Title

Design of non-viral vector with improved regulatory features towards therapeutic application

Authors

B. Sharan Sharma^1,2, Vaishna Prabhakaran¹, Ramtej J. Verma^2,*

Affiliation

¹Indrashil Institute of Science & Technology (IIST)/Indrashil University (IU), Kadi, Mehsana – 382740, Gujarat, India; ²Department of Zoology, Biomedical Technology & Human Genetics, University School of Sciences, Gujarat University, Navrangpura, Ahmedabad – 380009, Gujarat, India

Email

Ramtej J. Verma - E-mail: rjvermasosgu@gmail.com; Tel: +91-79-26302362; Fax: +91-79-26303196

Article Type

Research Article

Date

Received February 3, 2020; Accepted April 2, 2020; Published April 30, 2020

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 with 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 control region (DES-LCR). The designed vector, named as DES-LCR/MD1-EGFP, was constructed by cloning two fragments into the pBluescript backbone. Fragment 1 contains DES-LCR enhancer and DES-LCR promoter region while fragment 2 contains MD1 transgene and reporter EGFP (enhanced green fluorescent protein) gene separated by linker P2A (2A peptide). This vector design provides a framework for strong regulation with nonviral features. This design forms the foundation for application in conditions linked to multisystem diseases.

Keywords

DES-LCR, Microdystrophin, DMD, Non-viral vector, Gene therapy

Citation

Sharma et al. Bioinformation 16(4): 307-313 (2020)

Edited by

P Kangueane

ISSN

0973-2063

Publisher

Biomedical Informatics

License

This is an Open Access article which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly credited. This is distributed under the terms of the Creative Commons Attribution License.