Synthetic Dna Construct Encoding Transfer Rna

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

This invention provides a synthetic DNA construct designed to encode a transfer RNA (tRNA), featuring customizable leader sequences that precisely control how much tRNA is produced in cells. By including specific sequence motifs in the DNA's 5' leader region, the construct can adjust the expression level of the engineered tRNA, which can be designed to suppress genetic mutations—such as premature stop codons (nonsense mutations) or frameshifts—thus helping restore full-length, functional proteins in cells affected by genetic disease.

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

• Gene therapy for genetic diseases caused by nonsense (premature stop codon) or frameshift mutations, such as cystic fibrosis, Duchenne muscular dystrophy, Tay-Sachs disease, or Hurler syndrome.
• Development of drugs using engineered tRNA to correct genetic translation errors in patients' cells.
• Research applications for studying tRNA regulation, expression, and function in both normal and disease contexts.
• Production of custom tRNAs in biomanufacturing to enhance yields or properties of recombinant proteins.
• Restoring protein synthesis in diseases caused by tRNA sequestration or functional depletion, such as Charcot-Marie-Tooth (CMT) disease.

BenefitsContent extracted from patent full text and abstract with AI.

• Enables precise control over tRNA expression levels via engineered leader motifs, improving efficacy and safety in gene therapy.
• Allows delivery and production of engineered suppressor tRNAs in patients' cells using established DNA or viral vector platforms.
• Can address a wide range of genetic diseases associated with loss of function from premature stop codons or frameshift mutations.
• Improves stability and delivery efficiency—tRNAs are shorter and more robust than mRNAs, making cellular entry easier.
• Supports structural modifications to tRNA (anticodon, variable loop, T arm, etc.), allowing for broad customization and improved therapeutic potential.

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Patent Abstract

The invention relates to a synthetic DNA construct comprising (A) a nucleic acid encoding a transfer RNA and (B) a 5' leader sequence, the 5' leader sequence comprising one or up to three sequence motifs for controlling the expression level of the transfer RNA.