Ontology highlight
ABSTRACT:
SUBMITTER: Sharma BS
PROVIDER: S-EPMC7392091 | biostudies-literature | 2020
REPOSITORIES: biostudies-literature
Bioinformation 20200430 4
Viral vectors based gene therapy is often compromised by adverse immunological reactions raising safety concerns. Hence, improved design and development of non-viral vectors with strong regulatory regions is desired. We describe the design of a non-viral mammalian expression vector in which the primary transgene (a truncated dystrophin gene linked with Duchenne muscular dystrophy (DMD)) named microdystrophin delR4-R23/delCT (MD1) is under the transcriptional control of elements of desmin locus c ...[more]