Inherited diseases of photoreceptors and prospects for gene therapy
- Resource Type
- Authors
- Joseph C. Corbo; Cynthia L. Montana
- Source
- Pharmacogenomics. 9(3)
- Subject
- Pharmacology
Genetics
Retina
Genetic enhancement
Genetic Therapy
Biology
medicine.disease_cause
Virus
medicine.anatomical_structure
Retinal Diseases
medicine
Molecular Medicine
Humans
sense organs
Gene
Adeno-associated virus
Cis-regulatory element
Photoreceptor Cells, Vertebrate
- Language
- ISSN
- 1744-8042
The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials have yet been initiated to treat diseases caused by mutations in photoreceptor-enriched genes, there is a great deal of optimism regarding the prospects of treating these diseases using adeno-associated virus gene therapy.