| Project/Area Number |
26670168
|
|---|
| Research Category |
Grant-in-Aid for Challenging Exploratory Research
|
| Allocation Type | Multi-year Fund |
|---|
| Research Field |
Human genetics
|
|---|
| Research Institution | Kyoto University |
|---|
Principal Investigator |
|
|---|
| Project Period (FY) |
2014-04-01 – 2016-03-31
|
| Project Status |
Completed (Fiscal Year 2015)
|
| Budget Amount *help |
¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
|
|---|
| Keywords | ゲノム編集 / 遺伝子治療 / CRISPR / TALEN / 血液幹細胞 / CRISPR法 / TALEN法 |
|---|
| Outline of Final Research Achievements |
The Cas9 nucleases from the Streptococcus pyogenes, directed by a chimeric guide RNA (sgRNA) to any genomic locus followed by a 5’-NGG protospaceradjacent motif (PAM), are robust tools for inducing targeted double-stranded DNA breaks (DSBs) in human cells. The RNA-guided endonuclease (RGEN) was applied for human T cells, and then the system created recombinant cells. DSBs induce homology-directed repair (HDR). From the HDR pathway, site-directed insertion (knock-in, KI) was induced by provision of homologous donor construct and the RGENs significantly promote efficient its site-specific insertion in PSIP1 gene. We created to develop an efficient homozygous KI system via fluorescent gene transduction. We also developed an efficient TALEN-genome editing system to remove HIV proviral DNA.
|
