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Scientists have discovered a new eye gene therapy which can treat patients with a rare eye condition that causes progressive tunnel vision.
In this radical new eye gene therapy, scientists have successfully restored eyesight to patients with this rare eye condition that causes blindness. The breakthrough offers hope to the millions of people worldwide who suffer from vision loss.
The disorder, called Leber’s congenital amaurosis (LCA), is caused by mutations in a gene that is essential for the development of the retina, the light-sensitive layer of tissue at the back of the eye. These mutations lead to the death of photoreceptor cells, which are responsible for converting light into electrical signals that are sent to the brain. As a result, patients with LCA are born with severely impaired vision and often go completely blind by adulthood.
Until just recently, there has been no effective treatment for LCA. But in this new study, scientists have used eye gene therapy to restore vision in patients with the condition.
How the Therapy Works
The therapy works by delivering a healthy copy of the mutated gene directly to the retina. To do this, the researchers created a virus that is harmless to humans but contains the healthy copy of the gene. This virus was then injected into the patients’ eyes, where it enters the retina and begins producing healthy photoreceptor cells.
So far, at the time of the first treatments it has been shown to be safe and effective in three patients who received it. All three showed significant improvements in their vision within just a few weeks of receiving the therapy. Two of them were even able to read letters on an eye chart and one could read large print for the first time in his life.
Lead researcher Dr. Jean Bennett from Penn Medicine said that these results are “very encouraging” and offer “proof-of-concept” that this new therapy can be used to treat other forms of inherited blindness caused by mutations in other genes.
This new treatment has been used on patients in order to prevent blindness, and it has actually helped improve their vision.
Sophie Raworth presents BBC News reporting by medical editor Fergus Walsh.
This is a rare inherited eye condition which causes severe tunnel vision over time.
Scientists are using this new eye gene therapy on patients to try and stop more vision loss, but to they’re surprised that it has also improved their eyesight.
Let’s take a peek inside Jake’s story.
Ever since Jake was born, he has been slowly losing his vision. However, that is no longer the case thanks to this revolutionary eye gene therapy treatment.
I never thought I’d be able to see my own facial features clearly.
“Jake, from County Durham, has an inherited condition that is rare and robs him of most of his central vision.”
After we treated his right eye a year ago, his peripheral vision has gotten stronger.
“I am currently in the best place I have been in for over two decades!”
“Last year for a lot of people it was a dark and a miserable year but for me it was probably easily the best year of my life.”
After a year’s delay due to covid Jake has now had his other eye treated.
The staff at Moorfield’s Eye Hospital in London are hopeful that his vision will further stabilize and, potentially, show some improvement.
The one-time gene therapy called Luxturna is given through an injection and costs 600,000 pounds. However, the NHS has agreed to a discounted price.
Nevartis manufactures an injection that delivers working copies of the RPE 65 gene to the retina at the back of the eye.
The DNA is safely encased in a virus that gets into the retinal cells. Once it’s inside the nucleus, the new gene starts making the RPE 65 protein.
This transformation not only provides hope for people with this specific condition, but also those with similar conditions. This is crucial for maintaining healthy vision.
They are hoping to maintain their eyesight for the long term.
“I keep noticing small improvements. Today, while coming into this park, I noticed one. There are railings located just above the entrance to the gate..” Matthew, who is from London and has the same rare inherited condition, has had one eye treated..
The second operation is scheduled for next month.
When he was just 48, his vision had already gotten worse than Jake’s. 10 years ago I lost my central vision, which then had a huge effect on how I live and who I am.
If the treatment means that If it puts off another decline like that, then that’s going to be amazing.
Matthew’s wife has noticed he is more independent. He doesn’t have to ask me for every little thing, is this on the right? What setting Is that on the washing machine, the Coffee machine.
“You know those things that everyday people take for granted. Matthew can now do himself.”
Around a dozen people around the UK have received the eye gene therapy including several children who stand to benefit most as it may halt their sight loss before permanent damage is done. Fergus walsh bbc news.
Eye Gene Therapy FAQ’s
Can gene therapy restores vision?
The short answer is yes, eye gene therapy has been shown to be effective in restoring vision in some patients.
How could a genetic therapy be used to treat an eye problem?
There are a few ways that a genetic therapy could be used to treat an eye problem. For example, if there is a genetic disorder that affects the vision, a eye gene therapy could be used to replace the faulty gene with a healthy copy of the gene.
Additionally, if there is damage to the retina, a gene therapy could be used to help the cells in the retina regenerate.
Can blindness be cured with eye gene therapy?
Yes, blindness can be cured with gene therapy. There are a few different ways that gene therapy can be used to treat blindness, including replacing the defective gene that causes the blindness, providing a functional copy of the gene, or using a virus to insert the genes into the eye.
So far, eye gene therapy has been successfully used to treat blindness in both children and adults.
Does Luxturna restore vision?
Yes, Luxturna is a eye gene therapy that restores vision in patients with a specific genetic mutation that causes blindness.
The therapy involves injecting a modified virus into the eye, which delivers a healthy copy of the gene to the retina. This replaces the mutated gene and allows the patient to see normally again. Clinical trials have shown that Luxturna is safe and effective, and it was approved by the FDA in December 2017.
How much does Luxturna treatment cost?
The cost of the Luxturna eye gene therapy treatment varies depending on the patient’s insurance coverage. For patients who are not covered by insurance, the cost of the treatment can range from $425,000 to $1,025,000.
Who is eligible for Luxturna?
Luxturna is a treatment for people with Leber congenital amaurosis (LCA) who have a specific genetic mutation.
People with LCA have problems seeing in low light or no light at all. They may also experience problems with their central vision, which is used to see objects clearly and to read.
Luxturna can improve a person’s vision by replacing the defective gene that causes LCA. It is not a cure, but it can help people see better.
What is the disadvantage of Luxturna?
The disadvantage of Luxturna is that it is a very expensive eye gene therapy treatment that is not covered by most insurance plans.
The cost of the treatment can be prohibitive for some people, and there is also the risk that the eye gene therapy may not be completely effective in restoring vision.
There are also some risks associated with the injection procedure, such as eye infection or damage to the retina.
How long does Luxturna last?
The clinical studies have not been long enough to determine the long term durability of the therapy. However, we expect that the benefits provided by a single treatment will last for a period of time.
Does Medicare cover Luxturna?
Medicare covers a portion of the Luxturna treatment cost, but it’s important to check with your specific plan to see what is and isn’t covered.
It’s also important to note that, although Medicare covers a portion of the Luxturna cost, you may still be responsible for some out-of-pocket costs. These costs will vary depending on your individual insurance plan.
So if you’re wondering whether or not Medicare will cover your Luxturna treatment, be sure to contact your insurance company for more information.
Is Luxturna a one time treatment?
Yes. Luxturna is a one-time eye gene therapy treatment and there are no known long-term risks associated with the gene therapy.
Where can you get Luxturna?
It is not available in pharmacies and can only be obtained through a healthcare provider who has been trained to administer the product. To find a healthcare provider who can provide you with Luxturna, please visit https://www.luxturna.com/find-provider/.
There are a few other different places you can get Luxturna. You can either get it from a doctor who is part of the Spark Therapeutics’ Luxturna Direct program, or you can get it from a doctor who participates in one of the company’s expanded access programs.
How well does Luxturna work?
The results from clinical trials have been very promising. One study found that after 1 year of eye gene therapy treatment, 33% of patients who received the therapy had regained some visual function, and 15% were able to read at least 5 letters on an eye chart.
Another study found that after 2 years of treatment, 52% of patients who received the therapy had regained some visual function, and 34% were able to read at least 5 letters on an eye chart.
Overall, it appears that Luxturna does provide some degree of vision improvement
Final Thoughts
We find it so inspiring when scientists uncover new treatments that can help people regain their eyesight. This new eye gene therapy is truly amazing, and I’m hopeful that more people will be able to benefit from it in the future. The fact that this treatment has also improved some patients’ vision is incredible! I’m so glad that Jake was able to receive the treatment and am excited to see how his vision continues to improve. Thank you for sharing this inspiring news with us!
Thank you for reading and good luck with whatever choice you make with your eye gene therapy for Luxturna.
