I've been speaking with gene therapy researchers, and they do believe that NGLY1 is a good candidate for gene therapy because (1) NGLY1 is a relatively small gene and (2) we're pretty good at transfecting into the critical organs for NGLY1 (like the liver).
Of the treatment options we're researching right now, gene therapy is #3 on my priority list.
One of the reasons I'm bullish on gene therapy and related techniques is that it's one of the few approaches that could cure the many of the rare diseases out there today.
https://en.wikipedia.org/wiki/Gene_therapy
I've been speaking with gene therapy researchers, and they do believe that NGLY1 is a good candidate for gene therapy because (1) NGLY1 is a relatively small gene and (2) we're pretty good at transfecting into the critical organs for NGLY1 (like the liver).
Of the treatment options we're researching right now, gene therapy is #3 on my priority list.
One of the reasons I'm bullish on gene therapy and related techniques is that it's one of the few approaches that could cure the many of the rare diseases out there today.