The discovery of the DNA first and the sequencing of the human genome have created huge expectation for a future where it would be possible to radically cure/stop genetic diseases (and there are over 6,000 genetic disorders already identified).
Clearly the first step is to understand what might be wrong in the genes but this is not going to help unless there is a way to patch up the faulty genes.
A real turning point was the discovery of CRISPR / Cas9 and of methods and technology to use it. With CRISPR/Cas9 it has become possible to cut undesired parts of a gene and splice other parts together. Of course one thing is to be able to do this, quite a different story is to reach a point where you are sure you are actually doing what you want to do.
The problem, in biology, is that you are not working with a few cogs and wheels that you can identify, select and manipulate. You are dealing with millions / billions of molecules and you are playing some sort of statistical game. Sometimes this is ok, sometimes it is not and most of the times we are not sure what is the case.
Think about it: by using CRISPR/Cas9 on a single cell in the lab (in a Petri dish) you can see what is happening, but if you are “injecting” in a body the CRISPR/Cas 9 this is going to affect hundreds of thousands, millions of cells and you can’t be sure what is going on in each single one (most likely something slightly different in different cells). If, statistically speaking, the majority of them aree affected in the way you are expecting you should get a good result. On the other hand there is always the -remote- possibility that some of the changes may go awry and this might create problem. Again, if statistically the outcome is by far better than the option of don’t doing anything (very low risk of side effects) it makes sense to do that. This is what we call “medicine”.
In 2020 we had the first use of CRISPR on a person affected by sickle cell anemia, a genetic malfunction, and after a year she is doing extremely well. The technology used is thee one provided by Crispr Therapeutics. We can expect a growing usage of CRISPR in clinical medicine in the coming years. However, as mentioned, CRISPR is ok for deleting part of a gene with a (statistically) quite good accuracy but it is not that good when the goal is to create new sequences of DNA, new genes.
Now a new start up, Tessera Therapeutics, – watch the clip- has developed a new technology promising accurate deletion, like CRISPR based ones, as well as insertion of a single base pair (this is very interesting since a significant number of genetic malfunctions are caused by a single base pair alteration) and long stretches of DNA. If consistent high accuracy can be reached the cure by genomic alteration (repair) can speed up. The twenties, our present decade, where called in the previous decade as the age of genome, brain and smart materials. It seems that the expectation we had in the past decade are going to be fulfilled, although we should probably wait for the next decade, the thirties, to see their massive deployment. Of the three the smart material expectation is the one that is most advanced (particularly the design of new alloys and the embedding on IoT in materials), followed by the genome and the brain. This latter will probably take longer to become a game changer.
Interestingly, the engine powering all this progress is found in data and artificial intelligence. Without them progress would not be possible.