Synthetic Transfer Rna with Extended Anticodon Loop

Simple SummaryContent extracted from patent full text and abstract with AI.

This invention describes synthetic transfer RNA (tRNA) molecules engineered with an extended anticodon loop that can recognize and bind to four or five consecutive nucleotides, instead of the usual three. These synthetic tRNAs are designed to suppress frameshift mutations in messenger RNA (mRNA), which can occur due to genetic disruptions such as insertions or deletions, and thus restore proper protein synthesis. This approach can address genetic diseases caused by such mutations, such as neurofibromatosis, Duchenne muscular dystrophy, cystic fibrosis, and others.

Use CasesContent extracted from patent full text and abstract with AI.

• Gene therapy for genetic diseases with frameshift mutations (e.g., neurofibromatosis type 1, Duchenne muscular dystrophy, Crohn's disease, cystic fibrosis, Tay-Sachs disease, and neuronal ceroid lipofuscinosis).
• Suppression and correction of frameshift mutations in living cells to restore the production of functional proteins.
• Development of personalized medicines targeting patient-specific mutations.
• Research tool in molecular biology for studying translational fidelity, frameshifting, and non-canonical decoding.
• Potential use in synthetic biology for expanding or editing the genetic code.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides a method to suppress both +1 and -1 frameshift mutations with high specificity, thus potentially restoring function to proteins that would otherwise be non-functional.
• Higher specificity in binding to target mRNA sequences, reducing off-target effects and mispairings compared to prior technologies.
• Synthetic tRNA can be made more stable than natural or previously modified tRNAs, offering improved durability and delivery.
• Enables therapy at the mRNA translation level without altering the underlying DNA, which reduces concerns about permanent genetic changes.
• Versatile application for a range of genetic diseases associated with frameshift mutations.

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Patent Abstract

The invention relates to a synthetic transfer RNA with an extended anticodon loop. The invention provides a synthetic suppressor transfer RNA useful for the treatment of a genetic disease like neurofibromatosis associated with a frameshift mutation. The synthetic transfer RNA comprises an extended anticodon loop having a four-nucleotide anticodon or a five- nucleotide anticodon.