Replacing the PKP2 gene prolongs survival in ARVC patients
The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
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The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
How pure consistent and functional lower motor neurons can be precision reprogrammed from iPSCs for motor neuron disease research and drug discovery.
In vitro and in vivo experiments highlight the advantages of a new targeted spectroscopy system for many eye diseases.
Researchers found heightened SCAN enzyme activity in humans and mice with diabetes results in excessive nitric oxide on insulin receptors.
Advancements in enzyme-activated near-infrared fluorescent probes hold promise for evaluating responses to enzyme-targeting therapies.
Mitochondrial fragmentation mechanism that can cause ventilator-induced diaphragm dysfunction (VIDD) found, and a therapeutic to mitigate it.
Research suggests immune and non-immune cells can be reprogrammed by changing how nutrients are used.
A collaborative team has uncovered a method to extend the functionality of CAR-T cells.
Researchers from Brazil discovered that survival of the parasitic worm that cause the disease schistosomiasis, depends on expression of a specific type of RNA. In animal trials, inhibition of the molecule interrupted the infection.
This article is the second part of Drug Target Review’s Izzy Wood’s discussion with Olivia Cavlan, Chief Corporate Development and Strategy Officer at Alchemab Therapeutics Ltd, exploring the role of AI in target discovery, its applications in personalised medicine, and the evolving landscape of pharmaceutical development.
Japanese researchers successfully engineered iPSCs to secrete a modified enzyme, mNAGA, and restored enzyme activity in vitro and in a mouse model, opening new avenues for regenerative medicine for conditions such as Fabry Disease.
US researchers have uncovered a novel method using a tool used to visualise ion channels in mechanosensory neurons.
New imaging approach reveals that changes in retinal microcirculation may indicate cerebrovascular diseases that involve reduced blood flow.
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USC researcher Dr Steven Gazal and his team have identified human genome base pairs that have remained constant over millions of years of mammalian evolution. These base pairs are linked to human disease. Using DNA from 240 mammal species, they identified genetic variations affecting an individual's survival and reproduction.