Baby is healed with first personalized gene-editing treatment

It sounds like Spiderman tech.

Bear in mind that they intentionally choose something that was soluble - ie the easiest thing possible. So it's doesn't mean everything is now solvable.

For example it's no coincidence this is a liver disease as basically almost everything you inject in the bloodstream ends up concentrating in the liver by default - if you needed to target another organ with your LNP it would be much harder. Most of the time people are trying to stop stuff accumulating in the liver!

The liver has other special properties that are helpful as well.

Having said all that - it is still a massive achievement.

> That is one of the most incredible things I have ever read.

Biology is incredible - and you can do incredible things if you leverage it.

We don't get 50/50 of distinct genes from our parents - it is more like 30/70 and can be even 10/90. The whole DNA ratio in this equation is irrelevant, as we all have 99% of the same DNA. Also, in real world, one parent will consistently give more of their distinct genes than other parent and most likely that consistent gene part will have that single mutation that they would hope to avoid, but contain best genes that the parent can offer. Children from multiple partners could be a solution as it is a different math...

>>>There are also medical procedures (PGD) to bring that chance to virtually 0%. For that one gene only. DNA is a math of sum of genes and from what I have read humans are not better than nature(which is not perfect, but very basic) at selecting best specimens of eggs and sperm, but yes - whatever they have picked - PGD might be able to root out that one single mutation, and introduce variety of other mutations or miss good genes from other combinations. So, it all depends...

I feel a bit proud that humanity healed a baby with this tech before any viruses were constructed/released.

Here's the actual paper

https://www.nejm.org/doi/full/10.1056/NEJMoa2504747

This site has a much better write up than the NYT:

https://innovativegenomics.org/news/first-patient-treated-wi...

Under normal circumstances, developing and testing a new CRISPR therapy takes years, but this patient — and others born every day with severe genetic disorders — do not have years to wait. Getting all of the pieces together for this emergency need took rapid coordination amongst teams at multiple academic and for-profit organizations, only possible because of both years of preparation and some lucky connections.

...

Prior to receiving the CRISPR therapy, KJ’s prognosis was poor, but there were several factors that made KJ’s case a good candidate for a rapid CRISPR intervention. An ongoing research study at the IGI called INGENUITI enabled the team to immediately enroll KJ and his parents for genome sequencing and analysis. The mutation in KJ’s genome was a single base — just a one-letter change in his genetic code — and one that could be corrected using base editing, a gene-editing technique that only makes a single-letter edit. Additionally, the researchers needed to edit cells in the liver, where the faulty protein is made. The liver is currently one of the organs in the body that can be targeted for in vivo gene-editing therapies using lipid nanoparticles.

...

One of the first steps involved characterizing the mutation in KJ’s genome and designing the guide RNAs that allow the base editor to precisely target a specific letter in the patient’s genome for repair. Kiran Musunuru’s team at Penn accomplished that in record time. Next, the team at the IGI jumped in to do the necessary safety assessment work so that the FDA could assess the risks.