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

Recent developments in gene therapy research in India

Recent developments in gene therapy research in India

Authors: Selot R, Ghosh A

Keywords: NA

PMID: 38384245

ITF2357 regulates NF-κB signaling pathway to protect barrier integrity in retinal pigment epithelial cells

ITF2357 regulates NF-κB signaling pathway to protect barrier integrity in retinal pigment epithelial cells

Authors: Rayne R Lim, Binapani Mahaling, Alison Tan, Milan Mehta, Charanjit Kaur , Walter Hunziker, Judy E Kim , Veluchamy A Barathi , Arkasubhra Ghosh , Shyam S Chaurasia

Keywords: NA

PMID: 38430220

DOI: 10.1096/fj.202301592R

Tear biomarkers in dry eye disease Progress in the last decade

Tear biomarkers in dry eye disease: Progress in the last decade.

Authors: Kumar NR, Praveen M, Narasimhan R, Khamar P, D’Souza S, Sinha-Roy A, Sethu S, Shetty R, Ghosh A

Keywords: NA

PMID: 37026250

DOI: 10.4103/IJO.IJO_2981_22

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