Researchers Succeed in Activating Genes Without Cutting DNA

In a landmark shift for genetic medicine, researchers have unveiled a gentler form of CRISPR technology that can reactivate silenced genes without ever cutting a DNA strand. Developed by scientists at UNSW Sydney and St Jude Children’s Research Hospital, this method targets the epigenome—the chemical layer controlling gene expression—rather than the genetic code itself.

For decades, a scientific debate persisted regarding DNA methylation, where small chemical clusters called methyl groups attach to DNA. It was unclear if these groups were merely markers of inactive genes or the cause of the silence. This study, published in Nature Communications, confirmed that removing these methyl groups "brushed the cobwebs off" and turned the genes back on, while adding them back acted as "anchors" to shut them down again.

Unlike previous CRISPR generations that relied on breaking DNA strands—a process that carries a risk of unintended mutations and cancer—this epigenetic editing leaves the underlying DNA sequence untouched. This safety profile is particularly vital for treating lifelong conditions like sickle cell disease.

The research team specifically focused on the fetal globin gene, which is normally switched off after infancy. By removing the methyl "brakes," they can reactivate this gene to compensate for the defective adult globin responsible for sickle cell disorders. In a clinical setting, doctors could one day collect a patient’s blood stem cells, perform this epigenetic editing in a lab, and return the cells to the bone marrow to produce healthy red blood cells.

While currently in the laboratory phase using human cells, the researchers are preparing for animal model testing. Professor Kate Quinlan noted that this breakthrough could eventually treat a wide range of genetic conditions where genes are improperly expressed, marking the beginning of a new age in safer gene therapy.

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CRISPR Breakthrough Activates Genes Without Cutting DNA

This CRISPR breakthrough turns genes on without cutting DNA

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Keywords: Activating Genes Without Cutting DNA

Activating Genes Without Cutting DNA

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