Loeys-dietz syndrome

BL Loeys - 1993 - books.google.com
In 2005, Bart Loeys and Harry Dietz described a new syndromic aneurysmal entity. Its
patients were typically characterized by a clinical triad that included hypertelorism (widely …

Mutations in the U4 snRNA gene RNU4-2 cause one of the most prevalent monogenic neurodevelopmental disorders

D Greene, C Thys, IR Berry, J Jarvis, E Ortibus… - Nature Medicine, 2024 - nature.com
Most people with intellectual disability (ID) do not receive a molecular diagnosis following
genetic testing. To identify new etiologies of ID, we performed a genetic association analysis …

Update on recommendations for surveillance for children with predisposition to hematopoietic malignancy

LD Maese, MW Wlodarski, SY Kim… - Clinical Cancer …, 2024 - aacrjournals.org
Children harboring certain germline gene variants have an increased risk of develo**
myelodysplastic syndrome (MDS) and other hematopoietic malignancies (HM), such as …

Standardization of definition and management for bleeding disorder of unknown cause: communication from the SSC of the ISTH

RI Baker, P Choi, N Curry, J Gebhart, K Gomez… - Journal of thrombosis …, 2024 - Elsevier
In many patients referred with significant bleeding phenotype, laboratory testing fails to
define any hemostatic abnormalities. Clinical practice with respect to diagnosis and …

Molecular insights into the activation mechanism of GPR156 in maintaining auditory function

X Ma, LN Chen, M Liao, L Zhang, K **, J Guo… - Nature …, 2024 - nature.com
The class C orphan G-protein-coupled receptor (GPCR) GPR156, which lacks the large
extracellular region, plays a pivotal role in auditory function through G i2/3. Here, we firstly …

Loss-of-function mutations of the TIE1 receptor tyrosine kinase cause late-onset primary lymphedema

P Brouillard, A Murtomäki, VM Leppänen… - The Journal of clinical …, 2024 - jci.org
Primary lymphedema (PL), characterized by tissue swelling, fat accumulation, and fibrosis,
results from defects in lymphatic vessels or valves caused by mutations in genes involved in …

Constitutive activation mechanism of a class C GPCR

J Shin, J Park, J Jeong, JH Lam, X Qiu, D Wu… - Nature Structural & …, 2024 - nature.com
Class C G-protein-coupled receptors (GPCRs) are activated through binding of agonists to
the large extracellular domain (ECD) followed by rearrangement of the transmembrane …

The AnnotSV webserver in 2023: updated visualization and ranking

V Geoffroy, JB Lamouche, T Guignard… - Nucleic Acids …, 2023 - academic.oup.com
Much of the human genetics variant repertoire is composed of single nucleotide variants
(SNV) and small insertion/deletions (indel) but structural variants (SV) remain a major part of …

Bioinformatics of germline variant discovery for rare disease diagnostics: current approaches and remaining challenges

YA Barbitoff, MO Ushakov, TE Lazareva… - Briefings in …, 2024 - academic.oup.com
Next-generation sequencing (NGS) has revolutionized the field of rare disease diagnostics.
Whole exome and whole genome sequencing are now routinely used for diagnostic …

Evolutionarily new genes in humans with disease phenotypes reveal functional enrichment patterns shaped by adaptive innovation and sexual selection

JH Chen, P Landback, D Arsala, A Guzzetta… - Genome …, 2025 - genome.cshlp.org
New genes (or young genes) are genetic novelties pivotal in mammalian evolution.
However, their phenotypic impacts and evolutionary patterns over time remain elusive in …