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Recent advancements and applications of ophthalmic gene therapy strategies: A breakthrough in ocular therapeutics.
Maurya, Rashmi; Vikal, Akash; Narang, Raj Kumar; Patel, Preeti; Kurmi, Balak Das.
Afiliação
  • Maurya R; Department of Pharmaceutics, ISF College of Pharmacy, GT Road, Moga, 142001, Punjab, India.
  • Vikal A; Department of Pharmaceutics, ISF College of Pharmacy, GT Road, Moga, 142001, Punjab, India.
  • Narang RK; Department of Pharmaceutics, ISF College of Pharmacy, GT Road, Moga, 142001, Punjab, India; ISF College of Pharmacy & Research, Rattian Road, Moga, 142048, Punjab, India.
  • Patel P; Department of Pharmaceutical Chemistry, ISF College of Pharmacy, GT Road, Moga, 142001, Punjab, India.
  • Kurmi BD; Department of Pharmaceutics, ISF College of Pharmacy, GT Road, Moga, 142001, Punjab, India. Electronic address: bdkurmi@gmail.com.
Exp Eye Res ; 245: 109983, 2024 Aug.
Article em En | MEDLINE | ID: mdl-38942133
ABSTRACT
Over the past twenty years, ocular gene therapy has primarily focused on addressing diseases linked to various genetic factors. The eye is an ideal candidate for gene therapy due to its unique characteristics, such as easy accessibility and the ability to target both corneal and retinal conditions, including retinitis pigmentosa (RP), Leber congenital amaurosis (LCA), age-related macular degeneration (AMD), and Stargardt disease. Currently, literature documents 33 clinical trials in this field, with the most promising results emerging from trials focused on LCA. These successes have catalyzed further research into other ocular conditions such as glaucoma, AMD, RP, and choroideremia. The effectiveness of gene therapy relies on the efficient delivery of genetic material to specific cells, ensuring sustained and optimal gene expression over time. Viral vectors have been widely used for this purpose, although concerns about potential risks such as immune reactions and genetic mutations have led to the development of non-viral vector systems. Preliminary laboratory research and clinical investigations have shown a connection between vector dosage and the intensity of immune response and inflammation in the eye. The method of administration significantly influences these reactions, with subretinal delivery resulting in a milder humoral response compared to the intravitreal route. This review discusses various ophthalmic diseases, including both corneal and retinal conditions, and their underlying mechanisms, highlighting recent advances and applications in ocular gene therapies.
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Vetores Genéticos Limite: Animals / Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Terapia Genética / Vetores Genéticos Limite: Animals / Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article