Rare diseases poster 20

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In silico analysis of miRNAs in hydroxyurea induced foetal haemoglobin in sickle cell anaemia patients Smruti Sudha Biswal1, Anand Bodade2 1National Institute of Technology, Rourkela; 2NIIH, ICMR, Mumbai Methods and Materials

Abstract Sickle cell disease is a hereditary disease caused due to a change in 6 th position of beta globin chain from glutamic acid to valine. Various therapies are underway for the treatment, but hydroxyurea remains to be at the most sought form of treatment. However the exact molecular mechanisms of the miRNAs in this process remains elusive. Here in this study we have tried to elucidate the differentially expressed miRNAs and their plausible role in induction of foetal haemoglobin.

Introduction 1. Sickle cell anaemia is caused due to substitution of valine to glutamic acid in the 6th position of the beta globin 2.

Hydroxyurea (HU) is an oral, S-phase, cytotoxic, anti-metabolic and anti-neoplastic drug. It induces HbF in the sickle cell anaemic patients and is known to reduce the episodes of pain in the same 3. However, there the role of miRNAs in HUinduced HbF production in SCD patients is still unknown. 4. Here in this study we attempted to elucidate the role of top 50 differentially expressed miRNAs from the analysis of microarray data obtained through online databases. Their molecular mechanisms have been identified through functional annotation and KEGG pathway enrichment analysis

Aims and objectives Identifying and In silico analysis of miRNAs in hydroxyurea induced foetal haemoglobin in SCD patients

1. 2. 3. 4. 5.

Collection of dataset from Array express database (GSE32035 and GSE11060) Identification of DE expressed miRNAs Finding out targets of the identified DEmiRNAs Gene set enrichment analysis of the gene targets Pathway enrichment analysis of the identified gene targets

Fig 3: Heat maps of normalised data

Fig 1: Box plots of normalized data of GSE32025 and GSE11060

GSE32025

GSE11060

TYPE OF STUDY

Observational

Observational

OBSERVATIONAL

Cohort

Cohort

TIME PERSPECTIVE

Prospective

Prospective

ACTUAL

260 Participants

260 Participants

3

2

Fig 2: Volcano plots of the normalised data

hsa-miR-223*

Pathways in cancer

hsa-miR-126

Chronic myeloid leukemia

hsa-miR-30a*

MAPK signaling pathway

kshv-miR-K12-1

Neurotrophin signaling pathway

hsa-miR-342-3p

Pancreatic cancer

Table 2: DE miRNAs and the pathways enriched

Discussion

MODEL

ENROLLMENT NO OF COHORTS

Table 1: Details of study

Results 1. Out of the 50 DE miRNAs chosen from the lot of 2000 produced by the software, 32 DE miRNAs were identified to be downregulated while the remaining ones were upregulated. 2. A total of around 29000 genes were identified as targets for the DE miRNAs. Upon identification of targets, the gene set enrichment analysis was performed which identified around 125 enriched biological functions were identified. 3. Similarly through the pathway enrichment analysis, B cell receptor signalling pathway, T cell receptor signalling pathway, B-cell receptor signalling pathway, Maturity onset diabetes of the young (MODY), endocytosis, apoptosis, MAPK Signalling pathway, Axon guidance, ErB Signalling pathway, renal cell carcinoma, pancreatic cancer and endocytosis pathways were identified.

1. Through this study, we were able to identify different kinds of miRNAs like miR-29b-2, miR-552, miR-765, kshv-miR-K12-1 which were not only downregulated but also involved in different kinds of biological functions and pathways. 2. These miRNAs might be plausibly involved in the HbF induction process following HU 3. This information can be exploited to find out novel therapeutic targets for the treatment of sickle cell anaemia using the recently developed genome editing and gene therapy tools

Future Directions With this background we plan to conduct similar analysis in Indian SCD patient’s cohort. This will help in understanding the mechanism of action of HU in SCD patients via miRNAs. This will help in optimizing HU therapy in Indian cohort. This will definitely prove a better therapeutic approach in managing Sickle cell disease which is still a public health challenge in front of our country

References 1.Mnika K, Mazandu GK, Jonas M, et al. Hydroxyurea-Induced miRNA Expression in Sickle Cell Disease Patients in Africa. Front Gene 2019;10:509. 2.Gift D Pule, ShaheenMowla, Nicolas Novitzky, Charles S Wiysonge&AmbroiseWonkam (2015): A systematic review of known mechanisms of hydroxyureainduced fetalhemoglobin for treatment of sickle cell disease, Expert Review of Hematology

Acknowledgements

CSIR SRTP 2020


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