research News

The brain is indeed a target for treating ALS (amyotrophic lateral sclerosis), Northwestern Medicine scientists have discovered. This flips a long-standing belief that the disease starts in the spinal motor neurons and any therapy would need to target the spine as the key focus. A new Northwestern study shows the degeneration...
It has been shown that constitutively activated STAT3 is detected in many HCC cell lines and tissues. This suggests that STAT3 is a promising molecular target for HCC gene therapy. RNA interference (RNAi) is a post-transcriptional gene-silencing mechanism, in which the homologous RNA sequences could be introduced into cells that...
In a new study, researchers found that pediatric patients with sickle cell anemia (SCA) showed signs of impaired pulmonary function compared with control individuals without SCA. The study was a systematic review and meta-analysis conducted by a research team led by Amar Taksande, MD, of Jawaharlal Nehru Medical College in...
A new diagnostic technique which may greatly simplify the detection of Alzheimer’s disease has been discovered by researchers at McGill University and the affiliated Lady Davis Institute for Medical Research at Montreal’s Jewish General Hospital (JGH). Their results were published June 8 in the Journal of Alzheimer’s Disease. There is...
BETHESDA, Md. and WASHINGTON – The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD®) today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare bone marrow failure disease. PNH is characterized by the destruction of red blood cells, blood clots and impaired...
PASADENA, Calif. – Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced ARO-DUX4 as Arrowhead’s first muscle targeted investigational RNAi therapeutic candidate to utilize its proprietary Targeted RNAi Molecule (TRIMTM) platform. ARO-DUX4 is designed to target the gene that encodes human double homeobox 4 (DUX4) protein as a potential treatment for patients...
Worcester, MA — Advanced Cell Technology, Inc. (OTC:ACTC.PK) and its collaborators at OHSU reported today the long-term safety and efficacy of human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) produced under manufacturing conditions suitable for human clinical trials. Two important early potential hESC applications are the use of RPE...