Gene Therapy

Using a well-established non-human primate model, researchers were able to successfully treat alcohol use disorder with a gene therapy targeting the dopamine reward system.

In December 2018, a Chinese researcher, He Jiankui, shocked the world when he revealed the birth of the world’s first genetically edited babies. While it is clear that Jiankui egregiously violated university regulations and ethical standards, his…

In an exciting step forward, dogs with Duchenne muscular dystrophy (DMD) were treated with the CRISPR genome editor to restore production of the missing protein responsible for the disease. After more studies to prove it is safe and effective long-term,…

The discovery of CRISPR/Cas9 is one of the biggest scientific breakthroughs in recent memory. This technology, first discovered in microbes, enables the cleaving of DNA in order to remove or replace existing genes. For a crash course into the history of…

When you think of viruses, the yearly flu or even the Ebola or Swine flu outbreaks may come to mind. However, not all viruses cause disease – some even provide cures! Adeno-associated virus (AAV) can infect humans, but is not known to cause disease. In…

CRISPR 2.0 is causing quite the ruckus in the scientific community. Why? Imagine that you had written a note in permanent marker, but later decided you wanted to change a single word. Without the ability to erase, your options would be limited, and…

Gene therapy is an approach to treating genetic diseases by re-introducing a functional copy of a gene into cells to replace the mutated, disease-causing gene. To get these genes into the cells, scientists create a vector by packaging the DNA encoding…

Image of a mouse embryo, different cell types shown by different colors [Image: B0008305 Mouse embryo under a CC BY-NC-ND 2.0 license]Advances in gene editing technology have spurred considerable progress towards a treatment for Duchenne muscular…

While previous gene therapies often made unpredictable changes in the cell’s genetic structure, these researchers showed an efficient method of disrupting a specific gene without making any other changes in DNA, removing unacceptable side-effects.

Mitochondria provide the energy for cells, but unlike DNA, they are only inherited from our mothers through their eggs. Defective mitochondria cause diseases which are incurable in adulthood. However, they are treatable before conception with…