Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.
Galaxy Y ChoKyle BoloKaren Sophia ParkJesse D SengilloKaren Sophia ParkPublished in: Molecular diagnosis & therapy (2019)
Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.
Keyphrases
- gene therapy
- clinical trial
- optical coherence tomography
- diabetic retinopathy
- optic nerve
- current status
- minimally invasive
- genome wide
- multiple sclerosis
- risk factors
- phase ii
- coronary artery bypass
- oxidative stress
- systematic review
- randomized controlled trial
- open label
- double blind
- gene expression
- single molecule
- machine learning
- phase iii
- transcription factor
- atrial fibrillation
- surgical site infection