Integrating non-mammalian model organisms in the diagnosis of rare genetic diseases in humans

S Yamamoto, O Kanca, MF Wangler… - Nature Reviews …, 2024 - nature.com
Next-generation sequencing technology has rapidly accelerated the discovery of genetic
variants of interest in individuals with rare diseases. However, showing that these variants …

A comprehensive Drosophila resource to identify key functional interactions between SARS-CoV-2 factors and host proteins

A Guichard, S Lu, O Kanca, D Bressan, Y Huang, M Ma… - Cell reports, 2023 - cell.com
Development of effective therapies against SARS-CoV-2 infections relies on mechanistic
knowledge of virus-host interface. Abundant physical interactions between viral and host …

A nerve-wracking buzz: lessons from Drosophila models of peripheral neuropathy and axon degeneration

MRC Bhattacharya - Frontiers in Aging Neuroscience, 2023 - frontiersin.org
The degeneration of axons and their terminals occurs following traumatic, toxic, or
genetically-induced insults. Common molecular mechanisms unite these disparate triggers …

Bridging clinical care and research in Ontario, Canada: Maximizing diagnoses from reanalysis of clinical exome sequencing data

T Hartley, É Soubry, M Acker, M Osmond… - Clinical …, 2023 - Wiley Online Library
We examined the utility of clinical and research processes in the reanalysis of publicly‐
funded clinical exome sequencing data in Ontario, Canada. In partnership with eight sites …

[HTML][HTML] Charting the shared genetic architecture of Alzheimer's disease, cognition, and educational attainment, and associations with brain development

P Jaholkowski, S Bahrami, V Fominykh… - Neurobiology of …, 2024 - Elsevier
The observation that the risk of develo** Alzheimer's disease is reduced in individuals
with high premorbid cognitive functioning, higher educational attainment, and occupational …

Epilepsy due to potential loss of ATP6V1B2 function with mechanistic insight by a Drosophila Vha55 model

W Sheng, P Wang, Y Cai, C Zhai, H Wang… - Clinical …, 2024 - Wiley Online Library
Abstract ATP6V1B2 encodes the subunit of the vacuolar H+‐ATPase, which is an enzyme
responsible for the acidification of intracellular organelles and essential for cell signaling …

NDUFS7 variant in dogs with Leigh syndrome and its functional validation in a Drosophila melanogaster model

M Christen, A Gregor, R Gutierrez-Quintana… - Scientific Reports, 2024 - nature.com
Abstract Two Jack-Russell Terrier× Chihuahua mixed-breed littermates with Leigh syndrome
were investigated. The dogs presented with progressive ataxia, dystonia, and increased …

A recurrent missense variant in the E3 ubiquitin ligase substrate recognition subunit FEM1B causes a rare syndromic neurodevelopmental disorder

F Lecoquierre, AM Punt, F Ebstein, I Wallaard… - Genetics in …, 2024 - Elsevier
Abstract Purpose Fem1 homolog B (FEM1B) acts as a substrate recognition subunit for
ubiquitin ligase complexes belonging to the CULLIN 2-based E3 family. Several biological …

The implementation of an enhanced clinical model to improve the diagnostic yield of exome sequencing for patients with a rare genetic disease: A Canadian …

GU Ediae, G Lemire, C Chisholm… - American Journal of …, 2023 - Wiley Online Library
The introduction of clinical exome sequencing (ES) has provided a unique opportunity to
decrease the diagnostic odyssey for patients living with a rare genetic disease (RGD). ES …

GREGoR: Accelerating Genomics for Rare Diseases

M Dawood, B Heavner, MM Wheeler, RA Ungar… - arxiv preprint arxiv …, 2024 - arxiv.org
Rare diseases are collectively common, affecting approximately one in twenty individuals
worldwide. In recent years, rapid progress has been made in rare disease diagnostics due …