TECHNIQUES TO PREVENT PRODUCTION OF MUTATED PROTEINS
Several techniques—therapies involving triple-helix-forming oligonucleotides, antisense, and ribozymes—are used to stop the production of a mutated gene and keep the mutated cell from being copied.
This technique targets the DNA sequence of a mutated gene to prevent its transcription. Transcription is the process by which a complementary mRNA molecule is formed from a single-stranded DNA template. The information in the DNA is transferred to the mRNA, which is then used as a template to direct the construction of a protein. Triple-helix-forming oligonucleotide therapy uses a short, single-stranded piece of DNA that binds right into the groove between the double strands of the mutated gene’s DNA. The triple-helix that is produced prevents the segment of DNA from being transcribed into mRNA. |
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This technique turns off a mutated gene in a cell by targeting the mRNA transcripts copied from the gene. Genes are made of two paired strands of DNA. During transcription, the sequence of one strand is copied into a single strand of mRNA, which is called the sense strand in that it has the code to be read for making the protein. The opposite is called the antisense strand. Procedures to perform this therapy involve delivery of an RNA strand containing the antisense code of a mutated gene and binding the antisense RNA strands to the mutated sense mRNA strands, preventing the mRNA from being translated into a mutated protein. |
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This technique turns off a mutated gene by targeting transcripts copied from the gene, thus preventing the production of the mutated protein. Ribozymes are RNA molecules that act like enzymes, serving as scissors to cut RNA. Ribozyme therapy involves delivering the RNA strands that have been engineered to function as ribozymes, where they bind to mRNA encoded by the mutated gene. There the ribozyme cuts off the target RNA and prevents it from being translated into a protein. |
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