Diagnosis, prognosis, and treatment of leukodystrophies

MS van der Knaap, R Schiffmann, F Mochel… - The Lancet …, 2019‏ - thelancet.com
Leukodystrophies comprise a large group of rare genetic disorders primarily affecting CNS
white matter. Historically, the diagnostic process was slow and patient prognosis regarded …

Adulthood leukodystrophies

W Köhler, J Curiel, A Vanderver - Nature Reviews Neurology, 2018‏ - nature.com
The leukodystrophies are a group of inherited white matter disorders with a heterogeneous
genetic background, considerable phenotypic variability and disease onset at all ages. This …

Structure of the human respiratory complex II

Z Du, X Zhou, Y Lai, J Xu, Y Zhang, S Zhou… - Proceedings of the …, 2023‏ - pnas.org
Human complex II is a key protein complex that links two essential energy-producing
processes: the tricarboxylic acid cycle and oxidative phosphorylation. Deficiencies due to …

Patient-iPSC-derived kidney organoids show functional validation of a ciliopathic renal phenotype and reveal underlying pathogenetic mechanisms

TA Forbes, SE Howden, K Lawlor, B Phipson… - The American Journal of …, 2018‏ - cell.com
Despite the increasing diagnostic rate of genomic sequencing, the genetic basis of more
than 50% of heritable kidney disease remains unresolved. Kidney organoids differentiated …

Reanalysis of exome negative patients with rare disease: a pragmatic workflow for diagnostic applications

G Schobers, JH Schieving, HG Yntema, M Pennings… - Genome medicine, 2022‏ - Springer
Background Approximately two third of patients with a rare genetic disease remain
undiagnosed after exome sequencing (ES). As part of our post-test counseling procedures …

GGC Repeat Expansion of NOTCH2NLC in Adult Patients with Leukoencephalopathy

M Okubo, H Doi, R Fukai, A Fujita… - Annals of …, 2019‏ - Wiley Online Library
Leukoencephalopathies comprise a broad spectrum of disorders, but the genetic
background of adult leukoencephalopathies has rarely been assessed. In this study, we …

De novo mutations and rare variants occurring in NMDA receptors

W **angWei, Y Jiang, H Yuan - Current opinion in physiology, 2018‏ - Elsevier
Highlights•NMDAR rare variants are associated with various neuropsychiatric
disorders.•Functional consequences of variants differ among domains.•Evaluation of …

Pediatric palliative care in infants and neonates

BS Carter - Children, 2018‏ - mdpi.com
The application of palliative and hospice care to newborns in the neonatal intensive care
unit (NICU) has been evident for over 30 years. This article addresses the history, current …

Clinical application of next-generation sequencing to the practice of neurology

J Rexach, H Lee, JA Martinez-Agosto… - The Lancet …, 2019‏ - thelancet.com
Next-generation sequencing technologies allow for rapid and inexpensive large-scale
genomic analysis, creating unprecedented opportunities to integrate genomic data into the …

A population‐based cost‐effectiveness study of early genetic testing in severe epilepsies of infancy

KB Howell, S Eggers, K Dalziel, J Riseley… - …, 2018‏ - Wiley Online Library
Objective The severe epilepsies of infancy (SEI) are a devastating group of disorders that
pose a major care and economic burden on society; early diagnosis is critical for optimal …