Modelling human brain development and disease with organoids

M Birtele, M Lancaster, G Quadrato - Nature Reviews Molecular Cell …, 2024 - nature.com
Organoids are systems derived from pluripotent stem cells at the interface between
traditional monolayer cultures and in vivo animal models. The structural and functional …

Recent therapeutic gene editing applications to genetic disorders

E Deneault - Current Issues in Molecular Biology, 2024 - mdpi.com
Recent years have witnessed unprecedented progress in therapeutic gene editing,
revolutionizing the approach to treating genetic disorders. In this comprehensive review, we …

Modeling Parkinson's disease pathology in human dopaminergic neurons by sequential exposure to α-synuclein fibrils and proinflammatory cytokines

A Bayati, R Ayoubi, A Aguila, CE Zorca, G Deyab… - Nature …, 2024 - nature.com
Abstract Lewy bodies (LBs), α-synuclein-enriched intracellular inclusions, are a hallmark of
Parkinson's disease (PD) pathology, yet a cellular model for LB formation remains elusive …

Alpha synuclein determines ferroptosis sensitivity in dopaminergic neurons via modulation of ether-phospholipid membrane composition

L Mahoney-Sanchez, H Bouchaoui, I Boussaad… - Cell reports, 2022 - cell.com
There is a continued unmet need for treatments that can slow Parkinson's disease
progression due to the lack of understanding behind the molecular mechanisms underlying …

Modeling Lewy body disease with SNCA triplication iPSC-derived cortical organoids and identifying therapeutic drugs

Y **, F Li, Z Li, TC Ikezu, J O'Leary, M Selvaraj… - Science …, 2024 - science.org
Aggregated α-synuclein (α-SYN) proteins, encoded by the SNCA gene, are hallmarks of
Lewy body disease (LBD), affecting multiple brain regions. However, the specific …

Tom20 gates PINK1 activity and mediates its tethering of the TOM and TIM23 translocases upon mitochondrial stress

MA Eldeeb, AN Bayne, A Fallahi, T Goiran… - Proceedings of the …, 2024 - pnas.org
Mutations in PTEN-induced putative kinase 1 (PINK1) cause autosomal recessive early-
onset Parkinson's disease (PD). PINK1 is a Ser/Thr kinase that regulates mitochondrial …

The Parkinson's disease risk gene cathepsin B promotes fibrillar alpha-synuclein clearance, lysosomal function and glucocerebrosidase activity in dopaminergic …

J Jones-Tabah, K He, N Karpilovsky… - Molecular …, 2024 - Springer
Background Variants in the CTSB gene encoding the lysosomal hydrolase cathepsin B
(catB) are associated with increased risk of Parkinson's disease (PD). However, neither the …

Disruption of lysosomal proteolysis in astrocytes facilitates midbrain organoid proteostasis failure in an early-onset Parkinson's disease model

G Morrone Parfitt, E Coccia, C Goldman… - Nature …, 2024 - nature.com
Accumulation of advanced glycation end products (AGEs) on biopolymers accompanies
cellular aging and drives poorly understood disease processes. Here, we studied how AGEs …

Alpha‐synuclein‐associated changes in PINK1‐PRKN‐mediated mitophagy are disease context dependent

X Hou, THY Chen, S Koga, JM Bredenberg… - Brain …, 2023 - Wiley Online Library
Alpha‐synuclein (αsyn) aggregates are pathological features of several neurodegenerative
conditions including Parkinson disease (PD), dementia with Lewy bodies, and multiple …

Midbrain organoids for Parkinson's disease (PD)-A powerful tool to understand the disease pathogenesis

HWS Babu, SM Kumar, H Kaur, M Iyer, B Vellingiri - Life Sciences, 2024 - Elsevier
Abstract Brain Organiods (BOs) are a promising technique for researching disease
progression in the human brain. These organoids, which are produced from human induced …