Gene Therapy and Editing: Novel options for inherited retina
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Millions of people the world over suffer visual disability as a result of retinal dystrophy that involves the death of retinal cells that are important for the light-sensing function of the eye. Enormous progress has been made in other blinding conditions involving the cornea, lens, among others. However, the retinal dystrophies and optic nerve atrophies do not have any proven therapy to date.

In most cases, the retinal cells are present at birth, but undergo gradual death during the later stages of life. Inherited defects in many genes involved in retina-specific functions and vitamin A metabolism are linked to various forms of retinal dystrophies. These genetic defects affect the normal cellular functions of the retina, leading to gradual cell death and ultimately the patient becomes legally blind.

It is administered as an onetime injection behind the retina of an eye of patients genetically diagnosed to carry mutations in the RPE65 gene and also has sufficient viable retinal cells.

This is the first gene editing based drug approved by US-FDA, for the treatment of another early childhood retinal dystrophic condition called LCA10, caused by defects in the CEP290 gene. Such treatment considerations require a thorough genetic screening/genotyping to confirm the identity of the gene affected in a specific patient.

As of May 2020, the database lists about 271 genes to be associated with different forms of retinal dystrophies. This requires a larger library of gene delivery vectors to be developed and made available at affordable costs for the treatment of a large number of patients.

This mandates the need for developing indigenous and cost-effective therapeutics and ICMR has set up a dedicated task force on gene therapy research, to identify and support promising research ideas in this newly emerging area of biomedical research. A national guideline for gene therapy product development and clinical trials has been jointly formulated and released by the DBT and ICMR in 2019.

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