A new study shows that a group of neurons, previously thought to die in the course of development, in fact become incorporated into the brain's cortex. This research has implications for understanding -- and possibly treating --several brain disorders.
In what researchers are calling a game changer for future ataxia treatments, a new study showed the ability to turn down the disease progression of the most common dominantly inherited ataxia, Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease. A single gene mutation causes this neurodegenerative disease, making it an ideal target for researchers.
Chromosomes occupy different territories in the nucleus; their arrangement and communication with each other is still poorly understood. Scientists from Berlin and Jena published in EMBO Journal findings about structural chromosomal aberrations which have an effect on genome organization (chromosomal kissing) and disease progression.
Researchers at King's College London have shown that rats with spinal cord injuries can re-learn skilled hand movements after being treated with a gene therapy that could be switched on and off using a common antibiotic.
Recently published research from the University of Georgia and UConn Health provides new insight about the basic biological mechanisms of the RNA-based viral immune system known as CRISPR-Cas.
A new study that compared six of the most promising adeno-associated viral (AAV) gene therapy vectors in human retinal organoid models showed clear distinctions in the efficiency of gene transfer to both retinal pigment epithelial (RPE) and photoreceptor cells.
Therapeutic use of gene editing with the so-called CRISPR-Cas9 technique may inadvertently increase the risk of cancer, according to a new study from Karolinska Institutet, Sweden, and the University of Helsinki, Finland, published in Nature Medicine. Researchers say that more studies are required in order to guarantee the safety of these 'molecular scissors' for gene-editing therapies.
A novel approach to immunotherapy developed by researchers at the National Cancer Institute (NCI) has led to the complete regression of breast cancer in a patient who was unresponsive to all other treatments. This patient received the treatment in a clinical trial led by Steven A. Rosenberg, M.D., Ph.D., chief of the Surgery Branch at NCI's Center for Cancer Research (CCR), and the findings were published June 4, 2018 in Nature Medicine.
Research to improve our understanding of Duchenne muscular dystrophy (DMD), and the development of new therapies, has previously relied on mouse models. However, physiological differences between the two species has limited how successfully findings in mice can be applied to humans. A newly developed rabbit model, created through the use of CRISPR/Cas-9 genome editing, exhibits greater clinical similarity to human patients than the mouse models currently in use, with huge potential to advance DMD research.
Rates of inherited mutations in genes other than BRCA1/2 are twice as high in breast cancer patients who have had a second primary cancer -- including, in some cases, different types of breast cancer -- compared to patients who have only had a single breast cancer.