gene-therapy

A two-headed molecule designed by Stanford Medicine researchers hijacks one of the most common protein drivers of B-cell lymphoma, flipping it from its role as a promoter of cell growth into an arbiter of cell death.

A first-in-class therapy to target MYC, one of the most sought-after and difficult targets in cancer biology, showed promise in hard-to-treat blood cancers, according to a new preclinical study from The University of Texas MD Anderson Cancer Center published in Blood.

Dr. Mina Sedrak is developing strategies to improve physical recovery and reduce frailty in older adults following hematopoietic cell transplantation, commonly known as a bone marrow or stem cell transplant.

(Nature) – It is 20 years since induced pluripotent stem cells shook biomedicine. Public trust must be retained now that clinical trials are well under way. Earlier this year, the first such therapies — one for Parkinson’s disease and the … Read More

Three fatalities occurring recently in China clinical trials have spurred two members of U.S. Congress to urge the FDA to enact new policies designed to protect American patients.

A new Northwestern Medicine study has uncovered key molecular design principles that could help supramolecular therapeutics cross the blood-brain barrier, a major challenge for this novel approach to treating neurological disorders. Published in ACS Nano, the study examined how subtle changes in the structure of molecules called peptide amphiphiles affect their ability to move through […]

In a new study, scientists from Johns Hopkins Medicine report that an experimental mRNA-based platform has the potential to help deliver next-generation mRNA therapeutics, including vaccines to fight infectious diseases, cancer and autoimmune conditions, faster and more efficiently than the industry standard. In experiments with cells from people and mice, researchers at Johns Hopkins Medicine [&…

A team of Rowan researchers is turning collaborative research into a startup focused on helping bones heal faster and more effectively.

A team of Rowan researchers is turning collaborative research into a startup focused on helping bones heal faster and more effectively.

Scribe Therapeutics’ one-time epigenetic treatment is designed to slash the adherence burden of chronic cholesterol-lowering therapies while sidestepping the irreversibility of gene editing.

Regeneron wins ultra-rare disease drug approval, a validating win for mRNA research, and more biotech news from The Readout

A Regeneron drug for an ultra-rare disease that causes bone to grow where it shouldn't won U.S. regulatory approval, capping a three-decade effort

From cancer cells to climate-resilient trees: how CRISPR is transforming research at VIB From decoding gene function to tackling cancer and building a sustainable future, VIB researchers are using CRISPR to push the boundaries of life science. A cancer cell escaping therapy. A yeast cell revealing the secrets of gene regulation. A poplar tree engineered without introducing foreign DNA. At first g…

et al. developed a DNA sequence module which enables splicing-mediated control of gene expression via an inducer which is already an FDA-approved medicine. An extremely useful advance for gene therapy since it allows activation of therapeutic genes only when we want them to turn on. I expect this kind of tool will be used heavily […]

Researchers have developed a new way to turn ordinary antibodies into tiny disease-fighting molecules that can work inside human cells, potentially opening new paths for treating Alzheimer’s, Parkinson’s, Huntington’s disease, and motor neurone disease.

In the absence of clear-cut regulation, who should decide on where and how a technology that could change the course of human health should be applied?