Article

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I

2018-09-04

Abstract excerpt

<h4>Summary</h4> Lysosomal enzyme deficiencies comprise a large group of genetic disorders that generally lack effective treatments. A potential treatment approach is to engineer the patient’s own hematopoietic system to express high levels of the deficient enzyme, thereby correcting the biochemical defect and halting disease progression. Here, we present an efficient ex vivo genome editing approach using CRISPR...

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Literature Corpus work: d8d92f47-506b-5a0c-8bdf-70b6aef6074c
DOI: 10.1101/408757

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