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A team of UT Southwestern researchers has identified a gene involved in the growth of breast cancer, a finding that could lead to potential new targets for treatment. “The gene ZMYND8 is increased in breast cancer conditions, and higher levels of the gene correlate with poor survival of breast cancer patients,” says Yingfei Wang, Ph.D., […]
The Translational Genomics Research Institute (TGen), an affiliate of City of Hope, has identified a specific genetic target that could help explain the tremendous variation in how sick those infected with COVID-19 become. The study results, recently published in the journal mSphere, describe a molecule made from DNA—miR1307—as a potential dimmer switch that may influence […]
Treating tumors presents a tricky conundrum: a higher dose of radiation is more effective for treatment, but the higher the dose, the more danger it poses to healthy cells. Researchers at Yale have made significant inroads into solving this problem in a paper titled, “LRRC31 inhibits DNA repair and sensitizes breast cancer brain metastasis to […]
Use of thyroid hormone to boost hepatocyte proliferation enhanced the efficiency of CRISPR/Cas9-mediated gene correction in the mouse liver. This dietary induction of hepatocyte regeneration may be a viable clinical strategy to enhance gene repair in the liver, according to the peer-reviewed journal Human Gene Therapy. The study was done in a mouse model of […]
Many people think of new medicines as bullets, and in the pharmaceutical industry, frequently used terms like “targets” and “hits” reinforce that idea. Immuneering co-founder and CEO Ben Zeskind ’03, Ph.D. ’06 prefers a different analogy. His company, which specializes in bioinformatics and computational biology, sees many effective drugs more like noise-canceling headphones. Rather than […]
A lone genetic mutation can cause a life-changing disorder with effects on multiple body systems. Lysosomal storage diseases, for example, of which there are dozens, arise due to single mutations that affect production of critical enzymes required to metabolize large molecules in cells. These disorders affect multiple organs including, notably, the brain, causing intellectual disability […]
Many of the diseases caused by point mutations have no established therapeutic approaches. Prof. Tsukahara and colleagues (Japan Advanced Institute of Science and Technology) are studying a therapeutic method using artificial RNA editing. Artificial site-directed RNA editing is an important technique for modifying genes and ultimately regulating protein function. We are trying to modify the […]
Myeloproliferative neoplasms are blood diseases that affect the normal production of blood cells in the bone marrow. According to the World Health Organization (WHO) classification, myeloproliferative neoplasms are divided into 4 major types. These include chronic myeloid leukemia, polycythemia vera, essential thrombocythemia, and primary myelofibrosis. Over the last several years, there have been new research […]
A pilot gene therapy treatment for sickle cell disease, restoring patients’ ability to make fetal hemoglobin, has produced good results in the first three patients to receive it. Investigators at Boston Children’s Hospital reported the findings of their ongoing clinical trial this week at the American Society of Hematology (ASH) annual meeting. The three adult […]
A breakthrough gene therapy treatment for Haemophilia A has been shown to offer long-term benefits that have already transformed the lives of 13 men in the UK. Clinical researchers led by Professor John Pasi from Queen Mary University of London first announced in 2017 that 85% of men treated with a single infusion of a […]