Gene Therapy

New Clinical Trial Greatly Improves Genetic Rickets

A drug developed in part by Indiana University School of Medicine to alleviate the symptoms of X-linked hypophosphatemia (XLH), a disease which softens bones, has proven significantly more effective than conventional therapies.

Gene Therapy Could Heal Cells After Heart Attack

Gene therapy can make heart cells regenerate after a heart attack, King’s College London researchers have found. The study, published in Nature, stated that a small piece of genetic material called microRNA-199 delivered into a pig’s heart after myocardial infarction resulted in almost total cardiac recovery a month later.

New Cancer Pathway Opens Up New Treatment Possibilities

When the MDM2 gene acts with a specific protein found in cancer cells’ mitochondria, it can lead to cancer cell death. The study which discovered this fact, published by Mount Sinai researchers in Molecular Cell journal, could open new treatment opportunities for cancer patients in the future.

CEPI Provides $8.4 Million Grant to Fight “Disease X”

A new deal created by the Coalition for Epidemic Preparedness Innovations (CEPI) will use $8.4m to facilitate development of a vaccine platform to fight unknown pathogens. Under the deal, Imperial College London will work to create a self-amplifying RNA vaccine platform, which can then be made to rapidly develop anti-pathogen vaccines.

CRISPR Repairs DNA Even Without Donor Template

CRISPR-Cas9 can carry out precise genome editing even without the assistance of donor DNA templates, a team of scientists from Brigham and Women’s Hospital and the Broad Institute of MIT and Harvard have found.

Maize genome complexity traversed with Oxford Nanopore technology

  Maize is one of the most economically important crops globally and much effort has been spent generating the high quality B73 reference genome. However, the 10 chromosome, 2.3 gigabase (Gb) B73 reference genome was a substantial challenge due to the fact it is comprised of 85% transposable elements, 75% of which are long terminal […]