Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics

(Dana P.) #1

© Springer International Publishing AG 2017 91
S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering,
Advances in Experimental Medicine and Biology 1016,
DOI 10.1007/978-3-319-63904-8_5


Chapter 5

Genome Editing to Study Ca


2+

Homeostasis


in Zebrafish Cone Photoreceptors


Susan E. Brockerhoff


Abstract Photoreceptors are specialized sensory neurons with unique biological


features. Phototransduction is well understood due in part to the exclusive expres-


sion and function of the molecular components of this cascade. Many other pro-


cesses are less well understood, but also extremely important for understanding


photoreceptor function and for treating disease. One example is the role of Ca2+ in


the cell body and overall compartmentalization and regulation of Ca2+ within the


cell. The recent development of CRISPR/Cas9 genome editing techniques has made


it possible to rapidly and cheaply alter specific genes. This will help to define the


biological function of elusive processes that have been more challenging to study.


CRISPR/Cas9 has been optimized in many systems including zebrafish, which


already has some distinct advantages for studying photoreceptor biology and func-


tion. These new genome editing technologies and the continued use of the zebrafish


model system will help advance our understanding of important understudied


aspects of photoreceptor biology.


Keywords Zebrafish • Photoreceptors • Mitochondria • Genome editing • Calcium


5.1 Introduction

This review briefly summarizes the importance of studying Ca2+ homeostasis in


photoreceptors and how newly developed genome editing strategies in zebrafish can


be used to help answer outstanding questions in photoreceptor biology.


S.E. Brockerhoff, Ph.D. (*)
Departments of Biochemistry and Ophthalmology, University of Washington,
UW Medicine, 750 Republican St, Box 358058, Seattle, WA 98109, USA
e-mail: [email protected]

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