1) How realistic does it seem to give patients asymptomatic variants of dystonia (or comparatively harmless variants such as idiopathic torsion dystonia), for example via CRISPR on the DYT 1 gene? What would this look like? What are the main difficulties to doing this?
It’s very unrealistic in this regulatory climate and with current technology. CRISPR currently presents significant health risks, and I absolutely cannot see giving someone a “comparatively” harmless gene to boost IQ when IQ is a contentious concept and there’s not an equivalent health benefit to offset the health risk. A program of inserting potentially harmful genes to genetically engineer people for higher IQ would frankly make people shit the bed.
Main difficulties: convincingly establish the reality of a causal connection between DYT1 and IQ. Convince funders and researchers to do the work to establish sufficient preclinical data for safety to advance to a stage 1 human trial, convince funders and doctors to run that trial, find a real effect.
I am not overly concerned about regulations. The research can always be done in friendly jurisdictions. I don’t suppose the US would charge people for conducting clinical trials abroad (which are legal in the country where they were performed).
Places I agree with you:
The trials can be risky, and it can be hard (but not impossible) to get approval for one.
It’s likely that the link between DYT1 and IQ doesn’t exist.
It can be almost impossible to get US approval
Places I potentially disagree with you:
I predict that it’s possible to come to a robust theory on how dystonia and IQ is linked prior to experimentation.
I believe that difficulty of US approval is not blocking.
I believe that interested funders definitely exist
I do not anticipate issues with hiring researchers
Edit: I would be interested in how risky we exactly expect the trials to be, and what we can do to reduce this risk.
1) How realistic does it seem to give patients asymptomatic variants of dystonia (or comparatively harmless variants such as idiopathic torsion dystonia), for example via CRISPR on the DYT 1 gene? What would this look like? What are the main difficulties to doing this?
It’s very unrealistic in this regulatory climate and with current technology. CRISPR currently presents significant health risks, and I absolutely cannot see giving someone a “comparatively” harmless gene to boost IQ when IQ is a contentious concept and there’s not an equivalent health benefit to offset the health risk. A program of inserting potentially harmful genes to genetically engineer people for higher IQ would frankly make people shit the bed.
Main difficulties: convincingly establish the reality of a causal connection between DYT1 and IQ. Convince funders and researchers to do the work to establish sufficient preclinical data for safety to advance to a stage 1 human trial, convince funders and doctors to run that trial, find a real effect.
I am not overly concerned about regulations. The research can always be done in friendly jurisdictions. I don’t suppose the US would charge people for conducting clinical trials abroad (which are legal in the country where they were performed).
Places I agree with you:
The trials can be risky, and it can be hard (but not impossible) to get approval for one.
It’s likely that the link between DYT1 and IQ doesn’t exist.
It can be almost impossible to get US approval
Places I potentially disagree with you:
I predict that it’s possible to come to a robust theory on how dystonia and IQ is linked prior to experimentation.
I believe that difficulty of US approval is not blocking.
I believe that interested funders definitely exist
I do not anticipate issues with hiring researchers
Edit: I would be interested in how risky we exactly expect the trials to be, and what we can do to reduce this risk.