Important note: Information in this article was accurate in 2000. The state of the art may have changed since the publication date.
PRNewswire - Tuesday December 19, 2000
The usual process of producing proteins is highly accurate and ensures that individual mRNAs will almost always lead to very specific proteins being made. HIV, however, confronted by limitations of its small genome size, evolved ribosomal frameshifting as a strategy that enables the virus to make two critical proteins, instead of the usual one. Interfering with the normal frameshifting process causes the ribosome - the cell's protein-making engine - to misstep and thereby fail to produce one of these essential proteins.
Ultimately, this results in reduced viral replication.
"Ribosomal frameshifting is an innovative therapeutic target for HIV," said Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics. "Receiving the Phase I SBIR Grant provides scientific validation for our approach and will allow us to expand the application of our expertise in RNA-based drug discovery to a disease where new therapeutic targets are needed."
Frameshifting events are predictable and determine the precise ratios of structural and replication proteins required for formation of functional viruses. Since alterations in frameshifting frequencies cause major reductions in viral replication, the frameshifting process is a novel and potentially important viral target.
"Viruses often exploit the cellular translation system in many ways in order to gain an advantage over the cell," said Robert Rando, Ph.D., Senior Director of Biology, PTC Therapeutics. "The fact that HIV utilizes ribosomal frameshifting can be used against it to inhibit virus growth. We look at ribosomal frameshifting as a unique molecular target of the virus that can be utilized in our drug discovery efforts."
PTC has focused on developing both novel assays and chemistry that will select for small molecules that modulate gene expression at the post- transcriptional level. The mechanism of ribosomal frameshifting is a great example of how manipulating the cell's translation machinery by small molecules can lead to new and novel antiviral therapeutics.
PTC Therapeutics, Inc. applies an integrated RNA biology and chemistry platform to create small molecule drugs. PTC's technologies allow drug discovery programs to progress rapidly from gene sequencing to lead compounds by identifying small molecules that selectively bind to either RNA targets or proteins that interact with RNA. PTC is working in collaboration with Tularik, Inc. to discover therapeutics that can treat a large number of genetically inherited disorders, such as cystic fibrosis and inherited forms of cancer. Utilizing its technology platform, PTC is developing orally active, small molecule drugs to address a broad range of therapeutic targets. PTC's technology provides a general approach for all genetic-based diseases and the Company currently has drug discovery programs in oncology, infectious diseases and inflammatory disorders.
For more information please contact: Christopher Blaxland Chief Operating Officer PTC Therapeutics, Inc. Phone: (908) 222-7000 cblaxland@ptcbio.com www.ptcbio.com
Rod Smith Ogilvy Public Relations Worldwide-Boston Phone: (617) 577-0006 rsmith@fkhealth.com
SOURCE: PTC Therapeutics Inc.
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