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The large ribosomal subunit has several important structures essential to translation. These include the peptidyl transferase center (PTC) - which is the site where the peptide bond is formed - and a large, internal, water-filled tube through which the nascent polypeptide moves. This latter structure is called the Peptide Exit Tunnel, and it begins at the PTC and spans the body of the large ribosomal subunit. During translation, as the nascent polypeptide chain is synthesized, it passes through...
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PIWI-interacting RNAs, or piRNAs, are the most abundant short non-coding RNAs. More than 20,000 genes have been found in humans that code for piRNAs while only 2000 genes have been found for miRNAs. piRNAs can act at the transcriptional and post-transcriptional levels and have a vital role in silencing transposable elements present in germ cells. They are also involved in epigenetic silencing and activation. Previously, they were thought to function only in germ cells but new evidence suggests...
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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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In humans, more than 80% of the genome gets transcribed. However, only around 2% of the genome codes for proteins. The remaining part produces non-coding RNAs which includes ribosomal RNAs, transfer RNAs, telomerase RNAs, and regulatory RNAs, among other types. A large number of regulatory non-coding RNAs have been classified into two groups depending upon their length – small non-coding RNAs, such as microRNA, which are less than 200 nucleotides in length, and long non-coding RNA...
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Author Spotlight: AQRNA-seq Role in Mapping Small RNAs and Unraveling Protein Translation Mechanisms
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Engineered transfer RNAs for suppression of premature termination codons.

John D Lueck1, Jae Seok Yoon2, Alfredo Perales-Puchalt3

  • 1Department of Physiology and Pharmacology, University of Rochester School of Medicine and Dentistry, Rochester, NY, 14642, USA. john_lueck@urmc.rochester.edu.

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|February 20, 2019
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Engineered transfer RNAs (ACE-tRNAs) effectively suppress premature termination codons (PTCs) that cause genetic diseases. This novel approach shows high potential for treating inherited disorders by restoring protein production.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biochemistry

Background:

  • Premature termination codons (PTCs) cause 10-15% of inherited diseases.
  • Current PTC suppression therapies using small molecules show variable clinical success.
  • Targeting PTCs offers a promising therapeutic strategy for genetic disorders.

Purpose of the Study:

  • To develop a high-throughput assay for identifying anticodon engineered transfer RNAs (ACE-tRNAs).
  • To find ACE-tRNAs capable of efficiently suppressing PTCs and incorporating the correct amino acid.
  • To evaluate the therapeutic potential of ACE-tRNAs in preclinical models.

Main Methods:

  • A high-throughput, cell-based assay was designed to screen for ACE-tRNAs.
  • Genome-wide transcriptome ribosome profiling was used to assess ACE-tRNA interactions.
  • In vitro and in vivo studies were conducted in mammalian cells, Xenopus oocytes, and mice.

Main Results:

  • Identified ACE-tRNAs with high suppression activity against common disease-causing PTCs.
  • ACE-tRNAs demonstrated minimal off-target interactions with translation termination codons.
  • Effective PTC suppression and protein restoration were observed in multiple genes, including CFTR mutations, in vivo.

Conclusions:

  • ACE-tRNAs represent a potent and specific strategy for suppressing PTCs.
  • This approach shows promise for treating genetic disorders caused by nonsense mutations.
  • ACE-tRNAs offer a viable therapeutic alternative to small molecules for PTC suppression.