Scientists at UNSW Sydney have unveiled a novel CRISPR technology that may enhance the safety of gene therapy while addressing longstanding questions regarding gene regulation. Their research indicates that small chemical markers, known as methyl groups, actively suppress gene activity rather than merely indicating inactive genes.
Historically, the role of these methyl groups has been debated. A recent study published in Nature Communications demonstrates that their removal reactivates genes, while their reintroduction silences them again, confirming their direct influence on gene function. Professor Merlin Crossley, the study’s lead author, emphasized that these markers are integral to gene regulation.
CRISPR, an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, forms the basis of modern gene editing, allowing targeted modifications to DNA sequences. Initial CRISPR tools focused on cutting DNA to disable faulty genes. However, these methods often resulted in unintended consequences and potential side effects. The latest iteration, termed epigenetic editing, circumvents this issue by targeting the chemical markers rather than the DNA itself, aiming to restore gene activity without altering the genetic sequence.
The researchers believe this technique could lead to safer treatments for Sickle Cell disease, a condition characterized by abnormal red blood cell function. Professor Crossley pointed out that avoiding DNA cuts reduces cancer risks associated with gene therapy.
Initial experiments have been conducted in laboratory settings using human cells. The findings could have broader implications for various genetic conditions where gene expression is misregulated. Future studies aim to test this approach in animal models, exploring the potential for epigenetic editing in therapeutic and agricultural applications. The researchers view this as the beginning of a new era in gene therapy.Source: https://www.pressrundown.com/health/breakthrough-crispr-method-activates-genes-without-cutting-dna?lctg=6508b820e8179a307e601d89
