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 …

Insights into genetics, human biology and disease gleaned from family based genomic studies

JE Posey, AH O'Donnell-Luria, JX Chong, T Harel… - Genetics in …, 2019 - nature.com
Identifying genes and variants contributing to rare disease phenotypes and Mendelian
conditions informs biology and medicine, yet potential phenotypic consequences for …

A gene-specific T2A-GAL4 library for Drosophila

PT Lee, J Zirin, O Kanca, WW Lin, KL Schulze… - elife, 2018 - elifesciences.org
We generated a library of~ 1000 Drosophila stocks in which we inserted a construct in the
intron of genes allowing expression of GAL4 under control of endogenous promoters while …

[HTML][HTML] Regulatory roles of mitochondria and metabolism in neurogenesis

R Iwata, P Vanderhaeghen - Current opinion in neurobiology, 2021 - Elsevier
Neural stem cells (NSCs) undergo massive molecular and cellular changes during neuronal
differentiation. These include mitochondria and metabolism remodelling, which were …

A defect in mitochondrial fatty acid synthesis impairs iron metabolism and causes elevated ceramide levels

D Dutta, O Kanca, SK Byeon, PC Marcogliese… - Nature …, 2023 - nature.com
In most eukaryotic cells, fatty acid synthesis (FAS) occurs in the cytoplasm and in
mitochondria. However, the relative contribution of mitochondrial FAS (mtFAS) to the cellular …

Model organisms facilitate rare disease diagnosis and therapeutic research

MF Wangler, S Yamamoto, HT Chao, JE Posey… - Genetics, 2017 - academic.oup.com
Efforts to identify the genetic underpinnings of rare undiagnosed diseases increasingly
involve the use of next-generation sequencing and comparative genomic hybridization …

An efficient CRISPR-based strategy to insert small and large fragments of DNA using short homology arms

O Kanca, J Zirin, J Garcia-Marques, SM Knight… - Elife, 2019 - elifesciences.org
We previously reported a CRISPR-mediated knock-in strategy into introns of Drosophila
genes, generating an attP-FRT-SA-T2A-GAL4-polyA-3XP3-EGFP-FRT-attP transgenic …

The existence of a nonclassical TCA cycle in the nucleus that wires the metabolic-epigenetic circuitry

X Liu, W Si, L He, J Yang, Y Peng, J Ren… - Signal transduction and …, 2021 - nature.com
The scope and variety of the metabolic intermediates from the mitochondrial tricarboxylic
acid (TCA) cycle that are engaged in epigenetic regulation of the chromatin function in the …

High prevalence of multilocus pathogenic variation in neurodevelopmental disorders in the Turkish population

T Mitani, S Isikay, A Gezdirici, EY Gulec… - The American Journal of …, 2021 - cell.com
Neurodevelopmental disorders (NDDs) are clinically and genetically heterogenous; many
such disorders are secondary to perturbation in brain development and/or function. The …

Model organisms contribute to diagnosis and discovery in the undiagnosed diseases network: current state and a future vision

D Baldridge, MF Wangler, AN Bowman… - Orphanet Journal of …, 2021 - Springer
Decreased sequencing costs have led to an explosion of genetic and genomic data. These
data have revealed thousands of candidate human disease variants. Establishing which …