Document Type

Article

Publication Date

1-2022

Publication Title

Front Cell Dev Biol

Keywords

JMG

JAX Source

Front Cell Dev Biol 2022 Jan 3; 9:801819

Volume

9

First Page

801819

Last Page

801819

ISSN

2296-634X

PMID

35047510

DOI

https://doi.org/10.3389/fcell.2021.801819

Grant

NS054154, NS098523, NS105116, OD030187

Abstract

As sequencing technology improves, the identification of new disease-associated genes and new alleles of known genes is rapidly increasing our understanding of the genetic underpinnings of rare diseases, including neuromuscular diseases. However, precisely because these disorders are rare and often heterogeneous, they are difficult to study in patient populations. In parallel, our ability to engineer the genomes of model organisms, such as mice or rats, has gotten increasingly efficient through techniques such as CRISPR/Cas9 genome editing, allowing the creation of precision human disease models. Such

Comments

The authors would like to thank Jane Cha in the JAX Creative service and Emily Spaulding for assistance with Figure 1.

This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY).

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