The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

There are 20 cell and gene therapies on Europe's market versus 43 in the U.S. Find out more about this ecosystem.

A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...

“For example, if parents have a hereditary muscle ... Dr Noora clarified: “So far, there are no clinical trials on humans for gene therapy. All current research is focused on animal trials ...

The UK has approximately 17,500 individuals living with sickle cell disease, a condition particularly prevalent amongst those ...

Both bone marrow transplants and gene therapy require extensive conditioning ... high-quality care to every patient who needs it. For example, in a 2018 report to Congress, the Office of the ...

Scientists at UC San Francisco have pioneered a groundbreaking approach to fighting cancer using engineered fat cells. By ...

The ACP guideline, published in Annals of Internal Medicine, includes three recommendations centered around monotherapy: ...

Practical Applications of SCP-Nano Examples of nanocarriers analyzed ... programmable and AAVs are highly efficient carriers for gene therapy. The LNPs facilitate RNA delivery, which underlies ...

Liver diseases can be inherited (such as Wilson disease and alpha 1-antitrypsin deficiency) or acquired (for example hepatitis and liver cancer). The performance of AAV gene-therapy vectors is ...