UK Startup Begins Testing Gene Therapy To Cure Inherited Blindness
There was a time where a number of diseases seemed incurable, and those who had them were coerced at the hands of these diseases and forced to live difficult life and in often cases, an equally difficult death. However a UK-based startup “Nightstar Therapeutics” has recently managed to make strides on a new type of cure known as gene therapy.
There are still diseases that we don’t have a 100% cure. These can be various kinds of cancers or genetic diseases, something we can still not cure despite having modern technology and medicine. In times like these gene therapy serves as a beacon of hope, an experimental technique that uses genes to treat or prevent diseases by putting nucleic acid into a patient’s cells as a drug to treat disease hence transferring genes.
This procedure is something that Nightstar Therapeutics has been attempting to do – primarily targeting a rare case of inherited blindness. They believe that in the near future, it may be possible for doctors to treat a disorder without using surgery or drugs – which not only take a long time to fully function but can often leave behind a plethora of side-effects and abnormalities.
Various testing approaches have been utilized when it comes to gene therapy, such as placing a healthy copy of a gene in the place of the mutated gene that causes the disease.
Silencing, or removing a mutated gene that is not operative properly; and even inserting a new gene that helps fight a disease into the body.
Nightstar Therapeutics’ reports show that initial signs of visual improvements could be found in a Phase I/II trial with a gene therapy for a rare form of inherited blindness. 5 of the 18 patients on whom gene therapy was tested on have already shown enhancements in their vision in just a month.
Even though gene therapy is a viable option for a lot of diseases such as various kinds of cancers, inherited diseases and viral diseases, it is presently only being tested on diseases that have no other treatment option. With researches happening around gene therapy and private companies already beginning trials on human subjects could gene therapy be the treatment we use in the near future? This is a question that many medical experts have begun asking themselves.
Despite the increased traction around this topic – it is quite difficult to truly estimate the viability of gene therapy as a go-to cure for a wide array of ailments. The reason behind this is that genetic treatments have only been tested on a small group of subjects, and as such, the effects on mass populations is still unknown because of the larger genetic diversity among the many different people of the world.
It is unknown that this treatment would work on everyone or there might be exception or even some might have an adverse effect. More funding is required in this research. Needless to say even though we have a lot of issues to tackle before gene therapy becomes public, the revolution towards the future needs to begin from somewhere.
