limited number of cells of some types. Simply editing a gene in those cells where it is actively transcribed would result in cures for many conditions.

 

[anciendaze]

Here's a new report about successful treatment of factor VIII hemophilia. It looks like the era of gene editing has arrived before the vast majority of doctors were ready.