Tag: Gene Therapy

Chemical and biomolecular engineer Xue Sherry Gao of Rice University's Brown School of Engineering has won National Institutes of Health support for a new strategy to fight cystic fibrosis. The agency awarded Gao a four-year R01 grant of more than $2 million to adapt tools developed by her lab that increase the accuracy of CRISPR/Cas9-based

Findings A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule. This new approach to CAR T gene therapy — a type of immunotherapy that

Intravitreal injections with aflibercept (Eylea) don’t improve the visual acuity of people with nonproliferative diabetic retinopathy (NPDR), researchers say. The treatments do reduce the risk for center-involved diabetic macular edema and proliferative diabetic retinopathy, however, said Adam R. Glassman, MS, principal investigator of the DRCR Retina Network Coordinating Center, Tampa, Florida. “Some clinicians may decide

Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells. The trial will combine CRISPR technology developed at Innovative Genomics

Scientists at Nanyang Technological University, Singapore (NTU Singapore) have developed a technique to observe how radiation damages molecules over time-frames of just one quadrillionth of a second — or a femtosecond. The technique involves dissolving organic molecules in water to simulate the state molecules are found in biological tissue. This allows the research team to

A worldwide coalition of researchers and clinicians has agreed that light therapy is among the most effective interventions for the prevention of oral mucositis, painful ulcers in the mouth resulting from cancer therapy. The new guidelines from the Multinational Association of Supportive Care in Cancer (MASCC) and International Society of Oral Oncology (ISOO), published on

The Han Chinese are the world’s largest ethnic group, making up 91.6% of modern-day China. As DNA sequencing tools and statistical analyses software have advanced, scientists have been exploring the forces that helped shape the current genetic landscape of Han Chinese. Now, in a new study drawing from the largest study to date of three

Bochum-based psychologists have studied how the application of the stress hormone cortisol affects exposure therapy for anxiety disorders. The researchers knew from earlier studies that extinction learning, which constitutes the foundation of exposure therapy, can be reinforced by administering cortisol. However, the team headed by Professor Armin Zlomuzica at Zentrum für Psychotherapie (psychotherapy centre) at

What makes a biological clock tick? According to a new study from U of T Mississauga, the surprising answer lies with a gene typically associated with stem and cancer cells. In the first study of its kind for the field of circadian biology, UTM researchers used RNA sequencing to observe the expression of genes in

A team of researchers from New York University has engineered nanoscale protein micelles capable of both delivering chemotherapeutic drugs and of being tracked by magnetic resonance imaging (MRI). The innovation falls into the category of “theranostics,” meaning that it combines diagnostic capability and drug delivery, allowing researchers to administer therapy while also non-invasively monitoring the

Malaria, which claims hundreds of thousands of lives each year — mainly children and especially in Africa -, is one of the leading causes of death by an infectious agent, the Plasmodium falciparum parasite. In research on malaria, the genetic mutation that causes sickle cell anemia (also known as drepanocytosis), a chronic disease that is

A research group headed by Professor Outi Mäkitie, University of Helsinki, Finland, identified in families with childhood-onset osteoporosis disease-causing mutations in a gene that had previously not been connected with the skeletal system or osteoporosis. “Through extensive genetic research, we identified a gene defect underlying osteoporosis in two Finnish families with several affected family members.

Medical researchers at Melbourne’s Murdoch Children’s Research Institute have made a new discovery about how a baby’s sex is determined — it’s not just about the X-Y chromosomes, but involves a ‘regulator’ that increases or decreases the activity of genes which decide if we become male or female. The study, ‘Human Sex Reversal is caused

The CRISPR genome editing technique promises to be a “transformative leap” in genetic engineering and therapy, affecting almost every area of medicine. That includes plastic surgery, with potential advances ranging from prevention of craniofacial malformations, to therapeutic skin grafts, to new types of rejection-free transplants, according to a paper in the November issue of Plastic

Parents of newborns may be familiar with the metabolic disorder phenylketonuria: in Switzerland, all newborn babies are screened for this genetic disease. If a baby is found to have phenylketonuria, it needs a special diet so that the amino acid phenylalanine does not accumulate in the body. Excess phenylalanine delays mental and motor development. If

An interdisciplinary and international research group led by Dr. Volker Busskamp from the Center for Regenerative Therapies Dresden at the TU Dresden (CRTD) has decoded the regulatory impact on neuronal survival of a small non-coding RNA molecule, so-called miRNA, at the highest resolution to date. This deciphering of gene regulation primes applications for strengthening neurons

From the DNA of nearly 300,000 veterans, scientists have singled out a handful of genetic mutations that not only govern levels of cholesterol, but may also inform the development and use of drugs for cardiovascular disease and diabetes, according to researchers at the Stanford University School of Medicine and the Palo Alto Veteran Affairs Health

People with deep-vein thrombosis (DVT) can substantially cut their risk of potentially debilitating complications by starting adequate compression therapy in the first twenty-four hours of DVT therapy (known as the acute phase of treatment), suggests a study published today in the journal Blood. DVT occurs when a blood clot forms in a deep vein, commonly

Every cell in our body has the same set of genes, or genome, and can potentially become any type of cell. During development, the epigenome mediates the process that leads a cell to become a skin cell or a neuron, for instance. If the genome is like computer hardware, then the epigenome is the software

Lipid droplets: they were long thought of merely as the formless blobs of fat out of which spare tires and muffin tops were made. But these days, they’re “a really hot area of research,” says Michael Welte, professor and chair of biology at the University of Rochester. That’s in part because lipid droplets have been