The lentiviral vector delivers the SGSH gene to bone marrow cells, which, when implanted into the body are able to traffic to both the bone marrow and the brain and deliver SGSH enzyme throughout the ...

Sep. 16, 2024 — CREME is a virtual laboratory that allows scientists to simulate specific decreases in gene activity. It offers a powerful new tool for identifying and understanding important ...

Fruit flies aiding in research for Sanfilippo syndrome treatment. Researchers at Clemson University study enzyme defects and ...

Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the new results of an ...

Not all genes are active at all times. DNA methylation is one of several epigenetic mechanisms that cells use to control gene expression. This straightforward experiment demonstrated that it was ...

A scientist in China who said he had created the world's first gene-edited babies has been jailed for three years. He Jiankui was convicted of violating a government ban by carrying out his own ...

Gene therapy and gene editing are on the cutting edge of modern biotechnology. Gene therapies are used to correct genetic abnormalities by introducing genetic material at a cellular level and can ...

How Is Gene Expression Regulated? The amounts and types of mRNA molecules in a cell reflect the function of that cell. In fact, thousands of transcripts are produced every second in every cell.

It programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo ...

After that, the next step in CRISPR gene editing is usually to alter that piece of DNA. However, CRISPR has also been adapted to do other things too, such as turning genes on or off without ...

Current scientific techniques are not yet safe or effective enough to be used to create gene-edited babies, an international committee says. The technology could one day prevent parents from ...

Gene Park is a reporter for The Washington Post, covering video games and gaming culture. He joined The Post in 2015 as audience editor, and is a former reporter in the West Coast and Pacific region.