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A missense correcting tRNA therapy platform for genetic diseases

Award Number: R21TR006236
ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 05/01/2026
PERIOD OF PERFORMANCE END DATE: 04/30/2028

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A missense correcting tRNA therapy platform for genetic diseases - Project Abstract Among the thousands of genetic disorders in humans, most are rare and lack effective treatments. Suppressor tRNA therapy has come a long way to become a realistic treatment option for genetic diseases. Sup-tRNAs read through premature stop codons derived from genetic mutations in translation to generate full-length proteins. Compared to gene or mRNA-based therapies that deal with one gene or one mutation at a time and require safety and efficacy assessment for each therapeutic agent, a major advantage of tRNA therapy is the potential of using the same tRNA to treat many diseases that just share a common genetic mutation in many genes. However, about 30% of human genetic disorders are from missense mutations that cannot be treated with sup- tRNAs. For those disorders, missense-correcting tRNAs are needed which are charged with one amino acid but read the codon of another amino acid in translation. PI's lab has developed a missense-correcting tRNA identification platform and applied it to identify such tRNAs charged with Arg but read Gln/His/Trp/Cys codons which correspond to most frequent missense mutations in genetic disorders. Aim 1 will develop a high-throughput engineering platform for missense-correcting tRNAs that will be more efficient, and at the same time, less toxic to cells. We will screen tens of thousands of potential missense-correcting tRNAs simultaneously in cellular models. Aim 2 will screen for missense-correcting tRNA expression with reduced toxicity to cells. Our studies will establish a foundation for using missense-correcting tRNAs as a new therapeutic modality for rare diseases.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $451,000 )
2026	2026	UNIVERSITY OF CHICAGO	5801 S ELLIS AVE	CHICAGO	IL	60637	COOK	USA	National Center for Advancing Translational Sciences	000	1	4/28/2026	NEW	$451,000
														Subtotal = $451,000

Grand Total All Awards = $451,000

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A missense correcting tRNA therapy platform for genetic diseases

Award Number: R21TR006236

ORGANIZATION: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 05/01/2026

PERIOD OF PERFORMANCE END DATE: 04/30/2028

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