Alltrna’s Post

Name: Alltrna on LinkedIn: #podcast #rarecast #globalgenes #rna #trna #trnabiology #rarediseases…
Uploaded: 2024-08-12T19:20:48.282Z
Channel: Alltrna

Alltrna

7,007 followers

2mo

Of the ~300 million people around the world with a genetic disease, an estimated 10% have a nonsense mutation that causes a premature stop codon. Our CEO Michelle Werner shares with Daniel Levine how Alltrna is developing tRNA therapeutics as universal medicines for these diseases and why a single medicine has the potential to work across nonsense mutations regardless of the gene in which they occur. In this #podcast episode of #RARECast from #GlobalGenes, hear more about how Alltrna designs their tRNA medicines and why this therapeutic approach has the potential to address so many diseases at once. Listen here or read the interview transcript: https://lnkd.in/gV-CQJYk #RNA #tRNA #tRNAbiology #rarediseases #geneticmedicines #StopCodonDisease #tRNAmedicines #machinelearning #AI

Transcript

So what we think is really exciting about the T RNA is because that function that I just described of a T RNA is the same no matter which protein is being coded for, no matter which gene is affected. And actually it does the same job at each and every one of those those codons. So when you have a nonsense mutation, we actually observe in genetics databases that these same nonsense mutations occur. Across many, many, many different diseases. In fact, we have seen data that says the same nonsense mutations exist across thousands of different diseases. And what we're doing at Alterna is engineering TR Anas that actually can bind and read on these premature termination codons or these nonsense mutations. So as I described earlier, in Nature, there are no T RNA's that know how to bind to these premature termination codons. And that's why that translation process terminates too early because it reads it as a stop instead of an amino acid. But what we're doing is engineering these T RNA's that actually can bind to this premature. Termination code on and instead of actually reading it as a stop, it actually understands what amino acid should have been coded for should the mutation have not had not been there. So as if it it was intended by nature and then it carries on the function that it would normally do. It would be recognized endogenously by the ribosome. It would find that corresponding amino acid within that cellular environment and transfer it to the growing protein chain, thereby it restores. The protein translation process in a very precise way, just as it should have happened, should it it if it had been a healthy cell, and the result is a full length functional protein, which is what typically would be the result if that mutation had not been there in the first.

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Alnylam's RNAi Therapeutics are Disrupting Disease

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