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Advanced filters: Author: Alec B Wilkens Clear advanced filters

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

These preclinical studies outline a CRISPR-based methodology for correcting β-globin gene mutations in haematopoietic stem cells to advance the development of next-generation therapies for β-haemoglobinopathies.

Daniel P. Dever
Rasmus O. Bak
Matthew H. Porteus
Research07 Nov 2016
Nature

Volume: 539, P: 384-389
Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

Improved efficiency of CRISPR/Cas editing with chemically modified synthetic sgRNAs.

Ayal Hendel
Rasmus O Bak
Matthew H Porteus
Research01 Sept 2015
Nature Biotechnology

Volume: 33, P: 985-989

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CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

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