[qnleigh]

What enabled this treatment to be used now? Gene editing techniques have existed for a long time, but there were many reasons why they weren't being used in humans, like concerns about off-target edits and heritability. The article mentions something about gold nanoparticles, but this aspect was developed over the course of a few weeks, and in any case these aren't new either.

[directevolve]

Well, CRISPR-Cas9 as a tool for genetic engineering was only invented in 2012. A 13-year translation timeline is not unusual, maybe even unusually fast. CAR-T cell therapy for cancer took 30 years from discovery to clinic. It took about 30 years to go from the early attempts to use engineered lipid nano particles for drug delivery to the first FDA-approved medication using them, doxil.

With CRISPR, it took a long time to figure out how to reliably edit just the gene you want and acceptably minimize off-target edits, including by delivering the therapeutic to just the organ affected and getting the dose and release right.

The public is understandably leery about experimental medical techniques. If they had killed this newborn child with CRISPR therapy, then it might have set created a backlash delaying translation of this technique for years, possibly decades.

In biomedicine, we’re always looking for therapies that approximate the level of precision control available in software. Unfortunately, it’s never more than an approximation, and our ability to measure and predict the size of that error is always limited. That is why the field moves slowly.

[hirsin]

Expanding the timeline a bit, CRISPR was known as a possible gene targeting/editing tool by 2008 at least - I distinctly recall learning about it then in a guest lecture.

[qnleigh]

I mean to ask 'why now?' not 'what took so long?' What about the regulations or the science let this happen now, and not 5 years ago or 5 years into the future?

Also do we know that this was CRISPR?

[rolph]

Yes, This was a CRISPER technique.

https://www.chop.edu/news/worlds-first-patient-treated-perso...

[directevolve]

Yes, CRISPR’s mentioned in the article. Someone should write a book about it to break down the history leading up to this accomplishment.