A guide for the diagnosis of rare and undiagnosed disease: beyond the exome

S Marwaha, JW Knowles, EA Ashley - Genome medicine, 2022‏ - Springer
Rare diseases affect 30 million people in the USA and more than 300–400 million
worldwide, often causing chronic illness, disability, and premature death. Traditional …

30 years of repeat expansion disorders: What have we learned and what are the remaining challenges?

C Depienne, JL Mandel - The American Journal of Human Genetics, 2021‏ - cell.com
Tandem repeats represent one of the most abundant class of variations in human genomes,
which are polymorphic by nature and become highly unstable in a length-dependent …

[HTML][HTML] On the wrong DNA track: Molecular mechanisms of repeat-mediated genome instability

AN Khristich, SM Mirkin - Journal of Biological Chemistry, 2020‏ - Elsevier
Expansions of simple tandem repeats are responsible for almost 50 human diseases, the
majority of which are severe, degenerative, and not currently treatable or preventable. In this …

An update on the neurological short tandem repeat expansion disorders and the emergence of long-read sequencing diagnostics

SR Chintalaphani, SS Pineda, IW Deveson… - Acta Neuropathologica …, 2021‏ - Springer
Background Short tandem repeat (STR) expansion disorders are an important cause of
human neurological disease. They have an established role in more than 40 different …

[ספר][B] Amino acids: biochemistry and nutrition

G Wu - 2021‏ - taylorfrancis.com
Following its predecessor, the second edition of Amino Acids: Biochemistry and Nutrition
presents exhaustive coverage of amino acids in the nutrition, metabolism and health of …

[HTML][HTML] From anti-SARS-CoV-2 immune responses to COVID-19 via molecular mimicry

D Kanduc - Antibodies, 2020‏ - mdpi.com
Aim: To define the autoimmune potential of Severe Acute Respiratory Syndrome
Coronavirus 2 (SARS-CoV-2) infection. Methods: Experimentally validated epitopes …

Advancing genomic technologies and clinical awareness accelerates discovery of disease-associated tandem repeat sequences

T Gall-Duncan, N Sato, RKC Yuen… - Genome …, 2022‏ - genome.cshlp.org
Expansions of gene-specific DNA tandem repeats (TRs), first described in 1991 as a
disease-causing mutation in humans, are now known to cause> 60 phenotypes, not just …

Glial glutamine homeostasis in health and disease

JV Andersen, A Schousboe - Neurochemical Research, 2023‏ - Springer
Glutamine is an essential cerebral metabolite. Several critical brain processes are directly
linked to glutamine, including ammonia homeostasis, energy metabolism and …

Straglr: discovering and genoty** tandem repeat expansions using whole genome long-read sequences

R Chiu, IS Rajan-Babu, JM Friedman, I Birol - Genome biology, 2021‏ - Springer
Tandem repeat (TR) expansion is the underlying cause of over 40 neurological disorders.
Long-read sequencing offers an exciting avenue over conventional technologies for …

How to proceed after “negative” exome: A review on genetic diagnostics, limitations, challenges, and emerging new multiomics techniques

SB Wortmann, MM Oud, M Alders… - Journal of Inherited …, 2022‏ - Wiley Online Library
Exome sequencing (ES) in the clinical setting of inborn metabolic diseases (IMDs) has
created tremendous improvement in achieving an accurate and timely molecular diagnosis …