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Scientists Remove Mutant Gene That Causes Rapid Ageing in Children Using RNA Scissors
Last reviewed: 03.08.2025
Children who develop deep wrinkles, growth retardation, and rapid aging of bones and blood vessels as early as 1–2 years of age may be suffering from Hutchinson–Gilford progeria syndrome (HGPS), a rare and incurable genetic disorder that affects approximately one in eight million people. The average life expectancy for such patients is only 14.5 years, and there is currently no treatment that can completely cure the disease.
The only FDA-approved drug for progeria, lonafarnib (Zokinvy), is extremely expensive: about 1.4 billion South Korean won (approximately $1 million) per dose, and provides only a modest life extension of 2.5 years. The treatment often requires combinations with other drugs and is associated with serious side effects, highlighting the urgent need for more effective and safer therapies.
A research team led by Dr. Sung-Wook Kim from the Next Generation Animal Resource Center, Korea Institute of Bioscience and Biotechnology (KRIBB) has successfully developed the world's first precision RNA-targeted therapy for progeria based on next-generation gene regulation technology. Their innovative approach can selectively eliminate disease-causing RNA transcripts while preserving the function of normal genes, significantly improving safety and opening up new treatment options. The results are published in the journal Molecular Therapy.
What causes progeria?
HGPS is caused by a single mutation in the LMNA gene that results in the production of progerin, a toxic abnormal protein. Progerin disrupts the structure of the nuclear membrane of cells, accelerating cellular aging and causing symptoms similar to premature aging: brittle bones, hardening of blood vessels, and eventual failure of vital organs.
New approach: 'molecular scissors' against progerin
To counteract this, Dr. Kim's team developed RNA-guided “molecular scissors” based on RfxCas13d, combined with a custom-made guide RNA (gRNA) that recognizes progerin.
This precise technology distinguishes between mutant and normal RNAs, allowing selective destruction of progerin without damaging healthy lamin A protein.
Unlike traditional genome editing techniques such as CRISPR-Cas9, which permanently alter DNA and carry the risk of introducing errors outside the target region, the RNA-targeting method works temporarily, does not affect DNA, and is potentially reversible if unintended effects occur.
Results in mouse model
When this method was applied to mice with the progeria mutation, significant reversibility of disease symptoms was achieved, including:
- Hair loss
- Skin atrophy
- Spinal curvature
- Mobility disorders
The animals also recovered:
- Body weight
- Function of the reproductive organs
- Condition of the heart and muscles
In appearance and biomarkers, the treated mice were similar to healthy control animals.
Potential beyond progeria
Moreover, the study found that progerin levels are naturally elevated in aging human skin cells, and using the new RNA-targeted technology helped delay some signs of aging in these cells.
A universal platform for future medicine
Dr. Sung-Wook Kim, lead author of the study, said:
“This technology is not only applicable to Hutchinson-Gilford progeria syndrome, but also has therapeutic potential for more than 15% of genetic diseases caused by RNA editing errors. We expect that it will develop into a universal platform applicable to age-related diseases, cancer and neurodegenerative disorders.”