期刊
JOURNAL OF CLINICAL MEDICINE
卷 11, 期 16, 页码 -出版社
MDPI
DOI: 10.3390/jcm11164717
关键词
retinal gene therapy; subretinal injection; subretinal injection technique; vitreoretinal surgery
资金
- National Institute of Health Carlos III
- European Regional Development Fund (Institute of Health Carlos III/ISCIII) [PI20/01186]
- Retinitis Pigmentosa Patients of Gipuzkoa Foundation (BEGISARE)
- Torres Quevedo Research Fellowship from the Spanish Ministry of Science and Innovation [PTQ2019-010412]
- Spanish Ministry of Science and Innovation [DIN2020-011302]
Inherited retinal dystrophies (IRDs) affect a significant number of people worldwide, and gene therapy has shown potential in improving visual function for these patients. Subretinal injection is becoming popular among researchers and clinicians as a method to effectively deliver viral vectors for gene therapy. This review aims to provide insight into current subretinal injection techniques and understand the key factors for success in this procedure.
Inherited retinal dystrophies (IRDs) affect an estimated 1 in every 2000 people, this corresponding to nearly 2 million cases worldwide. Currently, 270 genes have been associated with IRDs, most of them altering the function of photoreceptors and retinal pigment epithelium. Gene therapy has been proposed as a potential tool for improving visual function in these patients. Clinical trials in animal models and humans have been successful in various types of IRDs. Recently, voretigene neparvovec (Luxturna (R)) has been approved by the US Food and Drug Administration for the treatment of biallelic mutations in the RPE65 gene. The current state of the art in gene therapy involves the delivery of various types of viral vectors into the subretinal space to effectively transduce diseased photoreceptors and retinal pigment epithelium. For this, subretinal injection is becoming increasingly popular among researchers and clinicians. To date, several approaches for subretinal injection have been described in the scientific literature, all of them effective in accessing the subretinal space. The growth and development of gene therapy give rise to the need for a standardized procedure for subretinal injection that ensures the efficacy and safety of this new approach to drug delivery. The goal of this review is to offer an insight into the current subretinal injection techniques and understand the key factors in the success of this procedure.
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