Tag: Gene Therapy

Scientists develop technique to observe radiation damage over femtoseconds

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

How cortisol affects exposure therapy for anxiety disorders

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

‘Master pacemaker’ for biological clocks identified

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

Visualizing better cancer treatment: Researchers engineer a protein micelle that can be visualized by MRI as it delivers hemotherapeutics

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

Earlier emergence of malaria in Africa

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

New form of hereditary osteoporosis

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.

CRISPR gene editing will find applications in plastic and reconstructive surgery

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

Genetic disease healed using genome editing

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

Decoding the regulation of cell survival: A major step towards preventing neurons from dying

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

Immediate compression could help prevent complications after deep-vein thrombosis: Study supports use of this simple, low-cost intervention even for patients without symptoms

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

Lipid droplets play crucial roles beyond fat storage

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

Stem cell research for cystic fibrosis leaps forward

The fight against cystic fibrosis (CF) has taken a major step forward, with pioneering research by University of Adelaide scientists showing that cells causing the debilitating genetic disorder could be successfully replaced with healthy ones. The research published in the journal Stem Cell Research and Therapy applies cell transplantation therapy, normally used in bone marrow

Next generation ALS drug silences inherited form of the disease in animal models: Preclinical study suggests drug may be ready for early stage clinical trials

NIH-funded researchers delayed signs of amyotrophic lateral sclerosis (ALS) in rodents by injecting them with a second-generation drug designed to silence the gene, superoxide dismutase 1 (SOD1). The results, published in the Journal of Clinical Investigation, suggest the newer version of the drug may be effective at treating an inherited form of the disease caused

New platform poised to be next generation of genetic medicines: The novel tool has the potential to treat a variety of genetic diseases

A City of Hope scientist has discovered a gene-editing technology that could efficiently and accurately correct the genetic defects that underlie certain diseases, positioning the new tool as the basis for the next generation of genetic therapies. This editing platform, discovered by City of Hope’s Saswati Chatterjee, Ph.D., eventually may be used to cure inherited