| Introduction: Contextual introduction. Body: Explain why there is need for new delivery mechanisms for genetic therapy. Conclusion: Write a way forward. |
Gene therapy is the introduction, removal or change in genetic material (typically means DNA and RNA) in the cells of a patient to treat an inherited or developed disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS etc.Gene Editing, Gene Silencing, CAR T Cell Therapyetc.are the various approaches to Gene Therapy.
Need for new delivery mechanisms for genetic therapy:
- The present technologies can only fix the genome in reachable parts of the body, and right now, reach is very limited.The liver, eyes and blood are the main places where cures might be possible.
- These technologies largely rely onviral vectors and lipid nanoparticles. But they can only efficiently deliver to certain Zip codes. Lipid nanoparticles’ routes are largely limited to the liver and eyes.
- These have other limitations, for example how much cargo they can hold. Some genes for fixing the diseases are too big to fit inside a virus. It can be tough to squeeze the instructionsfor making Crispr tools into a usable lipid nanoparticle.
- Some methods avoid the delivery problem altogether by taking cells out of the body, editing them in a lab, and giving them back to patient. But that strategy is lengthy, expensive and tough on patients.
- The fundamental issue with genetic medicines is that our bodies have evolved to keep bad things out of our cells. That’s great for staving off viruses or other pathogens, but also makes it incredibly hard to sneak a medicine in.
- It is also crucial to prevent the gene from being introduced into the wrong cells. It would be inefficient and potentially harmfulto deliver a gene to the wrong tissue.
Gene Therapy has a huge potential to cure rare and untreatable diseases. However, the approach to Gene Therapy requires extreme caution as it can have several long-terms unintended consequences. The field needs appropriate regulation to address the social, equity and ethical concerns.
