A step toward a more efficient way to make gene therapies to attack cancer, genetic disorders

A UCLA-led research team today reports that it has developed a new method for delivering DNA into stem cells and immune cells safely, rapidly and economically. The method, described in the journal Proceedings of the National Academy of Sciences, could give scientists a new tool for manufacturing gene therapies for people with cancer, genetic disorders and blood diseases.The study ’s co-senior author is Paul Weiss, a UCLA distinguished professor of chemistry and biochemistry, of bioengineering and of materials science and engineering. “We are figuring out how to get gene-editing tools into cells efficiently, safely and economically,” he said. “We want to get them into enormous numbers of cells without using viruses, electroshock treatments or chemicals that will rip open the membrane and kill many of the cells, and our results so far are promising.”In current practice, cells used for genetic therapies are sent to specialized labs, which can take up to two months to produce an individualized treatment. And those treatments are expensive: A single regimen for one patient can cost hundreds of thousands of dollars.“We hope our method could be used in the future to prepare treatments that can be performed at the patient’s bedside,” Weiss said.The method could be used withCRISPR, the genetic engineering technique that enables DNA to be edited with remarkable precision. However, using CRISPR efficiently, safely and economically in medical therapies has proven to be a ...
Source: UCLA Newsroom: Health Sciences - Category: Universities & Medical Training Source Type: news