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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
Series
Accession:
GSE131387
ID:
200131387

PubMed Full text in PMC Similar studies Analyze with GEO2RSRA Run Selector

2.

Illumina HiSeq 3000 (Homo sapiens)

Organism:
Homo sapiens
1140 Series
28276 Samples
Download data
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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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

SRA Run Selector

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