Collection-image

TOPICAL COLLECTIONS

Applications of Targeted Genome Editing Technology in Disease Modelling

Submit Abstract

Guest Editor

Sean Xin-Xin Zeng

Sean Xin-Xin Zeng

Sanford Burnham Prebys Medical Discovery Institute

Dr. Sean Xin-Xin Zeng’s research utilizes sophisticated vertebrate animal models, live-imaging techniques, genetic approaches, and pharmacological reagents to elucidate the cellular and molecular mechanisms of organ development, tissue formation, and related human diseases. His scientific career is devoted to understanding how progenitor cells contribute to organogenesis during embryonic development, and how to leverage vertebrate systems to model human genetic disorders.

Collection Overview

Advancements in the targeted genome editing field have opened promising curable doors to human genetic disorders, largely implemented by modifying faulty DNA sequences. Specifically, a handful of programmable nucleases producing site-specific DNA double-strand breaks, such as ZFNs, TALENs, and celebrated CRISPR)/Cas nucleases, have been popularly studied, rapidly evolved, and sophistically adopted. During the last decade, the scientific community, plus the public, have vividly witnessed the maturation of gene editing from concept to basic research, and towards clinical practice.

This collection intends to recruit methods and protocols, which can sufficiently showcase recent progress in developing above mentioned technologies, and particularly their application for disease modelling. For instance, the aim is to highlight detailed technical nuances that have been utilized in a wide variety of cell types and model organisms, to edit single base pair or multiple loci, in vitro and in vivo. A discussion on gRNA design strategy and its modifications, which are essentially associated with editing and delivery efficiency could also be included.

Articles

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes
7:17

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes

0 Views

•

Cited by 2

•

2022

•