Genetically‐targeted control of the spinal cord and peripheral nervous system
Application of genetically‐targeted stimulation for treating diseases or injuries outside the brain. Optogenetic and chemogenetic systems have been developed for dysfunction in the cochlea, spinal cord, dorsal root ganglia (DRGs), peripheral nerves, lower urinary tract, and enteric nervous system (ENS). Created with Biorender.com.
Anakaren Romero Lozano+3 more
wiley +1 more source
Adeno-Associated Virus-Engineered Umbilical Cord-Derived Mesenchymal Stromal Cells Overexpressing Human sFlt-1 for Anti-Angiogenesis. [PDF]
Choy EY+4 more
europepmc +1 more source
MR‐Guidance of Gene Therapy for Brain Diseases: Moving From Palliative Treatment to Cures
ABSTRACT Regulatory bodies in the U.S. and Europe recently approved a gene therapy for aromatic L‐amino acid decarboxylase (AADC) deficiency, a rare neurologic disorder where a genetic mutation prevents dopamine production in the brain. Affected children fail to develop normal motor and cognitive functions.
Dalton H. Bermudez+2 more
wiley +1 more source
An SDS-NaOH-based method to isolate genome of recombinant adeno-associated virus vectors for physical titer measurement. [PDF]
Zhu X+11 more
europepmc +1 more source
Severe hepatitis in pediatric patients: The Severe Hepatitis In Pediatric Patients (SHIPP) registry
Abstract Objectives Clusters of severe acute hepatitis in children were reported worldwide beginning in October 2021. Although most children recovered, some progressed to liver failure leading to death or liver transplantation. Herein, we characterize the clinical characteristics, and outcomes of this group of children through an international ...
Mohit Kehar+31 more
wiley +1 more source
Model-Based Characterization of the Metabolism of Recombinant Adeno-Associated Virus (rAAV) Production via Human Embryonic Kidney (HEK293) Cells. [PDF]
Khodadadi Karimvand S+3 more
europepmc +1 more source
Abstract Objectives Paediatric acute liver failure (PALF) is a rare but life‐threatening condition, yet comprehensive epidemiological data in Germany are lacking. Our study aimed to systematically analyse incidence, aetiology, and outcome of PALF in Germany.
Dominic Lenz+40 more
wiley +1 more source
Transcription factor EB activation rescues advanced αB-crystallin mutation-induced cardiomyopathy by normalizing desmin localization [PDF]
Benjamin, Ivor J+12 more
core +1 more source
Bee products as alternatives in the treatment of viral infections
Abstract Medicines used in the treatment of viral infections usually reduce symptoms. There is a need to develop drugs that inhibit the viruses and do not merely relieve the symptoms. Natural bee products possess many pharmacological properties and are widely used in folk medicine. There are many studies on the antibacterial effects of bee products but
Michał Otręba+3 more
wiley +1 more source