[chaostheory]

It’s good news, but it doesn’t mean that there still aren’t more challenges to overcome.

I think the article ends on a really important note:

“The increasing cost of manufacturing these treatments makes it extremely difficult, if not impossible, to develop and test gene therapy for many ultrarare diseases where the number of patients worldwide is very small and profitability low.

We were able to deliver these treatments to the children in our ongoing clinical trials thanks only to funding from a generous family whose own child is a participant”

[monkeycantype]

I'm not an expert, but I've started a biomedical science degree, and - struggling for the right word here - the 'systemisation' of the tools for genetic engineering is astonishing. There's a revolution happening here, not just in the cutting edge tools, but also in the streamlining and automation of workflow, I find it hard to believe custom genetic treatments are going to stay expensive for long

[ahevia]

Can you share where the uneducated can learn more about these advancements?

I’ve always had a passing interest in this subject. Will be cool to know how folks are systemizing this field

[monkeycantype]

I'm one of the uneducated and I enrolled in a biomedical science degree.

As I said i'm not an expert, just a first year student getting my mind blown.

But in my classes, we look at catalogs of genetic vectors:

here's a top google hit https://en.vectorbuilder.com/

These vectors contain a number of indicator genes, such as antibiotic resistance, so you can use the antibiotic to kill the bacteria that didn't take up your vector.

The vectors have a prepared insertion site to take your gene, that's right in the middle of another gene that produces a colored product.

If the your resultant bacteria produces the colored product, you know you gene didn't make it into the insertion site or it would have broken that gene.

There's vast catalogues of this stuff.

Then when you want to do full genetic sequence to see where your gene has inserted, that's pretty much automated for you.

https://www.thermofisher.com/au/en/home/life-science/sequenc...

You want to compare the genetic sequence to other organisms, there are online search engines for that:

https://blast.ncbi.nlm.nih.gov/Blast.cgi

If your experience is anything like mine, once you start searching for this, google ads is going to lure you into the rabbit hole with offers to 'automate your agrigenomic high throughput whole sequence workflow' and 'rapid de novo genome assembly'

Some of the stuff google is trying to sell me, seems only slightly more non-fictional than 'mystery flesh pit national park', which is of course a searchable phrase.

[mountainriver]

This is what I was hoping to hear, commoditizing all of the tools would be a huge benefit, I guess on the flip side is could make bioengineering of deadly things easier?

[jostmey]

Many exciting new biotechnologies are incredibly expensive like gene therapy, T cell therapy, or some other radical life-saving therapy. Regulations are one factor driving up costs, as well as the manufacturing costs. It is not easy and really annoying when people think it is overpriced

[DoreenMichele]

When you compare such to current costs of conventional treatment, it's often not that bad. That's before you factor in pain, suffering and lost productivity of the patient and their caregivers.

The problem is figuring out how to pay for such things to begin with.