Gene Therapy for Inherited Retinal Disorders: Update on Clinical Trials.
Stylianos MichalakisMaximilian GerhardtGünter RudolphSiegfried PriglingerClaudia PriglingerPublished in: Klinische Monatsblatter fur Augenheilkunde (2021)
Within the last decade, continuous advances in molecular biological techniques have made it possible to develop causative therapies for inherited retinal disorders (IRDs). Some of the most promising options are gene-specific approaches using adeno-associated virus-based vectors to express a healthy copy of the disease-causing gene in affected cells of a patient. This concept of gene supplementation therapy is already advocated for the treatment of retinal dystrophy in RPE65-linked Leber's congenital amaurosis (LCA) patients. While the concept of gene supplementation therapy can be applied to treat autosomal recessive and X-linked forms of IRD, it is not sufficient for autosomal dominant IRDs, where the pathogenic gene product needs to be removed. Therefore, for autosomal dominant IRDs, alternative approaches that utilize CRISPR/Cas9 or antisense oligonucleotides to edit or deplete the mutant allele or gene product are needed. In recent years, research retinal gene therapy has intensified and promising approaches for various forms of IRD are currently in preclinical and clinical development. This review article provides an overview of current clinical trials for the treatment of IRDs.
Keyphrases
- gene therapy
- clinical trial
- copy number
- genome wide
- optical coherence tomography
- crispr cas
- diabetic retinopathy
- genome wide identification
- end stage renal disease
- chronic kidney disease
- randomized controlled trial
- cell death
- stem cells
- gene expression
- ejection fraction
- early onset
- mesenchymal stem cells
- oxidative stress
- signaling pathway
- genome editing
- prognostic factors
- peritoneal dialysis
- phase ii
- open label
- duchenne muscular dystrophy