Use of human pluripotent stem cells to model monogenic diabetes

El-Khairi, Ranna; Hattersley, Andrew; Vallier, Ludovic

doi:10.1530/endoabs.51.oc7.2

Cited by 2 publications

(1 citation statement)

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“…Human pluripotent cells that are capable of differentiating into any type of cell and are self-renewing have been mutated through the use of the CRISPR/Cas9 tool. Here, mutations were induced in the hepatocyte nuclear factor (HNF) 1b gene that is thought to know to cause diabetes via pancreatic hypoplasia and β -cell dysfunction [74].…”

Section: Multifactorial Diseasesmentioning

confidence: 99%

Era of Genomic Medicine: A Narrative Review on CRISPR Technology as a Potential Therapeutic Tool for Human Diseases

Kotagama

Jayasinghe

Abeysinghe

2019

BioMed Research International

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Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) provides acquired immunity in microorganisms against exogenous DNA that may hinder the survival of the organism. Pioneering work by Doudna and Charpentier in 2012 resulted in the creation of the CRISPR/Cas9 genome editing tool on the basis of this concept. The aim of this was to create a rapid, efficient, and versatile genome-editing tool to facilitate genetic manipulation. The mechanism relies on two components: the RNA guide which acts as a sentinel and a Cas protein complex which functions as a highly precise molecular knife. The guide RNA can be modified to match a DNA sequence of interest in the cell and accordingly be used to rectify mutations that may otherwise cause disease. Within a few years following the development of the CRISPR/Cas9 tool, its usage has become ubiquitous. Its influence extends into many fields of biological sciences from biotechnology and biochemistry to molecular biology and biomedical sciences. The following review aims at shedding some light on to the applications of the CRISPR/Cas9 tool in the field of biomedical sciences, particularly gene therapy. An insight with relation to a few of the many diseases that are being tackled with the aid of the CRISPR/Cas9 mechanism and the trends, successes, and challenges of this application as a gene therapy are discussed in this review.

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Section: Multifactorial Diseasesmentioning

confidence: 99%