Detection of the R117H Mutation in the Human CFTR Genome for Mild Cystic Fibrosis Utilizing Allele Specific PCR

 

 

 

 

 

 

 

 

 

 

 

By: Zoe Buszka, Kalin Bayes, and Danielle Guist

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

LB 145 Cell and Molecular Biology

Lab Time:Tuesday and Thursday 10:20-12:10pm

Matt VanDer and Allison Vlk

 10/10/2017

 

WEBSITE

 

Title page written by: B945

Revised by: B980

Finalized by B925

Abstract

Written by: B980

Revised by: B925

Finalized by: B945

 

 

On the seventh chromosome of the human genome resides a mutation in the CFTR gene known as R117H; at the 117th amino acid a guanine is altered to an adenine. It has been found that 4 of 18 cystic fibrosis patients tested show signs of having this mutation (Gervais et al, 1993), however it is not routinely screened for. Developing an accurate, inexpensive test for this mutation allows for individuals to be tested for the mutation of interest and treated quickly. Polymerase chain reaction was used to amplify the target DNA, containing the R117H mutation, with designed primers.It was hypothesized that the designed primers for the R117H mutation that causes cystic fibrosis will bind to the nucleotides, at 56.5¼C, of the mutant DNA, but not to the wild type DNA. The resulting amplified DNA was then analyzed using agarose gel electrophoresis. The agarose gel showed three band lengths: one of 212 base pairs and one of 432 base pairs for DNA containing the R117H mutation, while one band of 307 base pairs was present containing the wild-type DNA. This allowed for the identification of an individual who possesses this specific mutation because the primers will bind to and replicate the mutant DNA. We predicted that three designed primers would detect the R117H mutation that leads to cystic fibrosis through genetic analysis because PCR is an effective method at amplifying target DNA for genetic mutations (Balogh et al 2004).

 

 

 

 

 

 

 

Discussion

Written by: B980

Revised by: B925

Finalized by B945

 

 

Experimental Summary

Cystic fibrosis is a recessive genetic mutation that causes the CFTR chloride channels within cells to operate poorly, if at all (Ying-chun, 2016). Patients with cystic fibrosis often have large amounts of secretions coming from their lungs, pancreas, and intestines that create a perfect environment for bacteria to culture, in addition to causing breathing difficulties (Rowe et al., 2005). At the 117th amino acid of the CFTR gene a guanine is altered to an adenine, which results in the R117H mutation. The R117H mutation can either extend the opening or closing time of the chloride channels present in the CFTR gene. We are performing this experiment to answer the question of whether or not a PCR test will be able to amplify DNA samples well enough to determine if an individual has the R117H mutation early on or if the individual is a carrier of the gene. We hypothesize that the designed primers for the R117H mutation that causes Cystic Fibrosis will bind to the nucleotides of the targeted mutation but not in wild type DNA because the primers were created correctly and the annealing temperature was carefully calculated. This will allow the mutation to become two visible bands when ran through a gel electrophoresis and causing the wild type DNA to form a single band.

It would be beneficial to create an assay that tests for the top 10 most common mutations causing cystic fibrosis. This can be done by creating a set of primers that amplify most of the mutations linked to cystic fibrosis to be further analyzed through restriction enzymes which cuts at different points in DNA that contains the mutations. This would provide the different band widths to identify individual mutations of cystic fibrosis in the human genome. This would be a beneficial test that would tell parents if they were positive for one of the mutations and how likely it would be for a coupleÕs child to develop cystic fibrosis if they decided to have one.

Original Predictions

The results of the experiment would have shown that the designed primers did successfully bind to the DNA strand and amplified the target DNA because there was a band present of 212 base pairs and 432 base pairs, in the agarose gel, that would be compatible to the R117H mutation band in the control experiment (Shrimpton, 2012). This means that our data supported our hypothesis and that our results would have aligned with previously published research. PCR is a common method for replicating DNA and assisting in performing genetic screenings successfully for point specific mutations. Our research was performed with the same basic steps as other published papers; the major difference was that we designed our primers to include DNA bands with longer lengths so they would be more prominent in gel electrophoresis.

While similar to other published papers, this research proves it is possible for the R117H mutation to be detected in patients if they have a genetic screening performed on them. It is rare that R117H is tested for due to the disease being unrecognized by doctors until significant symptoms become prevalent, however it should be because the frequency in those with cystic fibrosis is high (Gervais et al, 1993). These experimental results indicate that the assay should be performed successfully with no harm to the patient and no hassle with the analysis. This test can also help expecting parents to test their genomes for the R117H genetic mutation as early as possible (Malone et al, 1999).

Results and Ultimate Findings

In order to determine optimal PCR conditions for the R117H mutation, multiple experimental trials were run with adjustments in DNA concentration and magnesium sulfate concentration. In addition, these experimental trials were run to establish the optimal annealing temperature, which can directly affect the annealing rates of the published and designed primers. The optimal annealing temperature for our published primers was determined to be 56.5 ¼C (see Methods section). The optimal concentration of magnesium sulfate was 1.5 ul. Taq polymerase is a magnesium-dependent enzyme, so determining the optimum concentration to use was critical for the success of the PCR reaction (Markoulatos, 2002). After the optimal PCR conditions were determined, a clearly defined band of 307 base pairs long that contained the published primers for the wild-type DNA and a clearly defined 1 Kb plus ladder were produced. This is in support of our hypothesis that that the published primers would anneal properly to the wild-type DNA to form a single band of 307 base pairs.


 

 

Future Directions

If research was to resume, the PCR assay containing mutant DNA would be performed. The designed primers would be ordered and the mutant DNA obtained containing the R117H mutation. The optimal DNA concentration, magnesium sulfate concentration, and annealing temperature, for the mutant DNA and designed primers, would be found through multiple experimental trials. Performing a PCR assay containing the mutant DNA would further prove our hypothesis that our designed primers would anneal correctly producing two bands of 212 base pairs and 432 base pairs. This would provide evidence that using PCR is an effective assay for detecting the R117H mutation for genetic mutation screening in cystic fibrosis patients.

An experimental weakness that was encountered was properly preparing the PCR in order to replicate the target DNA. Creating the proper ratio of all reagents and the concentration is vital for the target DNA for PCR. Depending on the primer length different concentrations and binding agents were used to amplify DNA. Magnesium is an example of a reagent that is necessary for PCR to properly replicate DNA (Barlett, 2003). Another analysis of experimental flaws was inserting the samples into wells. Often, the DNA was allowed to exist in the saline solution, or a hole was poked into the gel. This caused the DNA in the saline solution or in the damaged wells to not run in proportion to the correctly filled and intact wells. In order to offset both affects, multiple trials were performed to clearly distinguish mistakes from true findings.

Figures

 

Figure 5: Results from Amplified Products Using Gel Electrophoresis. A shows the band length from gel electrophoresis, based off of the control upstream and downstream primers used in the control paper (Shrimpton, 2000) Successful replication of published primers. Well A contains 1 Kb+ ladder. Wells C-E successful PCR of published Primers. Well G contains control of Lambda control.  Wells B and F left empty. Multiple bands in Wells C-E due to excess TAQ and Primer Dimers. B represents the correlation of base pairs with the approximation dependent on the ladders measured distance travelled.