Track topics on Twitter Track topics that are important to you
The new method is an alternative strategy of gene supplementation, which involves supplementing the defective gene, such as the ones that can lead to inherited retinal degeneration, with a healthy one. The healthy gene is delivered via the adeno-associated virus (AAV). However, the virus can only hold a small healthy gene, and the vast majority of patients with defects in a larger gene cannot be treated with this method.
“To overcome this problem, we developed a single AAV gene therapy platform that allows local replacement of a mutated sequence with its healthy counterpart that can treat almost any mutation,” said Associate Professor Koji Nishiguchi, co-first author on the paper.
The single AAV gene therapy platform combines CRISPR-Cas9 technology with micro-homology-mediated end joining. These two things act essentially as genetic scissors and genetic glue respectively. Researchers can target a specific defective gene, cut it out and glue in a healthy replacement.
In blind mice, this approach rescued approximately 10% of photoreceptors, resulting in improved light sensitivity and an increase in visual activity. The improvement in vision was about the same result gene supplementation can produce.
“By treating mice blinded by inherited retinal degeneration with the new treatment, we show that a robust visual restoration can be achieved at a level similar to that mediated by conventional gene supplementation, assuring its potential for clinical application,” Nishiguchi said.
“The platform paves the way for treating patients with mutations in larger genes, which comprise the vast majority of those with inherited retinal degeneration. Furthermore, a similar approach can be applied to treat almost any ocular and non-ocular inherited conditions.”
The researchers are now applying the new genome editing platform to develop a therapy for human patients with retinitis pigmentosa, a group of rare conditions that can cause loss of peripheral vision and difficulty seeing at night. They will target common mutations among patients that remain untreatable by conventional gene therapy, and hope to have therapy in a clinical trial by as early as 2025.
Original Article: Gene therapy improves vision in blind miceNEXT ARTICLE
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...
Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...
Biological therapy involves the use of living organisms, substances derived from living organisms, or laboratory-produced versions of such substances to treat disease. Some biological therapies for cancer use vaccines or bacteria to stimulate the body&rs...