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Development of a novel assay for modulation of trans-splicing

Diaz Fernández, Paula (2014) MOBN19 20132
Degree Projects in Molecular Biology
Popular Abstract
The information contained in the DNA (Deoxyribonucleic acid) of an organism that will finally be expressed, is first converted into a molecule called RNA (Ribonucleic acid). This is mainly composed of two types of sequences: the exons, carrying the information that will give rise to the formation of proteins and will be finally expressed in the organism, and the introns, which mainly include regulatory signals. The splicing mechanism consist of the joining of the exons together, getting rid of the introns, to form what is called the mature mRNA. This is normally carried out by a complex called the spliceosome. On the other hand, in a less frequent process denominated trans-splicing, exons from different RNA molecules are joined together,... (More)
The information contained in the DNA (Deoxyribonucleic acid) of an organism that will finally be expressed, is first converted into a molecule called RNA (Ribonucleic acid). This is mainly composed of two types of sequences: the exons, carrying the information that will give rise to the formation of proteins and will be finally expressed in the organism, and the introns, which mainly include regulatory signals. The splicing mechanism consist of the joining of the exons together, getting rid of the introns, to form what is called the mature mRNA. This is normally carried out by a complex called the spliceosome. On the other hand, in a less frequent process denominated trans-splicing, exons from different RNA molecules are joined together, leading to the formation of a hybrid mature mRNA. The Spliceosome-mediated RNA trans-splicing (SMaRTTM) technology takes advantage of this process to be used in gene therapy for the correction of genetically linked disorders. Thus, it relies on the design of specific RNA molecules, called pre-trans-splicing molecules (PTMs), which can be delivered to the organism and ideally replace a specific segment of RNA which contains a mutation and would otherwise lead to the development of a disease. In this way, the information contained in the mature RNA molecule will express the healthy version, preventing the disorder.

In a previous study in the lab, an intron from the Synapsin gene (a gene with an important role in the synapses between neurons) was chosen to look for the best RNA fragments that could act as the binding domain (or the region that keeps the two RNA molecules together in order for trans-splicing to occur) and that will be included in the PTM. In this project the main goal was to validate the feasibility of four different fragments that had been selected according to their trans-splicing efficiencies. The results observed here match the ones expected, although further analyses need to be done. Therefore this supports the possible use of the screening designed in the lab to specifically direct trans-splicing with any purpose.

Supervisor: Tomas Björklund
Master´s Degree Projcet 45 credit in Molecular Genetics 2014
Department of Biology, Lund University (Less)
Please use this url to cite or link to this publication:
author
Diaz Fernández, Paula
supervisor
organization
course
MOBN19 20132
year
type
H2 - Master's Degree (Two Years)
subject
language
English
id
4675602
date added to LUP
2014-09-24 12:37:23
date last changed
2014-10-13 11:17:55
@misc{4675602,
  author       = {{Diaz Fernández, Paula}},
  language     = {{eng}},
  note         = {{Student Paper}},
  title        = {{Development of a novel assay for modulation of trans-splicing}},
  year         = {{2014}},
}