Study suggests postnatal gene therapy could treat Pitt-Hopkins syndrome
Researchers have found that restoring lost gene activity prevents many disease signs in an animal model of Pitt-Hopkins syndrome.
List view / Grid view
News
Mouse study shows lab-grown, self-sustainable muscle cells could repair diseases such as muscular dystrophy
A new study highlights how scientists have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice, findings that could lead to therapeutics to treat muscular dystrophy disorder.
CRISPR-dCas9 gene editing reverses brain genetic reprogramming caused by adolescent binge drinking
A new study in animals has shown that gene editing may be a potential treatment for anxiety and alcohol use disorder in adults who were exposed to binge drinking in their adolescence.
Retinal cell map could advance precise cell and gene therapies for retinal diseases
Researchers have identified distinct differences among the cells comprising a tissue in the retina, findings that could help develop precise therapies for retinal diseases.
Mesenchymal stem cell therapy may supress side effects from cancer drugs
In a new study, researchers from Osaka University have highlighted that mesenchymal stem cells could repress type 1 diabetes caused by cancer drugs.
New research could bring life-saving benefits of CAR T therapy to AML patients
Researchers have developed a novel method for enhancing CAR T therapy through a drug combination and cellular engineering that improves the strength and durability of the tumour-killing effect of a CAR T directed against AML.
A new target for CAR T cells in solid tumours
In a new study, researchers have shared the identification of a new potential target for CAR T cells that inhibits growth in lung and ovarian tumours.
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.
New discovery could open ways to repair damage in the human body after stroke or heart attack
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.
Integrating genomic data from different ancestries reduces bias in predicting disease risk
Researchers have developed a promising new tool that accurately uses genomic data to predict disease risk across diverse populations.
Using CRISPR to improve lung cancer treatments
Scientists have developed CRISPR with a new process that evaluates the impacts of gene edits that alter rather than deleting DNA code.
A revolutionary deep learning model to predict undesired drug-drug interactions
Scientists have developed a deep learning-based model trained on drug-induced gene expression signatures to predict drug-drug interactions.
Researchers use brain organoids to identify neural abnormalities and test gene therapy tools
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.
Study shows muscular dystrophies can be repaired with CRISPR-Cas9
Researchers have introduced a CRISPR-Cas9 tool into human muscle stem cells using mRNA, thus discovering a suitable method to treat muscular dystrophies.
Cilia-free stem cells could open a new way to study rare diseases
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.
