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Editing the genome of hiPSC with CRISPR/Cas9: disease models
- Source :
- Mammalian Genome
- Publication Year :
- 2016
-
Abstract
- The advent of human-induced pluripotent stem cell (hiPSC) technology has provided a unique opportunity to establish cellular models of disease from individual patients, and to study the effects of the underlying genetic aberrations upon multiple different cell types, many of which would not normally be accessible. Combining this with recent advances in genome editing techniques such as the clustered regularly interspaced short palindromic repeat (CRISPR) system has provided an ability to repair putative causative alleles in patient lines, or introduce disease alleles into a healthy "WT" cell line. This has enabled analysis of isogenic cell pairs that differ in a single genetic change, which allows a thorough assessment of the molecular and cellular phenotypes that result from this abnormality. Importantly, this establishes the true causative lesion, which is often impossible to ascertain from human genetic studies alone. These isogenic cell lines can be used not only to understand the cellular consequences of disease mutations, but also to perform high throughput genetic and pharmacological screens to both understand the underlying pathological mechanisms and to develop novel therapeutic agents to prevent or treat such diseases. In the future, optimising and developing such genetic manipulation technologies may facilitate the provision of cellular or molecular gene therapies, to intervene and ultimately cure many debilitating genetic disorders.
- Subjects :
- 0301 basic medicine
Induced Pluripotent Stem Cells
Biology
Genome
Article
03 medical and health sciences
Genome editing
Genetics
CRISPR
Animals
Humans
Genetic Predisposition to Disease
Allele
Induced pluripotent stem cell
Gene
Genetic Association Studies
Gene Editing
Genetic Variation
Isogenic human disease models
Human genetics
3. Good health
Disease Models, Animal
030104 developmental biology
CRISPR-Cas Systems
Genetic Engineering
Subjects
Details
- ISSN :
- 14321777
- Volume :
- 28
- Issue :
- 7-8
- Database :
- OpenAIRE
- Journal :
- Mammalian genome : official journal of the International Mammalian Genome Society
- Accession number :
- edsair.doi.dedup.....d20f141318682c1a40e06f45d74bf4c5