New Advances in Genetic Therapies for Pediatric Conditions
Genetic therapies have made significant advancements in recent years, and many of these breakthroughs offer hope for the treatment of various pediatric conditions caused by genetic mutations. Some notable advances in the field of genetic therapies for pediatric conditions include: Gene Replacement Therapy : This approach involves delivering a healthy copy of a defective gene to replace the mutated gene causing a specific disorder. One of the most well-known successes in this area is the approval of Luxturna for the treatment of inherited retinal disorders, which can cause blindness. Gene Editing with CRISPR : CRISPR-Cas9 technology has revolutionized the field of gene editing. Researchers are exploring its potential to correct genetic mutations responsible for pediatric conditions such as sickle cell anemia and muscular dystrophy. Clinical trials and preclinical studies are ongoing to assess the safety and efficacy of CRISPR-based therapies. Antisense Oligonucleotide Therapies : Th...