Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retinitis Pigmentosa Model

Sally Justus; Andrew Zheng; Yi-Ting Tsai; Wen-Hsuan Wu; Chun-Wei Hsu; Wei-Pu Wu; Alexander G Bassuk; Vinit B Mahajan; Stephen H Tsang

doi:10.1007/978-3-319-34148-4_9

Book chapter

Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retinitis Pigmentosa Model

Sally Justus, Andrew Zheng, Yi-Ting Tsai, Wen-Hsuan Wu, Chun-Wei Hsu, Wei-Pu Wu, Alexander G Bassuk, Vinit B Mahajan and Stephen H Tsang

Genome Editing, pp.149-162

Springer International Publishing

08/10/2016

DOI: 10.1007/978-3-319-34148-4_9

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Abstract

The future of precision medicine, genome editing has gained momentum in the field of ophthalmology because of the eye’s amenability to genetic interventions. The eye is an ideal target for gene therapy due to its accessibility, ease of noninvasive monitoring, significant compartmentalization, immunoprivileged status, optical transparency, and the presence of a contralateral control. One of the first gene therapy clinical trials was conducted in the eye for a severe form of early-onset retinal dystrophy called Leber congenital amaurosis, and it has encouraged further exploration of this technique as a viable treatment option for other inherited disorders across medical disciplines. This chapter highlights current ocular gene therapy approaches, clinical and preclinical experiments, and provides a case study of the bench-to-bedside personalized medicine approach taken for a novel and rare retinitis pigmentosa mutation.

Gene repair

D190N mutation

Gene therapy

Retinitis pigmentosa

Gene supplementation

Details

Title: Subtitle: Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retinitis Pigmentosa Model
Creators: Sally Justus - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Andrew Zheng - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Yi-Ting Tsai - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Wen-Hsuan Wu - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Chun-Wei Hsu - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Wei-Pu Wu - Department of Ophthalmology, College of Physicians and Surgeons, Columbia University, New York, USA
Alexander G Bassuk - Department of Pediatrics, The University of Iowa Children’s Hospital, Iowa City, USA
Vinit B Mahajan - Department of Ophthalmology, Iowa University Hospitals and Clinics, Iowa City, USA
Stephen H Tsang - Edward S. Harkness Eye Institute, New York-Presbyterian Hospital/Columbia University Medical Center, New York, USA
Resource Type: Book chapter
Publication Details: Genome Editing, pp.149-162
DOI: 10.1007/978-3-319-34148-4_9
Publisher: Springer International Publishing; Cham
Language: English
Date published: 08/10/2016
Academic Unit: Neurology; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Neurology (Pediatrics)
Record Identifier: 9984070491702771

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