Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated protein (Cas) is used by some bacteria and most archaea to protect against viral phage intrusion and has recently been adapted to allow for efficient editing of the mammalian genome. Whilst CRISPR/Cas-based technology has been used to modify genes in mammalian cells in vitro, delivery of CRISPR/Cas system into mammalian tissue and/or organs is more difficult and often requires additional vectors. With the use of adeno-associated virus (AAV) gene delivery system, active CRISPR/Cas enzyme can be maintained for an extended period of time and enable efficient editing of genome in the retina in vivo. Herein we outline the method to edit the genome in mouse retina using a dual AAV vector-mediated CRISPR/Cas9 system.

Item Type:	Other Book Chapter
Keywords:	CRISPR/Cas9, genome editing, gene delivery, AAV, retina
Research Division:	Technology
Research Group:	Medical Biotechnology
Research Field:	Gene and Molecular Therapy
Objective Division:	Health
Objective Group:	Clinical Health (Organs, Diseases and Abnormal Conditions)
Objective Field:	Hearing, Vision, Speech and Their Disorders
UTAS Author:	Li, F (Dr Fan Li)
UTAS Author:	King, AE (Professor Anna King)
UTAS Author:	Liu, G (Dr Guei-Sheung Liu)
ID Code:	127341
Year Published:	2018
Deposited By:	Menzies Institute for Medical Research
Deposited On:	2018-07-23
Last Modified:	2019-02-21
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