Are There Therapeutic Approaches to Modulate Gene Expression in Pediatrics?
Several therapeutic approaches aim to modulate gene expression:
1. Gene Therapy: This involves introducing, removing, or altering genetic material within a patient's cells. It's being explored for conditions like Duchenne muscular dystrophy. 2. RNA Interference (RNAi): This technique can silence specific genes and is being investigated for treating conditions like spinal muscular atrophy. 3. CRISPR-Cas9: This gene-editing technology can precisely modify genes and holds promise for correcting genetic defects.
