Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy
Joint Authors
Sanjurjo-Soriano, Carla
Kalatzis, Vasiliki
Source
Issue
Vol. 2018, Issue 2018 (31 Dec. 2018), pp.1-15, 15 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2018-05-08
Country of Publication
Egypt
No. of Pages
15
Main Subjects
Abstract EN
Inherited retinal dystrophies (IRDs) are a leading cause of visual impairment in the developing world.
These conditions present an irreversible dysfunction or loss of neural retinal cells, which significantly impacts quality of life.
Due to the anatomical accessibility and immunoprivileged status of the eye, ophthalmological research has been at the forefront of innovative and advanced gene- and cell-based therapies, both of which represent great potential as therapeutic treatments for IRD patients.
However, due to a genetic and clinical heterogeneity, certain IRDs are not candidates for these approaches.
New advances in the field of genome editing using Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) have provided an accurate and efficient way to edit the human genome and represent an appealing alternative for treating IRDs.
We provide a brief update on current gene augmentation therapies for retinal dystrophies.
Furthermore, we discuss recent advances in the field of genome editing and stem cell technologies, which together enable precise and personalized therapies for patients.
Lastly, we highlight current technological limitations and barriers that need to be overcome before this technology can become a viable treatment option for patients.
American Psychological Association (APA)
Sanjurjo-Soriano, Carla& Kalatzis, Vasiliki. 2018. Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy. Neural Plasticity،Vol. 2018, no. 2018, pp.1-15.
https://search.emarefa.net/detail/BIM-1210130
Modern Language Association (MLA)
Sanjurjo-Soriano, Carla& Kalatzis, Vasiliki. Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy. Neural Plasticity No. 2018 (2018), pp.1-15.
https://search.emarefa.net/detail/BIM-1210130
American Medical Association (AMA)
Sanjurjo-Soriano, Carla& Kalatzis, Vasiliki. Guiding Lights in Genome Editing for Inherited Retinal Disorders: Implications for Gene and Cell Therapy. Neural Plasticity. 2018. Vol. 2018, no. 2018, pp.1-15.
https://search.emarefa.net/detail/BIM-1210130
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1210130