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Evaluation of persistence and fate of <i>ex vivo</i> edited-HSC modified with donor template and Its role in correcting Sickle Cell Disease

2021-07-01

Abstract excerpt

Sickle cell disease (SCD) is caused by a single nucleotide transversion in exon 1 of the HBB gene that changes the hydrophobicity of adult globin (β A ), leading to substantial morbidity and reduced lifespan. Ex vivo autologous gene editing utilizing co-delivery of a designer nuclease along with a DNA donor template allows for precise homology-directed repair (HDR). These gene corrected cells when engrafted int...

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Literature Corpus work
e723a7d8-cdaf-5bf0-91a0-a4a436b09b7a
DOI
10.1101/2021.06.30.450644
Open publication

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Evaluation of persistence and fate of <i>ex vivo</i> edited-HSC modified with donor template and Its role in correcting Sickle Cell DiseaseDOI 10.1101/2021.06.30.450644
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