Results 81 to 90 of about 75,494 (295)
Treating Hearing Loss: From Cochlear Implantation to Gene Therapy
Advanced Science, EarlyView.Cochlear implantation is the primary treatment for deafness, restoring functional hearing in over a million people. Recently, gene therapy has enabled biological hearing restoration in a small number of patients with OTOF‐related mutations. This perspective evaluates both approaches, concluding that cochlear implants will remain the standard for most ...
Fan‐Gang Zeng +4 more
wiley +1 more source
Effect of the Butyrate Prodrug Pivaloyloxymethyl Butyrate (AN9) on a Mouse Model for Spinal Muscular Atrophy. [PDF]
, 2016 Spinal muscular atrophy (SMA) is an early-onset motor neuron disease that leads to loss of muscle function. Butyrate (BA)-based compounds markedly improve the survival and motor phenotype of SMA mice.
Butchbach, Matthew E.R. +1 more
core +2 more sources
Advanced Science, EarlyView.In this study, it is discovered that pathological accumulation of Runx2+ Schwann cell clusters during axonal regeneration is a key factor impairing nerve repair in aging. This pathology is associated with dysregulation of stress granule homeostasis.
Peilin Wang +10 more
wiley +1 more source
Pathogenesis of proximal autosomal recessive spinal muscular atrophy [PDF]
, 2008 Although it is known that deletions or mutations of the SMN1 gene on chromosome 5 cause decreased levels of the SMN protein in subjects with proximal autosomal recessive spinal muscular atrophy (SMA), the exact sequence of pathological events leading to ...
Šimić, Goran
core +1 more source
Parabiosis, Assembloids, Organoids (PAO)
Advanced Science, EarlyView.This review evaluates parabiosis, organoids, and assembloids as complementary disease models spanning systemic, organ, and multi‐organ levels. It highlights their construction strategies, applications, and current limitations, while emphasizing their integration with frontier technologies such as artificial intelligence, organ‐on‐a‐chip, CRISPR, and ...
Yang Hong +5 more
wiley +1 more source
Spinal Muscular Atrophy and Arthrogryposis
Pediatric Neurology Briefs, 1997 Four infants with neurogenic arthrogryposis who died of respiratory failure before 1 month of age had DNA testing of autopsy specimens for SMNT gene deletion in a study at the Children’s Hospital of Philadelphia, PA, and the Children’s Hospital at ...
J Gordon Millichap
doaj +1 more source
Advanced Science, EarlyView.Merging 4D printing with magneto‐responsive shape memory polymers opens new avenues for intelligent, reconfigurable systems. This review navigates cutting‐edge fabrication techniques, magnetic fillers, and smart polymer matrices, unveiling their potential in soft robotics, biomedical devices, and wearable tech.
Kiandokht Mirasadi +7 more
wiley +1 more source
Early stages of building a rare disease registry, methods and 2010 data from the Belgian Neuromuscular Disease Registry (BNMDR) [PDF]
, 2015 The Belgian Neuromuscular Disease Registry, commissioned in 2008, aims to collect data to improve knowledge on neuromuscular diseases and enhance quality health services for neuromuscular disease patients.
BNMDR Scientific Committee, the +7 more
core +2 more sources
Microgels for 3D Biofabrication
Aggregate, EarlyView.This review highlights microgels as a versatile platform advancing biomanufacturing and tissue regeneration. It details their fabrication methods, core functions (bioprinting inks, suspension support baths, porous sacrificial scaffolds), and pivotal applications (drug delivery, dynamic cell culture microenvironments, tissue engineering scaffolds ...
Ting Xie +11 more
wiley +1 more source
, 2012 Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by degeneration of alpha motor neurons resulting in hypotonia, progressive muscular weakness and atrophy.30 Spinal muscular atrophy is one of the leading hereditary causes of infant mortality,31 it comprises the second most common fatal progressive diseases after cystic fibrosis.28
openaire +3 more sources