| Types | DnaRegion
|
| Roles | Plasmid
plasmid
|
| Sequences | BBa_M39111_sequence (Version 1)
|
Description
The general purpose of our part is to help create a modified Semliki Forest Virus that contains micro-dystrophin mRNA in its capsid, which hopefully will be used to deliver said mRNA to muscle cells of muscular dystrophy patients. This DNA vector contains an RNA polymerase promoter, the packaging sequence from SFV (Semliki Forest Virus), the DNA sequence for micro-dystrophin, and an RNA polymerase terminator (Bba_B1006). Once this vector is transcribed into RNA in vitro, the resultant RNA is introduced into a cell in the lab. A separate helper vector (not included in this part) will be introduced into the same cell as well. This other vector will simply contain the genes for the structural proteins of SFV. Once the helper vector is translated to produce the structural capsid proteins, the virus will assemble within the cell, packaging the micro-dystrophin mRNA within the capsid of the virus. The purpose of removing the non-structural genes (other than the necessary packaging sequence) is to remove the replicating and virulent ability of the virus, so that it will not replicate within the body. This virus will then break free of the cell in which it was produced, at which point it can be harvested and injected into patients to treat dystrophin deficiency within muscle cells.
Notes
We had to make sure only to include the packaging sequences of SFV, so that it would not be able to replicate within the patient's cells. This should also reduce the virulent capabilities of the virus.
Source
This part came from a combination of a portion of the SFV genome, in addition to the micro-dystrophin genomic sequence and standard promoters and terminator.
