Zebrafish disease models in drug discovery: from preclinical modelling to clinical trials

EE Patton, LI Zon, DM Langenau - Nature reviews Drug discovery, 2021 - nature.com
Numerous drug treatments that have recently entered the clinic or clinical trials have their
genesis in zebrafish. Zebrafish are well established for their contribution to developmental …

Detecting liquid remnants of solid tumors: circulating tumor DNA minimal residual disease

EJ Moding, BY Nabet, AA Alizadeh, M Diehn - Cancer discovery, 2021 - aacrjournals.org
Growing evidence demonstrates that circulating tumor DNA (ctDNA) minimal residual
disease (MRD) following treatment for solid tumors predicts relapse. These results suggest …

[HTML][HTML] Single-cell atlas of the human brain vasculature across development, adulthood and disease

T Wälchli, M Ghobrial, M Schwab, S Takada, H Zhong… - Nature, 2024 - nature.com
A broad range of brain pathologies critically relies on the vasculature, and cerebrovascular
disease is a leading cause of death worldwide. However, the cellular and molecular …

Sha** the brain vasculature in development and disease in the single-cell era

T Wälchli, J Bisschop, P Carmeliet, G Zadeh… - Nature Reviews …, 2023 - nature.com
The CNS critically relies on the formation and proper function of its vasculature during
development, adult homeostasis and disease. Angiogenesis—the formation of new blood …

Genetic basis and therapies for vascular anomalies

A Queisser, E Seront, LM Boon, M Vikkula - Circulation research, 2021 - ahajournals.org
Vascular and lymphatic malformations represent a challenge for clinicians. The identification
of inherited and somatic mutations in important signaling pathways, including the PI3K …

[HTML][HTML] Novel targets, treatments, and advanced models for intracerebral haemorrhage

M Zille, TD Farr, RF Keep, C Römer, G **, J Boltze - EBioMedicine, 2022 - thelancet.com
Intracerebral haemorrhage (ICH) is the second most common type of stroke and a major
cause of mortality and disability worldwide. Despite advances in surgical interventions and …

Childhood stroke

PB Sporns, HJ Fullerton, S Lee, H Kim, WD Lo… - Nature Reviews …, 2022 - nature.com
Stroke is an important cause of neurological morbidity in children; most survivors have
permanent neurological deficits that affect the remainder of their life. Stroke in childhood, the …

Somatic mutations in “benign” disease

S Mustjoki, NS Young - New England Journal of Medicine, 2021 - Mass Medical Soc
Somatic Mutations in “Benign” Disease DNA mutations occur in nearly every tissue
throughout the human life span and accumulate at various rates in different tissues …

Contribution of somatic Ras/Raf/mitogen-activated protein kinase variants in the hippocampus in drug-resistant mesial temporal lobe epilepsy

S Khoshkhoo, Y Wang, Y Chahine… - JAMA …, 2023 - jamanetwork.com
Importance Mesial temporal lobe epilepsy (MTLE) is the most common focal epilepsy
subtype and is often refractory to antiseizure medications. While most patients with MTLE do …

Sotorasib for Vascular Malformations Associated with KRAS G12C Mutation

A Fraissenon, C Bayard, G Morin… - … England Journal of …, 2024 - Mass Medical Soc
KRAS gain-of-function mutations are frequently observed in sporadic arteriovenous
malformations. The mechanisms underlying the progression of such KRAS-driven …