The rapidly evolving view of lysosomal storage diseases

G Parenti, DL Medina, A Ballabio - EMBO molecular medicine, 2021 - embopress.org
Lysosomal storage diseases are a group of metabolic disorders caused by deficiencies of
several components of lysosomal function. Most commonly affected are lysosomal …

Mass spectrometry-based proteomics as an emerging tool in clinical laboratories

AG Birhanu - Clinical Proteomics, 2023 - Springer
Mass spectrometry (MS)-based proteomics have been increasingly implemented in various
disciplines of laboratory medicine to identify and quantify biomolecules in a variety of …

Proteomics reveals that methylmalonyl-CoA mutase modulates cell architecture and increases susceptibility to stress

M Costanzo, M Caterino, A Cevenini, V Jung… - International journal of …, 2020 - mdpi.com
Methylmalonic acidemia (MMA) is a rare inborn error of metabolism caused by deficiency of
the methylmalonyl-CoA mutase (MUT) enzyme. Downstream MUT deficiency, methylmalonic …

Diagnosing rare diseases after the exome

L Frésard, SB Montgomery - Molecular Case …, 2018 - molecularcasestudies.cshlp.org
High-throughput sequencing has ushered in a diversity of approaches for identifying genetic
variants and understanding genome structure and function. When applied to individuals with …

Dynamic interactomics by cross-linking mass spectrometry: map** the daily cell life in postgenomic era

L Santorelli, M Caterino, M Costanzo - OMICS: A Journal of …, 2022 - liebertpub.com
The majority of processes that occur in daily cell life are modulated by hundreds to
thousands of dynamic protein–protein interactions (PPI). The resulting protein complexes …

Targeted metabolomic analysis of a mucopolysaccharidosis IIIB mouse model reveals an imbalance of branched-chain amino acid and fatty acid metabolism

V De Pasquale, M Caterino, M Costanzo… - International journal of …, 2020 - mdpi.com
Mucopolysaccharidoses (MPSs) are inherited disorders of the glycosaminoglycan (GAG)
metabolism. The defective digestion of GAGs within the intralysosomal compartment of …

Advances in proteomic profiling of pediatric kidney diseases

TD Cummins, EA Korte, S Bhayana, ML Merchant… - Pediatric …, 2022 - Springer
Chronic kidney disease (CKD) can progress to kidney failure and require dialysis or
transplantation, while early diagnosis can alter the course of disease and lead to better …

Need and challenges in establishing newborn screening programs for inherited metabolic disorders in develo** countries

M Wasim, HN Khan, H Ayesha, FR Awan - Advanced Biology, 2023 - Wiley Online Library
Even in this post genomic era, no national level newborn screening (NBS) programs for
inborn errors of metabolism (IEMs) are yet available in several develo** countries …

Identification of volatile compounds from bacteria by spectrometric methods in medicine diagnostic and other areas: current state and perspectives

N Kunze-Szikszay, M Euler, T Perl - Applied Microbiology and …, 2021 - Springer
Diagnosis of bacterial infections until today mostly relies on conventional microbiological
methods. The resulting long turnaround times can lead to delayed initiation of adequate …

Next‐generation sequencing approaches and challenges in the diagnosis of developmental anomalies and intellectual disability

AL Bruel, A Vitobello, F Tran Mau‐Them… - Clinical …, 2020 - Wiley Online Library
Recent advances in next‐generation sequencing (NGS) technologies have revolutionized
the field of human genetics. Alongside a broad panel of bioinformatics tools and databases …