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Scientists design experimental protein booster for rare genetic diseases
Johns Hopkins scientists develop mRNA ‘booster’ as potential new way to treat rare genetic diseases marked by too low levels of specific cellular proteins
By Published Mar 11 2025
Johns Hopkins Medicine laboratory scientists say they have developed a potential new way to treat a variety of rare genetic diseases marked by too low levels of specific cellular proteins. To boost those proteins, they’ve created experimental versions of a genetic “tail” that attaches to so-called mRNA molecules that churn out the proteins.
Key Takeaways
- The development could pave the way for therapies for rare disorders including some forms of cancer.
- It also holds potential for therapies to treat immune system and neurodegenerative disorders, including SYNGAP deficiency, which results in learning disabilities and autism features in children.
- Next steps include a focus on making the best of an mRNA booster’s design to target a particular disease, and determine whether it can reverse symptoms in animal models of a disease.
A report on the researchers’ proof-of-concept work on laboratory-cultured cells and mice appears in the March 11 issue of Molecular Therapy—Nucleic Acids and could pave the way for therapies for diseases in which one copy of a person’s genes is missing or altered so that only half the amount of protein is made. Such disorders, although rare, include some forms of cancer, and immune system and neurodegenerative disorders, including SYNGAP deficiency, which results in learning disabilities and autism features in children.