Imagine having to live in a world of shadows, and no matter how you squint, everything that was normal has vanished into a very dark place. Such is not a nightmare but a reality for those millions who are victims of blindness because of hereditary causes. What if the same science were able to do the opposite? That is where CRISPR gene editing comes in: the handy tool whose rules have rewritten medicine and raised the hopes of people with a genetic disorder in their eyes.
In this review, we go over the uses of CRISPR in fighting inherited blindness, describe what science has managed to do so far and discuss what the future may look like for people suffering from the condition. Whether a person is a curious reader or has a bit of personal interest in this topic, rest assured that all will be explained here, using simple terms understandable by any.
What is Inherited Blindness?
Inherited blindness is a mutation of a gene required for normal vision, for example, like inherited conditions: Retinitis pigmentosa, LCA, Stargardt disease; Progressive loss of vision occurs, whether from childhood or in later years.
This would say that a disease such as Leber congenital amaurosis should, in most instances, be diagnosed within babies through parents when they begin to notice their child does not respond to sight. A friend very close to me shared how her little boy, Ethan, was not reaching for his toys, and it became a storm of doctors until the diagnosis came out: LCA. To hear that from her was a total devastation for her, which somehow drove her to seek more answers, bringing her deep into doing some research on CRISPR.
What is CRISPR?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It is a pair of molecular scissors that scientists can program to target and edit certain parts of the DNA sequence. Think of it as reading one typo inside a book. That’s what CRISPR does: finds that mistake and corrects it, almost like a red pen.
That is, it will correct genetic mutations leading to some forms of inherited blindness-CRISPR, so far as inherited blindness goes, either sight or may never let such a state occur, to say so.
Progress So Far
Amazingly, that is where CRISPR has gone on a journey of treating inherited blindness. Indeed, two decades later in 2020, the world could see the very first CRISPR-based therapy tested directly in humans for LCA. It was named EDIT-101; the treatment entails the injection of CRISPR components into the retina to correct the mutation in the CEP290 gene, which generally covers the cause of LCA.
Although the initial results that came out were quite encouraging number of patients had reported improvement in their perception of light and partial restoration of vision in a few cases treatment is not at all a complete cure as of now, but small victories have kept the hope alive.
Another very interesting development was base editing refinement upon CRISPR which can enable scientists to make changes without cutting into the DNA. This, scientists theorize, reduces some of the risks of unnecessary edits, say-and makes treatments not just far safer but more efficient, too.
Challenges and Ethical Considerations
Yet big as the promise is, a lot of problems remain with CRISPR: gene editing in a living organism is labyrinthine, and there’s always a possibility of off-target effects changes in the DNA that could cause health problems somewhere else.
There are ethics, too: whether people in this century should start editing genes in embryos, stopping inherited blindness for example. Where do you draw a line between disease and enhancement? These are going to be some of the questions society is going to have to make a decision upon if and when the technology advances further.
The potential of CRISPR is a huge fact. Scientists say genetic eye disorders are now theoretically capable of diagnosis and treatment before they show up, with only one tiny injection into the eye and a correction to a fault in a gene, and one’s vision would be restored.
It is this future that promises Ethan a better life for my friend’s son. Though these therapies are not available today, using CRISPR they are rapidly developing. For such development, it is for families like hers that will keep watching with hope that one day no child will grow up in the dark.
Why This Matters
The story of CRISPR and inherited blindness is little more than a tale of scientific advance. It is resilience, it’s hope; it’s that life force inside each human chest. And every time something better comes onto the stage, it catches our dreams: a world, definitely far off into the horizon, in which blindsides engineered by genetics shall no more plague humanity.
Either due to blindness inherited, this is certainly the most exciting time in every respect to be alive in so far as medical advancement. We all can donate to research organisations, be aware of the cause and, well any way we are able to support this life-changing technology.
In Conclusion, CRISPR gene editing is rewriting the future of people born blind. That is not sight; that is restored hope, independence, and quality of life. Though still a long way to go, as far as progress has been made so far, CRISPR does already testify to human ingenuity and the power of science to change lives.
If you’re as psyched about it as I am, let’s not let this conversation die down. Forward this article ahead to your friend, follow the latest and greatest that CRISPR is working on, but most importantly lose faith in the power of innovation.