Gene Therapy for Rare Genetic Disorders: Recent Breakthroughs

Abstract

Gene therapy has become an exciting new way to treat rare genetic diseases, with the possibility of long-lasting or even curing solutions.  Recent improvements in gene therapy have made it much safer and more effective, making it a possible way to treat genetic diseases that couldn't be cured before.  Researchers have made a lot of progress in the last few years in learning how genetic diseases work at the molecular level and how gene-based treatments can be used to treat them.  Better and more accurate treatments have been made because of this, especially for rare genetic diseases like spinal muscular atrophy (SMA), cystic fibrosis (CF), haemophilia, and some inherited eye diseases. The FDA's approval of the first gene treatment for spinal muscular atrophy, a serious neurological disease that affects babies, is one of the most important steps forward in gene therapy.  This treatment has been shown to stop the disease from getting worse and make movement skills much better, showing how gene therapy can change things.  Also, improvements in viral vector technology, like adeno-associated viruses (AAV), have made it easier to get restorative genes to the cells that need them.  Now, these vectors are being tweaked to make them more specific, lower defensive reactions, and make transport more effective overall. The creation of CRISPR-Cas9 gene-editing technology has also changed the field by making it possible to fix genetic changes at the DNA level.  It looks like CRISPR could help treat a number of rare genetic diseases, such as sickle cell anaemia and β-thalassemia.  These cutting-edge technologies have made gene therapy easier to get and more successful. They have also made it possible to precisely change genes. Even with these improvements, gene therapy for rare genetic diseases still has some problems.  Some of these are worries about the long-term effects of changing genes, the high cost of treatments, and the lack of good delivery methods.  Even so, these problems are still being worked on in continuing research and clinical studies, which brings gene therapy closer to becoming a common way to treat odd genetic diseases.  Gene therapy has a bright future ahead of it. It could change the field of genetic medicine and give people with diseases that couldn't be treated before hope.