Gene therapy: A potential cure for growth-hormone resistant dwarfism
Researchers have shown that gene therapy using a single-dose injection of a virus carrying the ‘good’ gene could possibly be used to cure growth-hormone resistant dwarfism.
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Researchers have shown that gene therapy using a single-dose injection of a virus carrying the ‘good’ gene could possibly be used to cure growth-hormone resistant dwarfism.
New data from high-resolution imaging has revealed the origin of circulatory system during development, a discovery that could help advance therapeutics for various vasculature-related pathologies.
Researchers have developed a promising new tool that accurately uses genomic data to predict disease risk across diverse populations.
Scientists have developed CRISPR with a new process that evaluates the impacts of gene edits that alter rather than deleting DNA code.
Scientists have developed a deep learning-based model trained on drug-induced gene expression signatures to predict drug-drug interactions.
Scientists used human brain organoids to reveal how a genetic mutation associated with a profound form of autism disrupts neural development, and gene therapy tools to recover the gene’s function effectively rescued neural structure and function.
Researchers have introduced a CRISPR-Cas9 tool into human muscle stem cells using mRNA, thus discovering a suitable method to treat muscular dystrophies.
A new study has shown that cilia-free human pluripotent stem cells could help scientists understand the causes of polycystic kidney disease and other cilia-linked illnesses.
Scientists have engineered a new class of material which is able to both withstand the harsh acidic conditions of the stomach and then dissolve predictably in the comparatively gentle environment of the small intestine.
Using nose organoids, researchers have highlighted key differences between the infection by SARS-CoV-2 and that of respiratory syncytial virus.
Neuroscientists have designed brain organoids to contain both mature neurons and astrocytic glial cells in relative proportions like the human brain.
Scientists have gained new insights into the way G protein-coupled receptors operate, a step toward the development of improved drugs with fewer side effects.
Researchers have discovered that failed epigenetic suppression of an X-chromosome gene called Xist leads to miscarriage and developmental abnormalities.
The University at Buffalo has received a $2.3 million grant from the US National Cancer Institute to identify metabolic vulnerabilities of ovarian cancer and to develop potential treatments for the disease.
In a new study, researchers have demonstrated the efficiency of T-cell immune response against the Omicron variant of SARS-CoV-2.