Gene Therapy for Retinal Dystrophy : Voretigene Neparvovec (Luxturna)

• What is Gene Therapy treatment for Eyes?

• What is Luxturna?

• Can anyone with Retinitis Pigmentosa receive Luxturna gene therapy?

• What is the process for receiving Luxturna treatment?

• What are the potential side effects of Luxturna?

• Is Luxturna available in India?

• Are there any clinical trials for gene therapy for retinitis pigmentosa in India?

• How can one enroll for gene therapy for retinitis pigmentosa in India?

These are some of the questions we attempt to address here.

What is Gene Therapy treatment for Eyes?

Gene therapy for the eyes is a type of treatment designed to address genetic disorders that affect vision. In a gene therapy procedure, a correct version of a problematic or missing gene is introduced into cells in the eye, typically using a vector (usually a modified virus). The goal is for these new genes to be integrated into the patient's own genetic material, allowing the cells to produce the proteins they need to function correctly.

What is Luxturna?

Gene therapy has shown promise for several eye conditions caused by specific genetic mutations. Leber Congenital Amaurosis (LCA), is one type of inherited retinal dystrophy (IRD) that leads to severe vision loss at an early age. The most well-known example of gene therapy in the eye is the treatment for LCA caused by a mutation in the RPE65 gene. This therapy, known as voretigene neparvovec-rzyl (or Luxturna), was approved by the U.S. Food and Drug Administration (FDA) in 2017.

What is the process for receiving Luxturna treatment?

The treatment works by delivering a normal copy of the RPE65 gene directly into the retinal cells. The therapy uses a modified, harmless virus to carry the gene into the cells. Once inside, the gene can begin producing a normal protein that the cells need to function correctly, with the aim of slowing or stopping the progression of the disease.

This treatment is administered by the Vitreo-Retinal Surgeon who injects the gene therapy solution into the subretinal space of the eye. It's important to note that Luxturna is not a cure. While it can significantly improve vision in some patients, it doesn't fully restore normal vision and it's not effective for all patients.

What are the potential side effects of Luxturna?

The potential side effects of Luxturna may include, eye infection, clouding of the eye lens, thinning of cornea and even retinal detachment in some rare cases. 

Can anyone with Retinitis Pigmentosa receive Luxturna gene therapy?

So if anyone is diagnosed with LCA / or Retinitis Pigmentosa (RP) - what should be the first step? Genetic Testing: One of the first things that may be recommended is genetic testing. This testing can help identify the specific gene mutation causing LCA or RP. This is particularly important because some treatments, like Luxturna, only work for mutation in the RPE65 gene, and the affected person may benefit from it.

Is Luxturna available in India?

Gene Therapy for RPE65 mutation - Luxturna is not available in India yet (as of Sep 2024).

Are there any clinical trials for gene therapy for retinitis pigmentosa in India?

No, clinical trials for gene therapy are not available presently.

How can one enroll for gene therapy for retinitis pigmentosa in India?

Anyone with RP / LCA must get themselves a genetic test to know if they would be eligible for this therapy. It's best to consult an Eye Surgeon beforehand. For further information kindly call on +91 8178519983, or email us on eyegenetics.info@gmail.com, drmayankbansal.md@gmail.com

#SIGHTGENICS

Retinitis Pigmentosa

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(Dr. Mayank Bansal MD(AIIMS), FRCS(Glasg, UK), is a senior consultant in vitreo-retinal surgery. He was awarded the top rank in Senior Residency at AIIMS, New Delhi. Trained at UCLA, USA, and a Fellow of the Royal College of Surgeons of Glasgow, UK - Dr. Bansal and his team follow best practices as performed in top hospitals of the world. )