Genome editing tool for gene therapy in human hematopoietic stem cells
| 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.
|
Report
(3 results)
Research Products
(21 results)
-
-
-
[Journal Article] Experimental evaluation of the zoonotic infection potency of simian retrovirus type 4 using humanized mouse model2015
Author(s)
Kei Sato, Tomoko Kobayashi, Naoko Misawa, Rokusuke Yoshikawa, Junko S. Takeuchi, Tomoyuki Miura, Munehiro Okamoto, Jun-ichirou Yasunaga, Masao Matsuoka, Mamoru Ito, Takayuki Miyazawa & Yoshio Koyanagi
-
Journal Title
Scientific Reports
Volume: 5
Issue: 1
Pages: 14040-14040
DOI
NAID
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
-
-
[Journal Article] APOBEC3D and APOBEC3F Potently Promote HIV-1 Diversification and Evolution in Humanized Mouse Model2014
Author(s)
Sato, K. Takeuchi, J. S. Misawa, N. Izumi, T. Kobayashi, T. Kimura, Y. Iwami, S. Takaori-Kondo, A. Hu, W. S. Aihara, K. Ito, M. An, D. S. Pathak, V. K. Koyanagi, Y.
-
Journal Title
PLoS Pathog
Volume: 10
Issue: 10
Pages: e1004453-e1004453
DOI
Related Report
Peer Reviewed / Open Access / Acknowledgement Compliant
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
-
