Dr Arkasubhra Ghosh
Director talks about GROW lab

Gene Therapy

Overview

Introduction

Gene therapy focuses on treating disease at its genetic source by introducing, modifying, or regulating genes within affected cells. At Narayana Netralaya Foundation, the Gene Therapy programme is dedicated to developing targeted genetic interventions for ocular diseases, with a primary focus on inherited and acquired retinal and corneal disorders.

The eye offers a uniquely favourable environment for gene therapy due to its immune privilege, compartmentalised structure, and accessibility for precise delivery, making it an ideal organ for sustained genetic intervention.

Why This Research Matters

A significant proportion of blinding ocular diseases are genetic in origin, yet effective treatments remain limited. In India, high genetic diversity and restricted access to advanced therapies further widen this gap. Building indigenous gene therapy capabilities is therefore essential to move beyond symptomatic management towards durable, disease-modifying solutions.

This programme aims to address this need by establishing an end-to-end gene therapy ecosystem, from molecular design to translational readiness.

Objectives

The Gene Therapy programme aims to:

  • Develop gene-based treatments for inherited and acquired ocular diseases
  • Design safe, efficient, and disease-specific gene delivery systems
  • Establish robust and scalable viral vector development pipelines
  • Enable translational pathways towards clinical application

Core Research Focus

The programme integrates multiple gene therapy strategies tailored to disease mechanisms and clinical feasibility. Key areas of focus include:

  • Gene augmentation therapies for loss-of-function mutations
  • Gene recombination and exon-skipping approaches to restore functional gene expression
  • Gene editing strategies for precise correction of pathogenic mutations
  • Gene regulation techniques to modulate abnormal gene activity

A major technical emphasis is placed on adeno-associated virus (AAV) vector engineering, including:

  • Design of novel and chimeric AAV capsids
  • Cell-type-specific and disease-specific targeting
  • Development of dual AAV vector systems for large genes

Additional research areas include:

  • Rational transgene design and truncation strategies
  • Optimisation of high-yield AAV production
  • Modulation of immune responses to sustain gene expression
  • Novel approaches to retinal and ocular gene delivery
  • Pharmaco-gene therapy combinations

Together, these efforts support a diverse portfolio of gene therapy research, spanning retinal degenerations, corneal disorders such as keratoconus, and translational trial planning.

Team

The Gene Therapy programme is led by a multidisciplinary team of scientists and clinician-researchers with expertise in:

  • Molecular Genetics
  • Retinal and Corneal Biology
  • Viral Vector Engineering
  • Translational and Clinical Ophthalmology

Close collaboration between laboratory researchers and clinicians ensures that research priorities remain aligned with patient needs and real-world clinical application.

nVault - Publications | Articles | Research

Multimodal Imaging in Unilateral Acute Idiopathic Maculopathy with Staphylococcus aureus Infection

Multimodal Imaging in Unilateral Acute Idiopathic Maculopathy with Staphylococcus aureus Infection

Authors: Padmamalini Mahendradas, Aditya Patil, Rahul Patil, Raghav Narasimhan, Radhika Sriram, Sai Bhakti Mishra, Ankush Kawali, Poornachandra Gowda, Rohit Shetty, Abhijit Sinha Roy

Keywords: NA

PMID: 39561034

DOI: 10.1080/09273948.2024.2425976

An objective assessment of vision performance at different reading distances after smooth incision lenticular keratomileusis (SILK) for myopia

An objective assessment of vision performance at different reading distances after smooth incision lenticular keratomileusis (SILK) for myopia

Authors: Rohit Shetty, Pooja Khamar, Raghav Narasimhan, Ritika Mullick, Anushree Bhatkal, Anisha Ramesh & Abhijit Sinha Roy

Keywords: NA

PMID: 40781520

DOI: 10.1038/s41598-025-13957-6

Comparative transcriptomic analysis of retinal response to diverse cellular stresses reveals relative contributions of different cell death processes and signalling networks

Comparative transcriptomic analysis of retinal response to diverse cellular stresses reveals relative contributions of different cell death processes and signalling networks

Authors: Subhradeep Sarkar, Ramaraj Kannan, Trailokyanath Panigrahi, Karthickraja Veeramani, Thirumalesh Mb, Shom Shanker Bhattacharya, Arkasubhra Ghosh

Keywords: NA

PMID: 41326361

DOI: 10.1038/s41419-025-08257-w

See All

Future Vision

The Gene Therapy programme is progressing towards:

  • Expanding therapeutic strategies to additional inherited ocular diseases
  • Advancing preclinical candidates towards clinical trials
  • Strengthening regulatory and translational readiness within India
  • Integrating gene therapy with advanced imaging, biomarkers, and functional outcome measures
  • Building long-term capacity for indigenous gene therapy development