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Series GSE84534 Query DataSets for GSE84534
Status Public on Jul 29, 2016
Title A multi-functional AAV-CRISPR-Cas9 and its host response
Organism Mus musculus
Experiment type Expression profiling by high throughput sequencing
Summary CRISPR-Cas9 delivery by AAV holds promise for gene therapy but faces critical barriers due to its potential immunogenicity and limited payload capacity. Here, we demonstrate genome engineering in postnatal mice using AAV-split-Cas9, a multi-functional platform customizable for genome-editing, transcriptional regulation, and other previously impracticable AAV-CRISPR-Cas9 applications. We identify crucial parameters that impact efficacy and clinical translation of our platform, including viral biodistribution, editing efficiencies in various organs, antigenicity, immunological reactions, and physiological outcomes. These results reveal that AAV-CRISPR-Cas9 evokes host responses with distinct cellular and molecular signatures, but unlike alternative delivery methods, does not induce detectable cellular damage in vivo. Our study provides a foundation for developing effective genome therapeutics
Overall design mRNA-Seq from muscles (9 samples; 3 mice x 3 conditions) and lymph nodes (9 samples; 3 mice x 3 conditions).
Contributor(s) Chew WL, Ng AH
Citation(s) 27595405
Submission date Jul 18, 2016
Last update date May 15, 2019
Contact name Alex Ng
Organization name Harvard Medical School
Department Genetics
Lab Church
Street address 77 Avenue Louis Pasteur Rm 233
City Boston
State/province MA
ZIP/Postal code 02115
Country USA
Platforms (1)
GPL19057 Illumina NextSeq 500 (Mus musculus)
Samples (18)
GSM2241596 Muscle TurboRFP (control, biological replicate 1)
GSM2241597 Muscle TurboRFP (control, biological replicate 2)
GSM2241598 Muscle TurboRFP (control, biological replicate 3)
BioProject PRJNA329547
SRA SRP078860

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Supplementary file Size Download File type/resource
GSE84534_Chew2016naturemethods-supptable1.xls.gz 11.5 Mb (ftp)(http) XLS
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Processed data are available on Series record

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