An experimental gene therapy has shown promise in a Phase 1/2 study of patients with an inherited cause of progressive blindness called choroideremia. Six patients receiving the treatment have shown improvements in their vision six months after treatment, according to an article in The Lancet.
The potential of stem cells as future therapies could be linked to a greater understanding of how mitochondria are transferred among cells in the human body leading to the repair of damaged tissue, according to a paper in The EMBO Journal on 15 January.
Sanofi SA is initiating a new approach to antibiotic development with a decision to team up with the Fraunhofer-Gesellschaft in Germany to research naturally occurring chemical and biological compounds for their potential as medicines.
A group of international researchers has described how abnormalities in the human genome affect cognition, explaining why some people develop neurological diseases such as schizophrenia and autism, and others do not. The findings were reported online in the journal Nature on 18 December.
A new £3 million research project aimed at identifying targets for the development of drugs against dementia has been launched by the charity, Alzheimer’s Research UK; the technology transfer company, MRC Technology; and Eisai Co, Ltd and Eli Lilly and Company.
A more than decade-long Swedish project aimed at mapping the human proteome has reached an important milestone with the release of data for more than 80% of the protein-coding genes in the human body, and RNA expression data for more than 90% of the genes.
A candidate proteasome inhibitor developed by scientists at the Karolinska Institutet (KI) and Vivolux AB is set to enter the clinic next year following promising preclinical data in animal models of multiple myeloma. The data are described in an article in Blood.
An experimental cancer therapy that is being developed by Apogenix GmbH has reportedly induced programmed cell death in different animal models of colon cancer, according to an article in the December issue of Molecular Cancer Therapeutics.
Five-year follow-up data from a pioneering trachea transplantation using tissue-engineered materials has confirmed that the procedure is safe and effective, according to a report in The Lancet. The follow-up report takes into account a stent procedure carried out in the recovery period to address scarring.
Scientists at the University of Leicester in the UK have reported that a drug compound successfully halted the progression of prion disease in mice. The study, published in Science Translational Medicine, builds on earlier work in neurodegeneration.