Inside Salk Winter 2017
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Professor Juan Carlos Izpisua Belmonte, co–first author JunWu and collaborators

have corrected a disease-causing mutation in early stage human embryos. The

technique, which uses the CRISPR-Cas9 system, corrected the mutation for a

common heart condition called hypertrophic cardiomyopathy at the earliest stage

of embryonic development so that the defect would not be passed on to future

generations. The scientists were surprised by just how safe and efficient the method

was. Not only did a high percentage of embryonic cells get repaired, but also gene

correction didn’t induce any detectable off-target mutations and genome instability—

major concerns for gene editing. In addition, the researchers developed a robust

strategy to ensure the repair occurred consistently in all the cells of the embryo.

Early gene-editing success holds promise

for preventing inherited diseases

NATURE

08/2017

NATURE

09/2017

Is it better to do a task quickly and make mistakes, or to do it slowly but perfectly? When

it comes to deciding how to fix breaks in DNA, cells face the same choice between two

major repair pathways. The decision matters, because the wrong choice could cause even

more DNA damage and lead to cancer. Professor Jan Karlseder and first author Nausica

Arnoult found that a tiny protein called CYREN helps cells choose the right pathway at

the right time, clarifying a longstanding mystery about DNA repair and offering

researchers a powerful tool that could guide better treatments for cancer.

The right way to repair DNA

2 INSIDE SALK

WINTER 2017

WWW.SALK.EDU

The science of DNA organization and repair

DISCOVERIES

STORE AND

WATCH

bit.ly/belmonte201712