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Items: 3

1.

Editing the Sickle Cell Disease Mutation in Primary Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates

(Submitter supplied) Site-specific correction of a point mutation causing a monogenic disease in autologous hematopoietic stem and progenitor cells (HSPCs) can be used as a treatment of inherited disorders of the blood cells. Sickle Cell Disease (SCD) is an ideal model to investigate the potential use of gene editing to transvert a single point mutation at the β-globin locus (HBB). We compared the activity of ZFNs and CRISPR/Cas9 for editing and homologous donor templates delivered as single-stranded oligodeoxynucleotides (ssODN), adeno-associated virus serotype 6 (AAV6), integrase-deficient lentiviral vectors (IDLV) and adenovirus 5/35 serotype (Ad5/35) to transvert the base-pair responsible for SCD in HBB in primary human CD34+ HSPCs. more...
Organism:
Homo sapiens
Type:
Expression profiling by high throughput sequencing
Platform:
GPL21290
24 Samples
Download data: TXT
2.

Illumina HiSeq 3000 (Homo sapiens)

Platform
Accession:
GPL21290
ID:
100021290
3.

Mock rep3

Organism:
Homo sapiens
Source name:
Mock control
Platform:
GPL21290
Series:
GSE131387
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Sample
Accession:
GSM3772958
ID:
303772958
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db=gds|term=GSM3772958[Accession]|query=1|qty=2|blobid=MCID_66284dc9f4364867151dc78a|ismultiple=true|min_list=5|max_list=20|def_tree=20|def_list=|def_view=|url=/Taxonomy/backend/subset.cgi?|trace_url=/stat?
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