Gene therapy beyond luxturna: a new horizon of the treatment for inherited retinal disease
| Autor: | Ramiro S. Maldonado, Dominic A Prado, Marcy Acosta-Acero |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
Genetic enhancement Genetic Vectors MEDLINE Disease 03 medical and health sciences 0302 clinical medicine Genome editing Retinitis pigmentosa Humans Medicine Gene Silencing Intensive care medicine Gene Editing biology business.industry Lentivirus Retinal Degeneration Genetic Therapy General Medicine Dependovirus biology.organism_classification medicine.disease Clinical trial Ophthalmology RPE65 030221 ophthalmology & optometry business 030217 neurology & neurosurgery |
| Zdroj: | Current Opinion in Ophthalmology. 31:147-154 |
| ISSN: | 1531-7021 1040-8738 |
| Popis: | Purpose of review Gene therapy offers, for the first time, the possibility to cure diseases such as retinitis pigmentosa. The positive outcomes that led to the U.S. Food and Drug Administration (FDA) approval of Luxturna to treat Leber congenital amaurosis caused by RPE65 mutations created an optimistic atmosphere in the research, clinical and patient community. Despite this first success, we must understand that this is not a 'one treatment for all'. This review aims to explain the basic concepts of gene therapy and how they translate in different approaches that are utilized in ongoing clinical trials here reviewed. Recent findings In 2017, the FDA approved the first gene therapy treatment. In parallel, other approaches have gained attention. Different delivery methods (adeno-associated virus, lentivirus), injection sites (subretinal, intravitreal, suprachoroidal) and methodologies (gene replacement, silencing, editing) are currently being tested. Summary Gene therapy is an evolving field in medicine and ophthalmology. Its success and application depends on several factors that are specific to the disease to treat. For now, we know it's a relatively safe approach and we look forward to the continued advancements of current ongoing clinical trials. |
| Databáze: | OpenAIRE |
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