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A Brief History of Genetic Therapy: Gene Therapy, Antisense Technology, and Genomics
Published in Eric Wickstrom, Clinical Trials of Genetic Therapy with Antisense DNA and DNA Vectors, 2020
The second basic technology of modern genetic therapy is antisense technology. This article will adopt the term antisense in a catholic application to refer collectively to four distinct disciplines. These include: classical antisense or anticode; ribozyme or catalytic RNA; triplex, triple helix or antigene; and aptamer approaches. It should be noted that a unified terminology has not been adopted to describe any or all of these technologies.
Biochemistry
Published in Sarah Armstrong, Barry Clifton, Lionel Davis, Primary FRCA in a Box, 2019
Sarah Armstrong, Barry Clifton, Lionel Davis
Macromolecular biological catalysts that accelerate chemical reactions whilst remaining themselves unchanged Almost all metabolic processes need enzymes to occur at rates fast enough to sustain lifeMost are globular proteins, much larger than their substrates. A few are catalytic RNA molecules (ribozymes)May be highly specific for a given substrate – specificity comes from their unique three-dimensional structuresSensitive to pH and temperature change
Nucleic Acids as Therapeutic Targets and Agents
Published in David E. Thurston, Ilona Pysz, Chemistry and Pharmacology of Anticancer Drugs, 2021
Ribozymes (the name derives from a combination of “ribonucleic acid” and “enzyme”) are catalytic RNA molecules that can carry out specific biochemical reactions in a similar manner to protein-based enzymes. They are similar to antisense oligonucleotides in that they target and bind to complementary sequences of mRNA. Many ribozymes have either a hairpin or hammerhead form (Figure 5.96) with a shaped active center and a unique secondary structure that allows them to cleave other RNA molecules at specific sequences. Although isolated ribozymes are not common in cells, their roles are essential for some components of the cellular machinery. For example, the functional large subunit component of the ribosome, the molecular machine that translates RNA into proteins, is essentially a ribozyme composed of RNA tertiary structural motifs that can be coordinated to metal ions such as Mg2+ as cofactors. Molecular model of a hammerhead ribozyme (Taken from Wikipedia, “Minimal hammerhead ribozyme structure” by I, Wgscott, under the Creative Commons Attribution-Share Alike 3.0 Unported license (https://creativecommons.org/licenses/by-sa/3.0/legalcode)).
Virus-associated ribozymes and nano carriers against COVID-19
Published in Artificial Cells, Nanomedicine, and Biotechnology, 2021
Beyza Dönmüş, Sinan Ünal, Fatma Ceren Kirmizitaş, Nelisa Türkoğlu Laçin
The hammerhead RNA is a single-stranded RNA motif that undergoes autocatalytic self-cutting, and because of its feature, it is not a real enzyme in the biological context [71]. The terms “hammerhead ribozyme” and “hammerhead RNA” can be used interchangeably [72]. The structure of hammerhead ribozymes consists of three helices called I, II and III attached to an 11-nucleotide length catalytic core. The catalytic core has a substrate recognition area containing two bodies on either side of the dividing region [73]. Hammerhead and hairpin ribozymes are small, sequence-specific RNA endonucleases. They can be manipulated easily for maintaining specificity and regulate catalytic activity [74]. However, a relatively insignificant small connection gains catalytic properties by removing the loop region from the strand and gives the ability to react with multiple substrates. Hammerhead ribozymes are the most remarkable catalytic RNA motifs for gene editing activities [75,76]. Besides, ribozymes have been frequently used in various viral inhibition studies [77–79].