New gene therapy platform-a way to treat blindness

A paper published today in the journal "eLife" describes this technology, which simplifies the development of gene therapy to treat genetic blindness diseases

"Decreased vision has a huge impact on the quality of life

Although blinding genetic diseases that affect the retina are considered rare, approximately 1 in 3,000 people worldwide carry one or more copies of damaged genes that cause retinal degeneration and blindness

Nowadays, several gene therapies are already on the market in Europe and the United States, and dozens of them have entered clinical trials.

To solve this problem, the researchers developed a computing platform called scAAVengr, which uses single-cell RNA sequencing to quickly and quantitatively evaluate-among dozens of options-which adeno-associated viral vector (AAV) is the best It is suitable for delivering gene therapy to specific parts of the retina

The traditional method of assessing AAV is very slow, requiring several years and many experimental animals

In contrast, scAAVengr uses single-cell RNA sequencing to detect whether the cargo has reached its destination safely

The use of the platform is not limited to the retina—researchers have shown that it is equally effective in identifying AAV targeting other tissues, including the brain, heart, and liver

Leah C Byrne said: "The rising tide makes all boats rise.

Journal Reference :

Bilge E Öztürk, Molly E Johnson, Michael Kleyman, Serhan Turunç, Jing He, Sara Jabalameli, Zhouhuan Xi, Meike Visel, Valérie L Dufour, Simone Iwabe, Felipe Pompeo Marinho, Gustavo D Aguirre; José -Alain Sahel, David V Schaffer, Andreas R Pfenning, John G Flannery, William A Beltran, William R Stauffer, Leah C Byrne.