Suppressor tRNA in gene therapy

Suppressor tRNAs are engineered or naturally occurring transfer RNA molecules that have shown promise in gene therapy for diseases caused by nonsense mutations, which result in premature termination codons (PTCs) in coding sequence, leading to truncated, often nonfunctional proteins. Suppressor tRNA...

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Bibliographic Details
Published in:Science China. Life sciences 2024-10, Vol.67 (10), p.2120-2131
Main Authors: Ruan, Jingjing, Yu, Xiaoxiao, Xu, Huixia, Cui, Wenrui, Zhang, Kaiye, Liu, Chenyang, Sun, Wenlong, Huang, Xiaodan, An, Lei, Zhang, Yue
Format: Article
Language:English
Subjects:
Animals
Biomedical and Life Sciences
Codon, Nonsense - genetics
Codons
Gene therapy
Genetic Therapy - methods
Humans
Life Sciences
Nonsense mutation
Nucleotide sequence
Protein Biosynthesis
Review
RNA, Transfer - genetics
tRNA
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Summary:Suppressor tRNAs are engineered or naturally occurring transfer RNA molecules that have shown promise in gene therapy for diseases caused by nonsense mutations, which result in premature termination codons (PTCs) in coding sequence, leading to truncated, often nonfunctional proteins. Suppressor tRNAs can recognize and pair with these PTCs, allowing the ribosome to continue translation and produce a full-length protein. This review introduces the mechanism and development of suppressor tRNAs, compares suppressor tRNAs with other readthrough therapies, discusses their potential for clinical therapy, limitations, and obstacles. We also summarize the applications of suppressor tRNAs in both in vitro and in vivo , offering new insights into the research and treatment of nonsense mutation diseases.
ISSN:1674-7305
1869-1889
1869-1889
DOI:10.1007/s11427-024-2613-y