StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

The article by Sarfaraz K. Niazi, PhD, argues that the FDA’s classification of future copies of messenger RNA (mRNA) products ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

We recently published a list of 10 Most Promising Gene Editing Stocks to Buy According to Hedge Funds. In this article, we ...

Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where it performs its gene ...

In addition, they add that there is a need for the harmonisation of definitions across regulatory agencies, with mRNA vaccines sometimes labelled as gene therapy, while others refer to gene ...

The in vivo cell therapy seed Carisma Therapeutics planted with Moderna in 2022 is starting to sprout. | The in vivo cell ...

Delivery of mRNA using LNP is already transforming disease treatments. It has applications in vaccine development, gene editing and protein replacement therapy. Recently, mRNA delivery has become ...

The scientists recently described their low-toxicity carriers in ACS Nano, in an article titled, “Widespread Gene Editing in the Brain via In Utero Delivery of mRNA Using Acid-Degradable Lipid ...