Results 31 to 40 of about 98,806 (295)

Evaluation of a new ELISA assay for monoclonal free‐light chain detection in patients with cardiac amyloidosis

open access: yeseJHaem, 2022
The causal protein of amyloid light‐chain (AL) amyloidosis is a monoclonal immunoglobulin free light chain (mFLC), which must be quantified in the serum for patient diagnosis and monitoring.
Hajer Abroud   +18 more
doaj   +1 more source

Allosteric Modulation of Pathological Ataxin‐3 Aggregation: A Path to Spinocerebellar Ataxia Type‐3 Therapies

open access: yesAdvanced Science, EarlyView.
This study uncovers a new allosteric site in the Josephin domain of ataxin‐3 targeted by the molecular tweezer CLR01, which modulates protein aggregation, improves synaptic function in neuronal cells, and delays motor dysfunction in animal models.
Alexandra Silva   +28 more
wiley   +1 more source

MSC Membrane‐Coated circPROSC‐siRNA Nanoparticles for Ameliorating Craniosynostosis by Inhibiting Premature Suture Ossification

open access: yesAdvanced Science, EarlyView.
CircPROSC promotes craniosynostosis by sponging miR‐6815‐5p, thereby activating WNT3A/β‐catenin signaling and enhancing RUNX2‐mediated osteogenesis. Targeted silencing of circPROSC with MSC membrane–coated siRNA nanoparticles (MM@Lipo/si‐circPROSC) delivered postnatally into cranial sutures attenuates premature suture closure in Twist1+/– mice ...
Zhenkun Weng   +6 more
wiley   +1 more source

Real-world treatment management in hereditary transthyretin amyloidosis – an experience report and proposal for therapy switch decision criteria

open access: yesNeurological Research and Practice
Background Hereditary transthyretin amyloidosis is a rapidly progressive and lethal disease. Thanks to the increasing number of disease-modifying treatments, prognosis has improved significantly.
Duc Chu Dieu   +9 more
doaj   +1 more source

Transplantation of GABAergic Interneuron Progenitors Restores Cortical Circuit Function in an Alzheimer's Disease Mouse Model

open access: yesAdvanced Science, EarlyView.
Transplantation of medial ganglionic eminence (MGE) interneuron progenitors into APP/PS1 cortices restored the slow oscillation characteristic of Alzheimer's disease. Donor cells survived, migrated, and matured into functional GABAergic interneurons, forming synaptic connections.
Shinya Yokomizo   +16 more
wiley   +1 more source

Nanoscopic Mapping of the Extracellular Space in Amyloid Plaque‐rich Cortex

open access: yesAdvanced Science, EarlyView.
The extracellular space and diffusion around amyloid plaques are examined using shadow imaging and single‐particle tracking. Increased diffusivity is found near plaques, extracellular matrix alterations, and plaque core penetrability that varies with amyloid phenotype.
Juan Estaún‐Panzano   +11 more
wiley   +1 more source

Genomics Review of Selective RET Inhibitors Sensitivity in Thyroid Cancer Clinical Trials

open access: yesAmerican Journal of Medical Genetics Part C: Seminars in Medical Genetics, EarlyView.
ABSTRACT RET gene is a driver of thyroid cancer (TC) tumorigenesis. The incidence of TC has increased worldwide in the last few decades, both in medullary and follicular‐derived subtypes. Several drugs, including multikinase and selective inhibitors, have been explored.
Sara Gil‐Bernabé   +5 more
wiley   +1 more source

Clinical profile and treatment outcome of older (>75 years) patients with systemic AL amyloidosis

open access: yesHaematologica, 2015
Systemic AL amyloidosis, a disease with improving outcomes using novel therapies, is increasingly recognized in the elderly but treatment and outcomes have not been systematically studied in this group of patients in whom comorbidities and frailty may ...
Sajitha Sachchithanantham   +10 more
doaj   +1 more source

Amyloidosis and anesthesia

open access: yesAnesthesia: Essays and Researches, 2017
The aim of this article is to provide a view of amyloidosis and discuss implications for the anesthetic management of patients with this condition.Urine samples from patients with plasma cell dyscrasias were obtained from a urine bank that gathers urine samples from patients who gave research use consent for specimens that would otherwise be considered
Wani, Zara   +2 more
openaire   +3 more sources

AL Amyloidosis [PDF]

open access: yesOrphanet Journal of Rare Diseases, 2012
Abstract Definition of the disease AL amyloidosis results from extra-cellular deposition of fibril-forming monoclonal immunoglobulin (Ig) light chains (LC) (most commonly of lambda isotype) usually secreted by a small plasma cell clone.
Desport, Estelle   +13 more
openaire   +4 more sources

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