For the first time researchers have successfully repaired a defective gene inside the human body by using CRISPR gene editing. It marks a significant advancement in genetic medicine. This achievement creates a real hope for people suffering from inherited conditions which seem to be incurable.
The ground-breaking clinical trial, led by Beam Therapeutics, targeted alpha-1 antitrypsin (AAT) deficiency which is an inherited disorder that causes severe lung and liver damage due to the malfunctioning of an AAT protein. The team used an intravenous infusion of lipid nanoparticles to transport CRISPR-based molecular instructions to the liver. These techniques repair a single nucleotide mutation in the AAT gene to enable liver cells to create a healthy form of the protein.
This trial is huge in the world of CRISPR technology. Previous efforts were based on approaches that silenced or knocked out genes, whereas the Beam approach achieves precise gene correction. It marks a transition from symptom treatment to curing the underlying problem of genetic diseases.
The findings, from nine patients, are encouraging. Blood tests reveal high levels of healthy AAT protein, and low levels of the harmful variant. These improvements indicate that the therapy may be a potential cure rather than lifelong treatment.
Beam therapy uses base editing which is better and more precise than CRISPR techniques. In conventional CRISPR methods, both strands of DNA cutting are followed. Whereas base editing allows scientists to convert one DNA to another. This process decreased the risk involved in gene editing, and also enhanced the precision.
Beam therapeutics are optimistic about this therapy and they believe that this therapy might achieve a breakthrough in in vivo gene editing and it could be a sustainable solution for several genetic disorders. This achievement is more than just a medical advancement. It is the beginning of a new era where editing DNA inside the body is not only feasible but a reality.
Sharmin Akter
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References:
- Beam Therapeutics. Beam Therapeutics Announces Positive Initial Data from BEAM-302 Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD). April 2025. Available at: https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-announces-positive-initial-data-beam-302-phase
- National Institutes of Health (NIH). Alpha-1 Antitrypsin Deficiency. MedlinePlus Genetics. https://medlineplus.gov/genetics/condition/alpha-1-antitrypsin-deficiency/