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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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...
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Gene Therapy00:59

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Angle Closure Glaucoma: Treatment01:28

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Angle-closure glaucoma, or closed-angle glaucoma, is an eye condition where the iris bulges out and blocks the iridocorneal angle, resulting in a buildup of aqueous humor and increased intraocular pressure. Immediate medical attention is necessary due to the sudden onset of symptoms. The treatment for angle-closure glaucoma includes short-term and long-term approaches. Short-term treatment involves using eye drops like pilocarpine to lower intraocular pressure by increasing aqueous humor...
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In open-angle glaucoma, the iridocorneal angle remains open, but the trabecular meshwork becomes stiff, slowing down the outflow of aqueous humor. This causes a buildup of aqueous humor in the anterior chamber, leading to a sudden increase in intraocular pressure. The treatment for open-angle glaucoma focuses on reducing the elevated intraocular pressure by either decreasing the secretion of aqueous humor or increasing its outflow.
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Glaucoma is an eye condition characterized by increased intraocular pressure that damages the retina and optic nerve, leading to irreversible blindness if left untreated. The human eye has various components, including the cornea, iris, pupil, lens, and optic nerve. Aqueous humor is secreted by the epithelium of the ciliary body in the posterior chamber and flows through the trabecular meshwork and canal of Schlemm, maintaining normal intraocular pressure. The trabecular meshwork and the canal...
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Translation01:31

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Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
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Precision Is Not Enough: When Tools Outpace Translation in Ocular Gene Therapy.

Maram E A Abdalla Elsayed1,2, Robert E MacLaren1,2

  • 1Nuffield Department of Clinical Neuroscience, University of Oxford, Oxford OX3 9DU, UK.

Genes
|March 28, 2026
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Summary

Gene therapy in the eye is advancing due to its accessibility and immune privilege. Research synthesizes current strategies, evaluates genome-editing limits, and discusses challenges for clinical use in ophthalmic practice.

Keywords:
AAVCRISPRCRISPR/Cas9Gene editingbase editingclinical trialsocular gene therapyprime editingtranslationvector

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Area of Science:

  • Ophthalmology
  • Molecular Biology
  • Genetics

Background:

  • The eye's unique characteristics, including surgical accessibility and immune privilege, make it an ideal site for gene therapy.
  • The contralateral eye serves as a valuable anatomical control in ocular gene therapy studies.

Purpose of the Study:

  • To review the historical development of gene discovery relevant to ocular conditions.
  • To synthesize current gene therapy strategies for inherited and acquired eye diseases.
  • To critically assess the limitations of CRISPR and genome-editing technologies in ophthalmology.

Main Methods:

  • Historical review of gene discovery.
  • Synthesis of current gene therapy approaches for ocular disorders.
  • Critical evaluation of genome-editing technologies like CRISPR.
  • Examination of scientific and translational challenges.

Main Results:

  • The eye is a prime location for gene therapy due to its accessibility and immune privilege.
  • Current gene therapy strategies show promise for various ocular conditions.
  • CRISPR and related technologies have limitations that need addressing for clinical application.

Conclusions:

  • Integrating genetic therapies into routine ophthalmic practice requires overcoming key scientific and translational hurdles.
  • Further research is needed to refine genome-editing tools and address delivery and safety concerns.
  • The eye remains a leading platform for advancing gene therapy applications.