🔆 Customised Gene-Editing Treatment for 9-Month-Old Boy ✅ Background: • A 9-month-old boy with a rare genetic disorder (CPS1 deficiency) successfully received a customised gene-editing treatment. • The treatment uses base editing, a new form of CRISPR-Cas9 technology developed by scientists at the University of Pennsylvania and Children’s Hospital of Philadelphia. ✅ What is CRISPR and Base Editing? • CRISPR is a gene-editing tool inspired by bacterial immune systems that can cut and modify DNA. • Base editing differs by enabling precise single-base changes in DNA without making double-strand breaks or inserting foreign DNA. • This precision reduces unintended effects and makes the tool compact and easier to deliver. ✅ How Base Editing Works: • It identifies the abnormal DNA sequence causing the illness. • Uses a guide RNA to direct the Cas9 enzyme to the target DNA location. • Instead of cutting both DNA strands, it modifies a single base to correct mutations (e.g., changing A-C mismatch to A-T). • DNA can naturally repair itself, but there is a chance of sequence regrowth, which scientists counter by re-editing the DNA. ✅ Significance and Future Prospects: • Base editing offers safer and more precise gene therapy options for rare genetic diseases. • The success opens hope for treating many uncommon genetic disorders. • Challenges remain for widespread adoption due to cost, regulatory approval, and the need for personalized treatment. • The technology is not yet common but represents a major step forward in gene therapy.