Exon skipping in DMD
Exon skipping is a technology to neutralize genetic defects by preventing the faulty parts of the gene being used. At the cellular level, a molecule called messenger RNA (mRNA) reads off (transcribes) the protein instructions from the gene. It then transports these instructions to the ribosomes in the cell where the protein is assembled. Messenger RNA is not a transcript of the complete gene sequence, but only of the exons, which are the sections of the gene that code for a portion of the protein. If one of the exons contains an error, this process may be halted, and the production of the full-length dystrophin protein cannot take place. However, when the faulty exon is eliminated (i.e. skipped), protein synthesis does take place and leads to a functional, albeit shorter, dystrophin protein. By using the exon-skipping technique the mutated exons in the dystrophin mRNA that contain errors are "skipped" and as a result the muscle cells are able to produce functional dystrophin protein.
About Amsterdam Molecular Therapeutics
AMT has a unique gene therapy platform that to date appears to circumvent many if not all of the obstacles that have prevented gene therapy from becoming a mainstay of clinical medicine. Using adeno-associated viral (AAV) vectors as the delivery vehicle of choice for therapeutic genes, the company has been able to design and validate what is probably the first stable and scalable AAV production platform. As such, AMT's proprietary platform holds tremendous promise for thousands of rare (orphan) diseases that are caused by one faulty gene. AMT currently has a product pipeline with six products at different stages of development.
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|SOURCE Amsterdam Molecular Therapeutics B.V|
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