Applications of CRISPR/Cas9 in retinal degenerative diseases

Ying Qian Peng, Luo Sheng Tang, Shigeo Yoshida, Ye Di Zhou

Research output: Contribution to journalArticle

12 Citations (Scopus)

Abstract

• Gene therapy is a potentially effective treatment for retinal degenerative diseases. Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) system has been developed as a new genome-editing tool in ophthalmic studies. Recent advances in researches showed that CRISPR/Cas9 has been applied in generating animal models as well as gene therapy in vivo of retinitis pigmentosa (RP) and leber congenital amaurosis (LCA). It has also been shown as a potential attempt for clinic by combining with other technologies such as adeno-associated virus (AAV) and induced pluripotent stem cells (iPSCs). In this review, we highlight the main points of further prospect of using CRISPR/Cas9 in targeting retinal degeneration. We also emphasize the potential applications of this technique in treating retinal degenerative diseases.

Original languageEnglish
Pages (from-to)646-651
Number of pages6
JournalInternational Journal of Ophthalmology
Volume10
Issue number4
DOIs
Publication statusPublished - Apr 18 2017

Fingerprint

CRISPR-Associated Proteins
Clustered Regularly Interspaced Short Palindromic Repeats
Retinal Diseases
Genetic Therapy
Leber Congenital Amaurosis
Induced Pluripotent Stem Cells
Dependovirus
Retinal Degeneration
Retinitis Pigmentosa
Animal Models
Technology
Research
Proteins

All Science Journal Classification (ASJC) codes

  • Ophthalmology

Cite this

Applications of CRISPR/Cas9 in retinal degenerative diseases. / Peng, Ying Qian; Tang, Luo Sheng; Yoshida, Shigeo; Zhou, Ye Di.

In: International Journal of Ophthalmology, Vol. 10, No. 4, 18.04.2017, p. 646-651.

Research output: Contribution to journalArticle

Peng, Ying Qian ; Tang, Luo Sheng ; Yoshida, Shigeo ; Zhou, Ye Di. / Applications of CRISPR/Cas9 in retinal degenerative diseases. In: International Journal of Ophthalmology. 2017 ; Vol. 10, No. 4. pp. 646-651.
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