In a new study that could ultimately lead to many new medicines, scientists from the Florida campus of The Scripps Research Institute (TSRI) have adapted a chemical approach to turn diseased cells into unique manufacturing sites for molecules that can treat a form of muscular dystrophy.
“We’re using a cell as a reaction vessel and a disease-causing defect as a catalyst to synthesize a treatment in a diseased cell,” said TSRI Professor Matthew Disney. “Because the treatment is synthesized only in diseased cells, the compounds could provide highly specific therapeutics that only act when a disease is present. This means we can potentially treat a host of conditions in a very selective and precise manner in totally unprecedented ways.”
The promising research was published recently in the international chemistry journal Angewandte Chemie.
Targeting RNA Repeats
In general, small, low molecular weight compounds can pass the blood-brain barrier, while larger, higher weight compounds tend to be more potent. In the new study, however, small molecules became powerful inhibitors when they bound to targets in cells expressing an RNA defect, such as those found in myotonic dystrophy.
Myotonic dystrophy type 2, a relatively mild and uncommon form of the progressive muscle weakening disease, is caused by a type of RNA defect known as a “tetranucleotide repeat,” in which a series of four nucleotides is repeated more times than normal in an individual’s genetic code. In this case, a cytosine-cytosine-uracil-guanine (CCUG) repeat binds to the protein MBNL1, rendering it inactive and resulting in RNA splicing abnormalities that, in turn, results in the disease.
In the study, a pair of small molecule “modules” the scientists developed binds to adjacent parts of the defect in a living cell, bringing these groups close together. Under these conditions, the adjacent parts reach out to one another and, as Disney describes it, permanently hold hands. Once that connection is made, the small molecule binds tightly to the defect, potently reversing disease defects on a molecular level.
“When these compounds assemble in the cell, they are 1,000 times more potent than the small molecule itself and 100 times more potent than our most active lead compound,” said Research Associate Suzanne Rzuczek, the first author of the study. “This is the first time this has been validated in live cells.”
The Latest on: Specific therapeutics
via Google News
The Latest on: Specific therapeutics
- Virios Therapeutics Achieves Over 50% Enrollment Milestone in its Phase 2b Clinical Trial for Fibromyalgiaon December 1, 2021 at 4:43 am
Virios Therapeutics, Inc. , a development-stage biotechnology company focused on advancing novel antiviral therapies to treat debilitating chronic diseases, including fibromyalgia, announced today ...
- New therapeutic target for HIV identifiedon December 1, 2021 at 4:14 am
Scientists in Montreal and London have identified the key role played by transcription factor RORC2 in HIV infection: the molecule attaches itself to parts of the virus's genome yet doesn't prevent it ...
- G1 Therapeutics Initiates Phase 2 Trial to Support the Antitumor Mechanism of Action (MOA) of Trilaciclib in the Tumor Microenvironmenton November 30, 2021 at 10:00 pm
G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, today announced that the Company has initiated a Phase 2, single arm, open-label study of trilaciclib in patients with ...
- XNK Therapeutics to present at ABGSC Private Companies Seminar on December 6on November 30, 2021 at 5:14 pm
XNK Therapeutics AB's CEO, Johan Liwing, will present at the ABGSC Private Companies event on December 6. The presentation starts at 09:30CET and will be livestreamed via The presentation will be ...
- Oligonucleotide Synthesis Market worth $14.1 billion by 2026: Industry-Specific Challenges, Opportunities and Trends Affecting the Growthon November 30, 2021 at 5:05 pm
The oligonucleotide synthesis market is dominated by a few globally established players such as Danaher Corporation (US), Thermo Fisher Scientific, Inc. (US), Merck ...
- Silence Therapeutics Begins Exclusive Nasdaq Tradingon November 30, 2021 at 4:10 am
Silence’s American Depositary Shares, each representing three Ordinary Shares (the “ ADSs ”), are now listed and only tradeable on the Nasdaq Global Market (“ Nasdaq ”) under the ticker symbol SLN.
- Defence Therapeutics Vaccines and Program Developmenton November 30, 2021 at 12:15 am
Defence Therapeutics Inc. (CSE: DTC) (FSE: DTC) (OTC Pink: DTCFF) ("Defence" or the "Company"), is a publicly-traded biotechnology company working on engineering the next generation vaccines, Antibody ...
- Parvus Therapeutics Announces Development and Manufacturing Agreement with Resilience to Advance Novel Autoimmune Drug Candidate PVT201on November 29, 2021 at 7:27 am
Parvus Therapeutics, a biopharmaceutical company developing Navacim™ platform-based disease-specific immunoregulator therapeutics to treat ...
- Global Targeted Oncology Therapeutics for Lung Cancer: Leading Competitors in the Field of Next-Generation Therapeuticson November 29, 2021 at 6:45 am
Lung Cancers" report has been added to ResearchAndMarkets.com's offering. A number of factors are changing the way market participants view the cancer therapeutics market. These factors are evident in ...
via Bing News