AAV-mediated gene therapy for retinal disorders: from mouse to man
| Autor: | Prateek K. Buch, James W B Bainbridge, Robin R. Ali |
|---|---|
| Rok vydání: | 2008 |
| Předmět: |
Retinal degeneration
Pathology medicine.medical_specialty Retinal Disorder viruses Genetic enhancement Genetic Vectors Gene delivery Biology Bioinformatics Viral vector chemistry.chemical_compound Mice Retinal Diseases Genetics medicine Animals Humans Molecular Biology Retina Gene Transfer Techniques Retinal Genetic Therapy Dependovirus medicine.disease medicine.anatomical_structure RPE65 chemistry Models Animal Molecular Medicine Safety |
| Zdroj: | Gene therapy. 15(11) |
| ISSN: | 1476-5462 |
| Popis: | A wide range of retinal disorders can potentially be treated using viral vector-mediated gene therapy. The most widely used vectors for ocular gene delivery are based on adeno-associated virus (AAV), because they elicit minimal immune responses and mediate long-term transgene expression in a variety of retinal cell types. Proof-of-concept experiments have demonstrated the efficacy of AAV-mediated transgene delivery in a number of animal models of inherited and acquired retinal disorders. Following extensive preclinical evaluation in large animal models, gene therapy for one form of inherited retinal degeneration due to RPE65 deficiency is now being tested in three concurrent clinical trials. Here, we review different approaches for treating inherited retinal degenerations and more common acquired retinal disorders using AAV-based vectors. |
| Databáze: | OpenAIRE |
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