Gene therapy-1.0 Imagine a miracle cure with just one injection Ur whole disease would permanently disappear, Hard to believe but so true in this modern technological world.
We all might be familiar with gene therapy concept, one which brought revolution to mankind disease treatment, by directly targeting the disease-causing gene. 1990 marked the start of gene therapy by curing a little girl who had no hope of living, Since then we have faced many ups and down in gene therapy technology and it’s development. Overall we could say that it is a ‘ technique Of treating a disease or disorder by inserting a gene into a patient’s cell instead of using drugs and surgery’. Although it is a miracle technology to cure the disease it has its own setback and issue.
Risk and setback of gene therapy:-
Gene therapy has faced one set back after the other due to which it’s development took rather slow and sometimes stopped, earlier gene therapy failure reasons or setback can be seen because of following reasons. 1. Delivery mechanism failure:- earlier the delivery mechanism used was not fully efficient or effective, due to which they failed to deliver the gene to specific target Case study:- 1990- a 4-year-old girl suffering From ADA was treated in national institute of health, But due to the failure of delivery mechanism, even after getting cured she suffered from leukemia due to the failure of the delivery mechanism 2.faliure of delivering the gene to the target-Due to gene targeting more than one target location the person developed cancer or much other disorder 3.vector used caused dysfunctional of organ- vector or virus used lead to damage to the immune system or other organs Case study: -1999- 17-year-old boy suffering from OTC deficiency, the virus used for the treatment of disease lead to failure of his immune system leading to multiple organ failure or death 4.only applicable to monogentic disease:- disease which has more than more target location or multiple target location, Gene therapy cannot be used to permanently cure them Such as heart failure, cancer, diabetes, cancer although in cancer-like disease gene therapy can be used to suppress cancer-causing cells and with chemotherapy can be used to treat it completely Gene therapy – 2.0 (new breakthrough the technology) Even after many sets back gene therapy are a major break out last year, due to which many more diseases are cured by gene therapy and even more are being constantly researched
1.SCID was treated in a 4-year-old boy by experimental gene therapy at Boston children hospital by successfully replacing the gene responsible for damaging the immune system. 2. Europe has granted the approval of treatment of two inheridetary diseases by gene therapy which is flyers disorder that makes the fat build up in bloodstream and strimeluris a form of SCID
1. The United States became the first country to market spark therapeutic treatment for a progressive form of blindness, spending over $80,000. The 2.44-year-old man became the first person to Recieve treatment of gene editing therapy that directly modifies his gene cell this technology can be used to further modify gene and to increase ones age etc. 3.development for the cure for hemophilia and epidermolysis Bullmore (skin disease) for a 7-year boy whose skin was scraped off.
Development of CRISPER-as 9, a genome-editing tool. Which caused a new wave of DNA modification and got recently approved by US government. It is an editing tool which can directly cut the damaged gene permanently so it could not create further damage. Although in recent cases, there have been few viruses which r immune to CRISPER But further technology are still being developed. And there still arises many set back and complication but it is believed that gene therapy will continue developing further and enhance the treatment of disease by multiple folds especially diseases which cute is yet to be found.