Multi-omics identifies large mitoribosomal subunit instability caused by pathogenic MRPL39 variants as a cause of pediatric onset mitochondrial disease

SSC Amarasekera, DH Hock, NJ Lake… - Human molecular …, 2023 - academic.oup.com
MRPL39 encodes one of 52 proteins comprising the large subunit of the mitochondrial
ribosome (mitoribosome). In conjunction with 30 proteins in the small subunit, the …

Biallelic loss‐of‐function variants in EXOC6B are associated with impaired primary ciliogenesis and cause spondylo‐epi‐metaphyseal dysplasia with joint laxity type …

PO Simsek‐Kiper, P Jacob, P Upadhyai… - Human …, 2022 - Wiley Online Library
Spondylo‐epi‐metaphyseal dysplasias with joint laxity, type 3 (SEMDJL3) is a genetic
skeletal disorder characterized by multiple joint dislocations, caused by biallelic pathogenic …

Proteomics analysis of prefrontal cortex of Alzheimer's disease patients revealed dysregulated proteins in the disease and novel proteins associated with amyloid-β …

A Montero-Calle, R Coronel… - Cellular and Molecular …, 2023 - Springer
Background Alzheimer's disease (AD) is a progressive, chronic, and neurodegenerative
disease, and the most common cause of dementia worldwide. Currently, the mechanisms …

The exocyst complex is an essential component of the mammalian constitutive secretory pathway

C Pereira, D Stalder, GSF Anderson… - Journal of Cell …, 2023 - rupress.org
Secreted proteins fulfill a vast array of functions, including immunity, signaling, and
extracellular matrix remodeling. In the trans-Golgi network, proteins destined for constitutive …

A micro-costing study of mass-spectrometry based quantitative proteomics testing applied to the diagnostic pipeline of mitochondrial and other rare disorders

F Santos Gonzalez, DH Hock, DR Thorburn… - Orphanet Journal of …, 2024 - Springer
Background Mass spectrometry-based quantitative proteomics has a demonstrated utility in
increasing the diagnostic yield of mitochondrial disorders (MDs) and other rare diseases …

Copy number variants in genomes of local sheep breeds from Russia

AV Igoshin, TE Deniskova, AA Yurchenko… - Animal …, 2022 - Wiley Online Library
Copy number variants (CNVs) are genomic structural variations that contribute to many
adaptive and economically important traits in livestock. In this study, we detected CNVs in …

Exocyst stimulates multiple steps of exocytic SNARE complex assembly and vesicle fusion

C Lee, D Lepore, SH Lee, TG Kim, N Buwa… - Nature Structural & …, 2024 - nature.com
Exocyst is a large multisubunit tethering complex essential for targeting and fusion of
secretory vesicles in eukaryotic cells. Although the assembled exocyst complex has been …

The exocyst subunit EXOC2 regulates the toxicity of expanded GGGGCC repeats in C9ORF72-ALS/FTD

DO Halim, G Krishnan, EP Hass, S Lee, M Verma… - Cell reports, 2024 - cell.com
Summary GGGGCC (G 4 C 2) repeat expansion in C9ORF72 is the most common genetic
cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). How this …

RNA Landscapes of Brain and Brain-Derived Extracellular Vesicles in Simian Immunodeficiency Virus Infection and Central Nervous System Pathology

Y Huang, A Abdelgawad, A Turchinovich… - The Journal of …, 2024 - academic.oup.com
Background Brain tissue-derived extracellular vesicles (bdEVs) act locally in the central
nervous system (CNS) and may indicate molecular mechanisms in human …

Untargeted proteomics enables ultra-rapid variant prioritization in mitochondrial and other rare diseases

DH Hock, NJ Caruana, LN Semcesen, NJ Lake… - medRxiv, 2024 - medrxiv.org
Only half of individuals with suspected rare diseases receive a definitive genetic diagnosis
following genomic testing. A genetic diagnosis allows access to appropriate patient care and …