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Intravitreal Injections in the Ovine Eye
Published on: July 5, 2022
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Intravitreal enzyme replacement for inherited retinal diseases
Ana Catalina Rodriguez-Martinez1,2,3, James Wawrzynski2,3,4, Robert H Henderson2,3,4
1UCL Institute of Ophthalmology.
Current Opinion in Ophthalmology
|January 3, 2024
Summary
Intravitreal enzyme replacement therapy (ERT) shows promise for treating Batten disease (CLN2) retinopathy. This approach, using recombinant human tripeptidyl-peptidase 1 (rhTPP1), appears safe and may slow retinal thinning.
Area of Science:
- Ophthalmology
- Genetics
- Neurology
Background:
- Metabolic retinal diseases, such as neuronal ceroid lipofuscinosis type 2 (CLN2) or Batten disease, lead to progressive vision loss.
- Batten disease is caused by a deficiency in the lysosomal enzyme tripeptidyl-peptidase 1 (TPP1), resulting in neurodegeneration and blindness.
- Current treatments like intraventricular enzyme replacement therapy (ERT) with cerliponase alfa slow neurodegeneration but do not address retinopathy.
Conclusions:
- Intravitreal ERT with rhTPP1 is a potentially safe and effective treatment for CLN2 retinopathy.
- Further research is needed to determine optimal dosage, frequency, and patient selection for IVT ERT in CLN2.
- Investigating IVT ERT offers a promising avenue for managing the ocular manifestations of Batten disease.
