Publication:
Generation of four H1 hESC sublines carrying a hemizygous knock-out/mutant MECP2

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SCHOOL OF MEDICINE
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Zeng, Ruizhu
Sidik, Harwin
Robinson, Kim S.
Zhong, Franklin L.
Pouladiahi, Mahmoud A.

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NO

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Abstract

Rett syndrome (RTT) is a childhood neurodevelopmental disorder caused by mutations in MECP2. To study the molecular mechanisms underlying RTT, four sublines of H1 hESCs were generated, carrying a hemizygous knockout or mutant allele of MECP2. Exons 3 and 4 of MECP2 were targeted using the CRISPR/Cas9 nuclease system.

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Elsevier

Subject

Medicine

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Stem Cell Research

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DOI

10.1016/j.scr.2019.101533

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GoalOpen Access
03 - Good Health and Well-being
Over the last 15 years, the number of childhood deaths has been cut in half. This proves that it is possible to win the fight against almost every disease. Still, we are spending an astonishing amount of money and resources on treating illnesses that are surprisingly easy to prevent. The new goal for worldwide Good Health promotes healthy lifestyles, preventive measures and modern, efficient healthcare for everyone.

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