UNIVERSITY OF TORONTO IMAGE: PROFESSOR PHILIP M. KIM CREDIT: UNIVERSITY OF TORONTO Researchers at the University of Toronto and New York University have developed a novel technology that can engineer proteins to target any stretch of DNA in the human genome, opening a door toward gene therapies for a broader range of health conditions. The researchers fed data...
UNIVERSITY OF HOUSTON IMAGE: MUNA NAASH, UNIVERSITY OF HOUSTON JOHN S. DUNN ENDOWED PROFESSOR OF BIOMEDICAL ENGINEERING, IS EXPANDING A METHOD OF GENE THERAPY WITH THE HOPES IT WILL RESTORE VISION LOSS IN USHER SYNDROME TYPE 2A. CREDIT: UNIVERSITY OF HOUSTON A University of Houston researcher is expanding a method of gene therapy with the...
by UT Southwestern Medical Center These representative hearts from 12-week-old mice show a normal heart (left) and an enlarged heart that’s characteristic of dilated cardiomyopathy. Credit: UT Southwestern Medical Center Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse...
by Broad Institute of MIT and Harvard Credit: CC0 Public Domain Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed. Researchers have engineered viruses called adeno-associated viruses (AAVs) to deliver cargo—such as a functioning copy of a...
by University College London Schematic of the Activity-dependent Gene Therapy. Credit: Gabriele Lignani A new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells, has been developed by UCL researchers. Many brain diseases, such as epilepsy, are caused by excessive activity of a small number of brain cells. These...
by Beth Miller, Washington University School of Medicine in St. Louis In new research, Hong Chen and her team developed an intranasally delivered gene therapy method that had comparable or better outcomes than existing methods while having minimal effect on the body’s other organs. Credit: Chen lab Researchers have been experimenting with different ways to...
UT SOUTHWESTERN MEDICAL CENTER IMAGE: THIS IMAGE SHOWS A SEVERELY CYSTIC KIDNEY FROM A ADPKD MOUSE MODEL (LEFT). DELETING THE PKD1 MIR-17 BINDING SITE MARKEDLY REDUCES CYST GROWTH (RIGHT). YELLOW INDICATES KIDNEY COLLECTING DUCTS AND BLUE INDICATES NUCLEI. CREDIT: UT SOUTHWESTERN MEDICAL CENTER Blocking the inhibition of PKD1 and PKD2 gene expression by deleting a binding site for microRNAs...
by Cedars-Sinai Medical Center Credit: CC0 Public Domain Cedars-Sinai investigators have developed an investigational therapy using support cells and a protective protein that can be delivered past the blood-brain barrier. This combined stem cell and gene therapy can potentially protect diseased motor neurons in the spinal cord of patients with amyotrophic lateral sclerosis, a fatal...
While Sangamo’s sickle cell partnership with Sanofi ground to a halt earlier this year, the California biotech’s Fabry disease program, still in its early stages, has been quietly chugging along. In Tuesday’s update on the gene therapy trial, adding two more patients from its last interim readout in November, Sangamo reported that it has begun...
by Trinity College Dublin Rescue of ARPE19 cells insulted with NaIO3. 5.0 × 104 ARPE19 cells were transduced with AAV2/8-ophNdi1 5-h post-seeding; MOI = 5.4 × 105 (K–O). Twenty-eight-hour post-transduction cells were insulted with 5-mM NaIO3 (F–O) and 24-h post-insult cells were fixed and stained with Phalloidin-iFluor 647 (F-actin, light blue), and CPN60 (mitochondrial marker, magenta) and 8-OHdG-Alexa Fluor 488 (oxidative stress marker, green) immunocytochemistries;...
