Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …

Towards a better understanding of diabetes mellitus using organoid models

BS Beydag-Tasöz, S Yennek… - Nature Reviews …, 2023 - nature.com
Our understanding of diabetes mellitus has benefited from a combination of clinical
investigations and work in model organisms and cell lines. Organoid models for a wide …

Directed differentiation of pancreatic δ cells from human pluripotent stem cells

L Chen, N Wang, T Zhang, F Zhang, W Zhang… - Nature …, 2024 - nature.com
Dysfunction of pancreatic δ cells contributes to the etiology of diabetes. Despite their
important role, human δ cells are scarce, limiting physiological studies and drug discovery …

Towards a functional cure for diabetes using stem cell-derived beta cells: are we there yet?

S Bourgeois, T Sawatani, A Van Mulders, N De Leu… - Cells, 2021 - mdpi.com
Diabetes mellitus is a pandemic metabolic disorder that results from either the autoimmune
destruction or the dysfunction of insulin-producing pancreatic beta cells. A promising cure is …

Nanomedicine in the Treatment of Diabetes

A Andreadi, P Lodeserto, F Todaro, M Meloni… - International Journal of …, 2024 - mdpi.com
Nanomedicine could improve the treatment of diabetes by exploiting various therapeutic
mechanisms through the use of suitable nanoformulations. For example, glucose-sensitive …

Islet organoid as a promising model for diabetes

X Zhang, Z Ma, E Song, T Xu - Protein & cell, 2022 - academic.oup.com
Studies on diabetes have long been hampered by a lack of authentic disease models that,
ideally, should be unlimited and able to recapitulate the abnormalities involved in the …

Maturity onset diabetes of the young—new approaches for disease modelling

D Skoczek, J Dulak… - International Journal of …, 2021 - mdpi.com
Maturity-onset diabetes of the young (MODY) is a genetically heterogeneous group of
monogenic endocrine disorders that is characterised by autosomal dominant inheritance …

Human pluripotent stem cells to model islet defects in diabetes

D Balboa, DG Iworima, TJ Kieffer - Frontiers in Endocrinology, 2021 - frontiersin.org
Diabetes mellitus is characterized by elevated levels of blood glucose and is ultimately
caused by insufficient insulin production from pancreatic beta cells. Different research …

CRISPR and iPSCs: recent developments and future perspectives in neurodegenerative disease modelling, research, and therapeutics

T Sen, RP Thummer - Neurotoxicity Research, 2022 - Springer
Neurodegenerative diseases are prominent causes of pain, suffering, and death worldwide.
Traditional approaches modelling neurodegenerative diseases are deficient, and therefore …

Concise review: human pluripotent stem cells for the modeling of pancreatic β-cell pathology

D Balboa, J Saarimäki-Vire, T Otonkoski - Stem Cells, 2019 - academic.oup.com
Pancreatic β-cells are the only source of insulin. Disturbances in β-cell development or
function may thus result in insulin deficiency or excess, presenting as hyper-or …