A collaborative effort led by UCL, King's College London, and Moderna scientists has yielded promising results in utilizing messenger RNA (mRNA) technology to address a rare genetic liver disease known as argininosuccinic aciduria. The research, conducted in a mouse model of the disease, demonstrates the potential therapeutic applications of mRNA, the same technology employed in Covid-19 vaccines.
Published in Science Translational Medicine, the study focused on correcting argininosuccinic aciduria, an inherited metabolic disorder affecting approximately one in 100,000 newborns. The disease disrupts protein breakdown, leading to elevated ammonia levels in the blood and an imbalance of glutathione regulation crucial for liver detoxification.
The researchers successfully employed mRNA to correct the genetic anomaly in mice, providing a potential breakthrough for treating this rare liver disease in humans. The team plans to progress to human trials over the next few years, with ongoing investigations into other rare inherited metabolic diseases such as propionic and methylmalonic acidaemias.
Rare diseases, often arising from DNA errors, impact around 300 million people globally, yet less than 5% of these conditions have approved therapies. Traditional gene therapy, involving the replacement of faulty genes, faces challenges related to safety and widespread applicability.
The study aimed to explore the viability of mRNA technology as an alternative solution. mRNA, a molecule containing instructions for protein synthesis, was encapsulated in lipid microdroplets, enabling targeted delivery to liver cells when injected intravenously.
In a trial involving 31 mice, the researchers observed the correction of glutathione regulation and successful disease treatment. While the benefits of each mRNA treatment lasted approximately seven days in mice, the researchers anticipate longer intervals between treatments in human applications.
Positron emission tomography (PET) scans tracked the correction of glutathione regulation and validated the treatment's success. Mice treated with mRNA therapy at birth exhibited significantly extended lifespans compared to untreated counterparts, highlighting the therapeutic potential of mRNA in addressing genetic liver diseases.
A collaborative effort led by UCL, King's College London, and Moderna scientists has yielded promising results in utilizing messenger RNA (mRNA) technology to address a rare genetic liver disease known as argininosuccinic aciduria. The research, conducted in a mouse model of the disease, demonstrates the potential therapeutic applications of mRNA, the same technology employed in Covid-19 vaccines.
Published in Science Translational Medicine, the study focused on correcting argininosuccinic aciduria, an inherited metabolic disorder affecting approximately one in 100,000 newborns. The disease disrupts protein breakdown, leading to elevated ammonia levels in the blood and an imbalance of glutathione regulation crucial for liver detoxification.
The researchers successfully employed mRNA to correct the genetic anomaly in mice, providing a potential breakthrough for treating this rare liver disease in humans. The team plans to progress to human trials over the next few years, with ongoing investigations into other rare inherited metabolic diseases such as propionic and methylmalonic acidaemias.
Rare diseases, often arising from DNA errors, impact around 300 million people globally, yet less than 5% of these conditions have approved therapies. Traditional gene therapy, involving the replacement of faulty genes, faces challenges related to safety and widespread applicability.
The study aimed to explore the viability of mRNA technology as an alternative solution. mRNA, a molecule containing instructions for protein synthesis, was encapsulated in lipid microdroplets, enabling targeted delivery to liver cells when injected intravenously.
In a trial involving 31 mice, the researchers observed the correction of glutathione regulation and successful disease treatment. While the benefits of each mRNA treatment lasted approximately seven days in mice, the researchers anticipate longer intervals between treatments in human applications.
Positron emission tomography (PET) scans tracked the correction of glutathione regulation and validated the treatment's success. Mice treated with mRNA therapy at birth exhibited significantly extended lifespans compared to untreated counterparts, highlighting the therapeutic potential of mRNA in addressing genetic liver diseases.
Dr. Baruteau, one of the lead researchers, emphasized the unprecedented therapeutic potential of mRNA for incurable genetic diseases, particularly liver conditions. The collaborative effort between academia and industry, supported by funding from organizations including Moderna, the Medical Research Council, and Wellcome, showcases the promising future of mRNA technology in advancing treatment options for rare genetic disorders.
Dr. Baruteau, one of the lead researchers, emphasized the unprecedented therapeutic potential of mRNA for incurable genetic diseases, particularly liver conditions. The collaborative effort between academia and industry, supported by funding from organizations including Moderna, the Medical Research Council, and Wellcome, showcases the promising future of mRNA technology in advancing treatment options for rare genetic disorders.
