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Items: 1 to 20 of 26

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.

RNP_AAV6_BG_HPRT rep3

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772979
ID:
303772979
4.

RNP_AAV6_BG_HPRT rep2

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772978
ID:
303772978
5.

RNP_AAV6_BG_HPRT rep1

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772977
ID:
303772977
6.

RNP_AAV6_BG rep3

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772976
ID:
303772976
7.

RNP_AAV6_BG rep2

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772975
ID:
303772975
8.

RNP_AAV6_BG rep1

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772974
ID:
303772974
9.

RNP_ssODN rep3

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772973
ID:
303772973
10.

RNP_ssODN rep2

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772972
ID:
303772972
11.

RNP_ssODN rep1

Organism:
Homo sapiens
Source name:
RNP with DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772971
ID:
303772971
12.

AAV6_BG_HPRT rep3

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772970
ID:
303772970
13.

AAV6_BG_HPRT rep2

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772969
ID:
303772969
14.

AAV6_BG_HPRT rep1

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 2
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772968
ID:
303772968
15.

AAV6_BG rep3

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772967
ID:
303772967
16.

AAV6_BG rep2

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772966
ID:
303772966
17.

AAV6_BG rep1

Organism:
Homo sapiens
Source name:
DNA donor template, AAV6 based vector, version 1
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772965
ID:
303772965
18.

ssODN rep3

Organism:
Homo sapiens
Source name:
DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772964
ID:
303772964
19.

ssODN rep2

Organism:
Homo sapiens
Source name:
DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772963
ID:
303772963
20.

ssODN rep1

Organism:
Homo sapiens
Source name:
DNA donor template, single-stranded oligodeoxynucleotides
Platform:
GPL21290
Series:
GSE131387
Download data
Sample
Accession:
GSM3772962
ID:
303772962
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