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Lecture 13, concept 09: Computational design of new protein folds

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The lecture introduces the cutting-edge field of computational protein design, moving beyond the traditional approach of stabilizing new sequences within existing natural folds. Instead, researchers aim to create entirely novel protein structures that have never been observed in nature. Since bioinformatics alone cannot predict these unknown forms, scientists must rely on physical laws and computational tools like the Rosetta program to assemble small structural fragments into a new fold. This process involves iteratively placing side chains and running simulations to enhance stability, followed by rigorous laboratory testing to validate successful designs before further modifications are made. A significant advancement in this field comes from David Baker's group, which has pioneered the creation of custom protein building blocks designed to self-assemble into larger, periodic structures resembling crystals. By combining small helices and sheets with disulfide bridges and chemical treatments, they create rigid components that can spontaneously aggregate into miniature machines on a nanometer scale. This approach represents a form of biological nanotechnology, operating at scales far smaller than traditional mechanical nanotechnology, and holds the potential for future applications in bacteria and other advanced biotechnological fields. The lecture also highlights the successful design of beta-barrel membrane proteins, which are notoriously difficult to fold due to their delicate stability and complex insertion mechanisms into cell membranes. Designing these proteins requires not just constructing a stable structure but also performing "anti-design" to ensure the protein does not form its final shape prematurely in water. By carefully tuning the hydrophobicity of the pore and reducing the tendency to form beta sheets outside the membrane, researchers achieved a state where the structure only folds correctly upon inserting into the lipid bilayer. Ultimately, these breakthroughs demonstrate that computational design can overcome the limitations of natural evolution to create functional proteins with unprecedented properties. The ability to engineer completely new folds, from rigid crystalline arrays to specialized membrane channels, opens up limitless possibilities for synthetic biology and medicine. As research progresses, we are likely to see the emergence of miniature biological machines that perform tasks far beyond current capabilities, marking a transformative era in protein science where human design dictates the architecture of life's fundamental machinery.
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so that's the last thing we do i want to show you something that's really state of the art today most of the previous proteins design i've showed you have the fact in common that they kind of piggybacked on existing folds and i just try to stabilize a new sequence in that fold that is smart and it's in general what you want to do don't reinvent the wheel but what if we could create completely new faults remember this landscape that i spoke about in the bioinformatics lecture that we know most of the existing fold space but instead of this base what if i could design a protein out here it's a completely new fold a fold that we've never seen in nature before in this case there is no way we could rely on plain bioinformatics it's not going to work we don't have anything like it this is something that i need to stabilize based on the laws of physics basically there is one group in the world that i would argue significantly further ahead than anybody else here and that's david baker in washington the groups that are behind this rosetta program and what they do in most of this case is roughly the same thing as for rosetta but you can't piggyback as much unknown structures you're assembling small fragments in this case we try to assemble small fragments into a new fold that we've either guessed or have divine inspiration or try to optimize what it should look like and then we're iteratively placing side chains see if we can improve the stability moving the side chains around maybe running a short simulation to see if we can stabilize things and then we need to go in the lab test this take back the successful cases try to modify the successful cases a bit there's an insane amount of work here but there is also an exceptionally impressive stream of new papers coming out from where they showed that they can't do this they can't design vaccines they can design proteins and they can design completely new folds for proteins this is david one thing that they did just two years ago i think it was that they instead of building traditional proteins they figured out what if we could design some sort of large building blocks almost like the fibrous proteins we talked about earlier in this class now we can't have very long genes so that their idea is that what if we start by creating some small bricks and what they did here is that they usually use small helix and sheet combinations two three secondary structure elements then they would use several disulfide bridges so cysteines that would mean that i'm going to lock them up with disulfides create very nice small rigid components and for a few of these they also used chemical treatment afterwards to try to get the n and the c terminal to stick together again the reason for doing this is creating small building blocks where each building block will be very rigid and stick together once you've done that you have your small bricks and if these bricks now stick together we can start to change the amino acids on the surface of the brick so if i have two bricks here what if i create a periodic pattern here so that the bricks would like to pack then the bricks might spontaneously self-aggregate into larger structures and do they this is a shock and all paper too that i have for you in canvas they had a whole range of structure where they can create almost crystals formed out of proteins with repeating patterns because again each building block here is small but the patterns they create and not quite with macroscopic scales but almost the use cases for this is unlimited you can imagine using this in bacteria we might create those small miniature structures and everything the reason why i don't have any super specific examples for you yet is that it's still research of course but we're literally creating a type of protein structure engineering that is like nanotechnology the only difference is that traditional mechanics-based nanotechnology that has structures in the ballpark of 900 nanometers these proteins they have structures in the ballpark of 10 nanometers so that it's almost on the border pico technology not quite but far smaller structures that can do far more advanced things and i bet we're going to start to see miniature machines here in the near future too when i introduce the membrane protein class to you i figure that there's only one class of proteins that is not so common the beta barrel proteins and membranes so maybe i could let go of those chance has a way of fun playing funny tricks at you because because i did that just two weeks later after that lecture of course david baker published an amazing paper in nature where they showed that they could design beta barrels so now i have to share beta barrels with you anyway so beta barrels are not common membrane proteins but they do exist and when they fold they kind of they form some sort of semi-beta heat like structure on the outside and then they gradually slide into the membrane by themselves we know very little about their folding we also know that their delicate stability and everything so this is probably some of the hardest proteins imaginable to fold but david managed to do that this is also a paper i've shared you because it's a remarkable example how to use protein design in practice and designing completely new beta-barrel proteins so what did they do well first they used all these rules that we've gone through having anchors on the top of the membrane proteins to anchor say tyrosines to the head group regions here you need a hydrophobic exterior of the pore and then a moderately hydrophilic interior of the pore so literally have a poor functionality to go through it and then carefully select the number of beta strands we have to form a large beta sheet but it turns out that's not enough because if you do this and create something that is a very stable beta sheet it's not going to work at all the reason for that is that if something is too stable as a beta sheet they will just form a beta sheet out in the water and if it's stable as a beta sheath out in the water it's no particular reason for it to go into the membrane so the challenge here is not just design but for proteins you also need to do anti-design so the greatest challenge for them here was actually not to design a beta sheet anybody me included could do that the hard part is that they had to reduce the property of this protein to form a beta sheet out in water and when they reduced this property sufficiently enough and made it relatively hydrophobic then you got it to a state where it would not form beta sheets out in water but it will form beta sheets while it is inserting into the membrane and then they managed to show that it doesn't hurt so these actually do form stable new folds of beta barrel membrane proteins