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Gene therapy for eye disease

UCL Institute of Ophthalmology Department of Genetics. Gene therapy for eye disease. An experimental technique that uses the delivery of genetic material to treat or prevent disease by: Delivering a working copy of a damaged gene that causes disease – gene supplementation therapy

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Gene therapy for eye disease

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  1. UCL Institute of Ophthalmology Department of Genetics Gene therapy for eye disease • An experimental technique that uses the delivery of genetic material to treat or prevent disease by: • Delivering a working copy of a damaged gene that causes disease – gene supplementation therapy • Inactivating, or “knocking out,” a mutated gene that is functioning improperly – gene silencing • Introducing a new gene into the body to help fight a disease – neuroprotection or angiostatic therapy

  2. What is gene therapy? • A carrier called a vector delivers the therapeutic gene to the patient's target cells – the most common vector is a virus that has been genetically altered to carry the therapeutic gene. • Which vector we choose to use depends on the cells we want to target and the size of the gene we wish to deliver to these cells. • The three types of virus usually used to study gene therapy in the eye are lentivirus (such as HIV), adenovirus and adeno-associated virus (AAV).

  3. Adeno-associated virus (AAV) • AAV is the most commonly-used vector, as it is not harmful even in its "wild" form. • AAV efficiently delivers genes to light-sensitive photoreceptor cells and the underlying retinal pigment epithelium (RPE), making it an ideal gene therapy vector for inherited retinal disorders When injected under the retina, AAV efficiently delivers a reporter gene, encoding Green Fluorescent Protein, to photoreceptor and RPE cells

  4. Direct injection of viral vectors into the eye. • For our gene therapy clinical trials, the surgical procedure involves injecting the virus under the retina, producing a temporary retinal detachment • Recovery time is usually rapid and it is often possible to go home the same day • Quality of sight is reduced temporarily until recovery is complete, which is usually expected to take a few weeks.

  5. Targeting different cells in the eye • Ideally, the therapeutic gene should only be delivered to the cells that would naturally produce this protein • The vector may infect many cell types when it is injected, so by using a cell-specific promoter, we can restrict gene expression to certain cell types within a tissue: (a) photoreceptor cells and RPE; (b) RPE only, (c) cone photoreceptor cells only; (d), (e) corneal cells and (f) Muller cells in the inner retina

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