Therapeutic Applications of Nucleic Acid Based Technologies




  • To understand the fundamental concepts of RNA biogenesis and metabolism in eukaryotes
  • To understand the concept of gene silencing; To identify the main characteristics of viral vectors for gene therapy
  • To understand the concept of RNA dominant human diseases
  • To know the main methods of delivery of nucleic acids into eukaryotic cells
  • To understand the main working principles, advantages and limitations, of diverse technologies used to investigate nucleic acid-protein interactions
  • To understand the basic principles of nucleic acid hybridization techniques with special emphasis on microarrays and its implementation in personalized medicine
  • To know the basic principles of RNA seq technology and its implementation in personalized medicine
  • To be able to perform the following techniques: Nucleic acid transfection, preparation of cellular extracts, cDNA synthesis, PCR, and Western blot

General characterization





Responsible teacher

Celso Vladimiro Ferreira de Abreu Cunha


Weekly - 3

Total - Available soon

Teaching language



The frequency of 2/3 of all classes is mandatory.


Selected papers

O’Driscoll CM et al. (2019) Oral delivery of non-viral nucleic acid-based therapeutics - do we have the guts for this? European Journal of Pharmaceutical Sciences Volume 133, 15 May 2019, Pages 190-204

 Nakamori, M. et al. Nucleic Acid–Based Therapeutics for Parkinson’s Disease  Neurotherapeutics (2019) 16: 287.

 Watts, J.K. Nucleic Acid Therapeutics for Neurological Diseases   Neurotherapeutics (2019) 16: 245.

 Chen, al. Nucleic Acid-Based Therapeutics for Pulmonary Diseases AAPS PharmSciTech (2018) 19: 3670.



Advances in Nucleic Acid Therapeutics (2019). Editors: Sudhir Agrawal, Michael J Gait. Royal Society of Chemistry. ISBN-13: 978-1788012096


Advances in Nanomedicine for the Delivery of Therapeutic Nucleic Acids (2017). Edited by: Surendra Nimesh, Ramesh Chandra and Nidhi Gupta. Woodhead Publishing. ISBN 978-0-08-100557-6

Teaching method

Available soon

Evaluation method

Available soon

Subject matter

Theoretical programme: Fundamental concepts of RNA biology in eukaryotes. Methodologies and strategies for nucleic acid transfection. siRNA and shRNA for gene silencing. Gene silencing-based therapies. Viral vectors. Methods to investigate nucleic acid-protein interactions. Immunoprecipitation, pull-down, Affinity purification with MS2 aptamers and tobramicin, pull-down of methylated RNA, RADAR chromatography, CLIP.  Microarray analysis and RNA seq for diagnosis in personalyzed medicine.

Practical programme: In vitro culture of eukaryotic cells. Transfection of eukaryotic cells with siRNAs. Preparation of total cellular extracts from transfected cells. Total RNA purification from transfected cells. cDNA synthesis and PCR. Analysis of PCR amplified products by agarose gel electrophoresis. SDS-PAGE and Western blot for analysis of total cellular extracts. Protein transfer to nitrocellulose membranes. Detection of proteins with specific antibodies.


Programs where the course is taught: