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Article

Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells.

Science translational medicine - 12 Oct 2016

DeWitt Mark A, Magis Wendy, Bray Nicolas L, Wang Tianjiao, Berman Jennifer R, Urbinati Fabrizia, Heo Seok-Jin, Mitros Therese, Muñoz Denise P, Boffelli Dario, Kohn Donald B, Walters Mark C, Carroll Dana, Martin David I K, Corn Jacob E

Abstract excerpt

Genetic diseases of blood cells are prime candidates for treatment through ex vivo gene editing of CD34+ hematopoietic stem/progenitor cells (HSPCs), and a variety of technologies have been proposed to treat these disorders. Sickle cell disease (SCD) is a recessive genetic disorder caused by a single-nucleotide polymorphism in the β-globin gene (HBB). Sickle hemoglobin damages erythrocytes, causing vasoocclusion,...

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Share to Adult Stem Cells
Share to Anemia, Sickle Cell

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Gene Editing

Share to Hemoglobin, Sickle

Share to Heterografts

Share to Mice, Inbred NOD

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Share to Translational Research, Biomedical

Source

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Identifiers

PMID: 27733558
DOI: 10.1126/scitranslmed.aaf9336
PMCID: PMC5500303

Genes

SCD
HBB

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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