UNIVERSITY OF MASSACHUSETTS (20240209376). Specific Oligonucleotide-Programmed Readthrough of Nonsense Codons simplified abstract
Specific Oligonucleotide-Programmed Readthrough of Nonsense Codons
Organization Name
Inventor(s)
Denis Susorov of Worcester MA (US)
Andrei Korostelev of Shrewsbury MA (US)
Seraj Zahra of Worcester MA (US)
Specific Oligonucleotide-Programmed Readthrough of Nonsense Codons - A simplified explanation of the abstract
This abstract first appeared for US patent application 20240209376 titled 'Specific Oligonucleotide-Programmed Readthrough of Nonsense Codons
The invention described in the abstract is related to genetic engineering, specifically focusing on compositions and methods to treat genetically-based diseases and disorders. One example includes nucleic acid oligomers that promote translation readthrough of premature stop codons, leading to the production of functional proteins.
- Nucleic acid oligomers are designed to promote translation readthrough of premature stop codons.
- DNA and modified nucleic acid oligos bind at the +4 through +8 nucleotide positions downstream of a premature stop codon.
- Successful promotion of readthrough of a premature stop codon in a cystic fibrosis gene has been demonstrated.
Potential Applications: - Treatment of genetically-based diseases and disorders - Development of therapies for conditions caused by premature stop codons
Problems Solved: - Addressing the production of non-functional proteins due to premature stop codons - Providing a potential solution for genetic diseases with limited treatment options
Benefits: - Improved translation readthrough of premature stop codons - Potential for developing targeted therapies for specific genetic conditions
Commercial Applications: Title: Genetic Engineering Solutions for Treating Genetic Diseases This technology could be utilized in pharmaceutical companies for developing novel treatments for genetic disorders, potentially leading to new revenue streams and market opportunities in the healthcare industry.
Questions about Genetic Engineering Solutions for Treating Genetic Diseases: 1. How do nucleic acid oligomers promote translation readthrough of premature stop codons? 2. What specific genetic diseases could potentially benefit from this technology?
Original Abstract Submitted
this invention is related to the field of genetic engineering. in particular, it is related to compositions and methods to treat genetically-based diseases and disorders. for example, nucleic acid oligomers are contemplated that promote translation readthrough of premature stop codons that produce non-functional proteins. dna and modified nucleic acid oligos that bind at the +4 through +8 (+4, +5, +6, +7 and +8) nucleotide position downstream of a premature stop codon successfully promoted readthrough of a premature stop codon in a cystic fibrosis gene.
