Related Experiment Video

Updated: Jun 21, 2025

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models
06:16

Subconjunctival Administration of Adeno-associated Virus Vectors in Small Animal Models

Published on: March 16, 2022

2.9K

Gene therapies in pediatric ophthalmology

Alejandra Daruich1,2, Matthieu P Robert1,3, Dominique Bremond-Gignac1,2

  • 1Ophthalmology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris Cité University, Paris, France.

Frontiers in Ophthalmology
|July 10, 2024
PubMed

Insights

Voretigene neparvovec, the first gene therapy for inherited retinal dystrophy (IRD), shows promise in children. Ongoing research explores further gene therapy applications in pediatric ophthalmology for vision impairment.

Area of Science:

  • Ophthalmology
  • Genetics
  • Medical Research

Background:

  • Genetic pediatric eye diseases cause significant vision loss.
  • Voretigene neparvovec is the first approved gene therapy for inherited retinal dystrophy (IRD).
  • Early childhood administration of voretigene neparvovec demonstrates safety and efficacy.

Purpose of the Study:

  • To review recent advancements in gene therapy for pediatric inherited retinal dystrophies.
  • To discuss ongoing studies and unanswered questions regarding gene therapy in young children.
  • To explore various gene therapy approaches in pediatric ophthalmology.

Main Methods:

  • Literature review of recent reports.
  • Analysis of ongoing clinical studies.
  • Synthesis of current knowledge on gene therapy for pediatric eye diseases.

Main Results:

  • Voretigene neparvovec is a safe and well-tolerated treatment for IRD.
  • Early intervention with gene therapy in children yields encouraging efficacy.
  • Several promising gene therapy approaches are under investigation.

Conclusions:

  • Gene therapy offers a new frontier in treating pediatric genetic eye diseases.
  • Further research is needed to address specific questions related to early childhood gene therapy.
  • The field of gene therapy in pediatric ophthalmology is rapidly evolving.

Keywords:
aniridiaatalurenchildrengene therapygenetic ocular diseasesoptogenetic therapypediatric ophthalmologyvoretigene neparvovec

More Related Videos

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium06:48

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

Published on: August 7, 2015

21.2K
Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye05:09

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

Published on: November 25, 2012

33.2K

Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
25.4K
iPS Cell Differentiation01:22

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
2.7K