Functional genomics in stroke: current and future applications of iPSCs and gene editing to dissect the function of risk variants.

Published version
Repository DOI

Type
Article
Change log
Authors
Granata, Alessandra  ORCID logo  https://orcid.org/0000-0001-5051-8201
Abstract

Stroke is an important disease with unmet clinical need. To uncover novel paths for treatment, it is of critical importance to develop relevant laboratory models that may help to shed light on the pathophysiological mechanisms of stroke. Induced pluripotent stem cells (iPSCs) technology has enormous potential to advance our knowledge into stroke by creating novel human models for research and therapeutic testing. iPSCs models generated from patients with specific stroke types and specific genetic predisposition in combination with other state of art technologies including genome editing, multi-omics, 3D system, libraries screening, offer the opportunity to investigate disease-related pathways and identify potential novel therapeutic targets that can then be tested in these models. Thus, iPSCs offer an unprecedented opportunity to make rapid progress in the field of stroke and vascular dementia research leading to clinical translation. This review paper summarizes some of the key areas in which patient-derived iPSCs technology has been applied to disease modelling and discusses the ongoing challenges and the future directions for the application of this technology in the field of stroke research.

Description

Funder: British Heart Foundation

Keywords
Stroke, Induced Pluripotent Stem Cells, Small Vessel Disease, Disease Modeling, Genome Editing, Genetic Risk Variant, Humans, Genomics, Precision Medicine, Gene Editing
Journal Title
BMC cardiovascular disorders
Conference Name
Journal ISSN
1471-2261
Volume Title
23
Publisher
Sponsorship
Medical Research Foundation (RG98759)