Researchers have corrected a disease-causing gene mutation with a single infusion carrying a treatment that precisely targeted the errant gene.

This was the first time a mutated gene has been restored to normal.

The small study of nine patients announced Monday by the company Beam Therapeutics of Cambridge, Mass., involved fixing a spelling error involving the four base sequences — G, A, C and T — in DNA. The effect was to change an incorrect DNA letter to the right one. The result was a normal gene that functioned as it should, potentially halting liver and lung damage of patients with a rare disorder.

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The small study in patients with a rare disorder that causes liver and lung damage showed the potential for precisely targeted infusions.

Cells function through an intricate network of proteins, each designed for specific tasks like metabolism, tissue repair, and immune defense. These proteins are built using genetic blueprints in our DNA. A process called alternative splicing enables a single gene to generate multiple mRNA transcripts — molecules carrying genetic instructions — allowing for protein diversity.

In healthy cells, this process maintains balance. Cancer cells, however, disrupt that process to fuel their unchecked growth by disabling proteins that regulate cell proliferation.

The researchers focused on a genetic element known as a poison exon. This natural “off switch” prevents the production of certain proteins by marking their RNA messages for destruction before they can be translated. Cancer cells suppress the poison exon in a key gene called TRA2β. Without this regulation, TRA2β levels rise, promoting tumor growth and making cancer cells more aggressive.

Researchers have unveiled the first real look at a mitochondrial protein strongly linked to Parkinson’s disease, revealing key details in how its malfunction might play a critical role in the disease’s progress.

Scientists have known for more than two decades that mutations in the gene for a protein called PTEN-induced putative kinase 1 (PINK1) can trigger early-onset Parkinson’s, but the mechanisms at play have remained a mystery.

A team of scientists from the Walter and Eliza Hall Institute of Medical Research (WEHI) in Australia used advanced imaging technology to not only determine the structure of PINK1, but to show how the protein attaches to cellular power houses and how they are activated.

What if everything we thought we knew about cancer was wrong?

For decades, scientists have debated what really causes cancer. Is it genetic mutations, as the Somatic Mutation Theory suggests? Is it a metabolic dysfunction, as the Metabolic Theory argues? Or is there a deeper, overlooked truth—one that could redefine cancer treatment as we know it?

In this episode, Dr. Ralph Moss and Ben Moss break down the battle between competing cancer theories, why conventional wisdom is being challenged, and what the latest research is uncovering about cancer stem cells, metabolism, and the Warburg Effect.

🔥 Are we on the verge of a breakthrough—or have we been on the wrong path all along?

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