2023 Fiscal Year Annual Research Report
Full-length dystrophin restoration by in vivo RNA editing via a minidCas13X.1 conjugated ADAR
| Project/Area Number |
22KF0410
|
|---|
| Allocation Type | Multi-year Fund |
|---|
| Research Institution | National Center of Neurology and Psychiatry |
|---|
Principal Investigator |
青木 吉嗣 国立研究開発法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 部長 (80534172)
|
|---|
| Co-Investigator(Kenkyū-buntansha) |
SAIFULLAH SAIFULLAH 国立研究開発法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 外国人特別研究員
|
|---|
| Project Period (FY) |
2023-03-08 – 2024-03-31
|
| Keywords | RNA編集 / 筋ジストロフィー |
|---|
| Outline of Annual Research Achievements |
We changed nonsense codon (TAA) into sense codon (TGG) by using minidCas13X.1 and ADAR editor to the target site of the DMD transcript in a sequence-specific manner with the help of complementary guide RNAs. As a progress, we have made seven retroviral vectors (four guide RNAs vectors, two ADAR vectors with minidCas13X.1 and MS2 protein, one EGFP) and confirmed by sanger sequencing. To confirm the expression and retroviral packaging, we transduce the EGFP vectors into h2kmdx23 primary myoblast cells isolated from mdx23 mouse. The 70% cells have shown EGFP florescence according to microscopical observation. Which indicated that viral vector construction and packaging are good enough for next experiment. However, we have not yet checked the editing event in the cellular system.
|
Research Products
(1 results)
