About Stop Codon Disease скачать в хорошем качестве

About Stop Codon Disease

Across all diseases, 30 million patients can be classified as having Stop Codon Disease. In 10 percent of genetically driven diseases, both rare and common, a single base pair of DNA is mutated, changing the code for an amino acid to a stop signal and causing a truncated or absent protein. These types of mutations, called nonsense mutations, can happen anywhere in the genome. If it occurs in the gene for dystrophin, it can cause Duchenne muscular dystrophy, but if it occurs in the gene for Factor VIII, it can cause hemophilia. As a result, the same exact mutation can be the cause of multiple diverse diseases. Alltrna is developing engineered tRNA medicines to readthrough premature termination codons and restore production of affected proteins, no matter what protein is affected. A tRNA medicine that could universally treat patients across multiple indications in Stop Codon Disease could accelerate and scale the development of potential disease-modifying medicines for millions of patients, in a way that no other genetic medicine can.