Question

1. Gene therapy involves inserting a functional copy of a gene into the genome to treat or prevent disease. However, in reality this process is not as easy as it sounds. What were some challenges the researchers faced as they tried to develop a gene therapy to treat Leber Congenital Amaurosis (LCA)?

2. Luxturna gene therapy officially costs $425,000 per eye ($850,000 for both eyes). Do you think that this is an appropriate price for this treatment?

Answer 1

1. There are various challenges faced by a gene therapy technique. These are-

gene delivery and activation, immune response, disrupting important genes in target cells, the possibility of causing a tumour, targeting wrong cells, etc.

2. Luxturna is a liquid that is injected directly into the eye with a microscopic needle during a surgical procedure. The drug supplies a third gene -- a normal RPE65 gene -- to cells in the retina. This third version of the gene does not remove or replace the two mutated genes causing the disease, which remain inside the cells. However, the normal gene delivered by the treatment is able to function within the retinal cells and correct vision problems.

It is a highly microscopic and very delicate procedure in which a slight mistake can cause wrong delivery and instead of healing it may cause adverse effects. Also, Luxturna liquid preparation requires a large sum of money. But 850,000 for both eyes is a huge sum of the money. According to me, some cost-cutting can be made in it.