New Method to Help Treat Cancer and Genetic Disorders through Gene Therapy

A UCLA team of researchers is developing a quicker, cheaper, and safer method that could deliver DNA into stem cells and immune cells.
Fabienne Lang

A move toward a better way to use gene therapies to attack cancer and genetic disorders has just been thought up by a UCLA-led team of researchers

The team has focused on developing a new method for bringing DNA into stem cells and immune cells in a safe, economical, and fast manner. The research could assist scientists by giving them a new tool to manufacture gene therapies for people with cancer, genetic disorders, and blood diseases. 

Their findings were published in the journal Proceedings of the National Academy of Sciences.


Genetic editing tools

"We are figuring out how to get gene-editing tools into cells efficiently, safely and economically," Paul Weiss, study co-author and UCLA professor of chemistry and biochemistry 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."

New Method to Help Treat Cancer and Genetic Disorders through Gene Therapy
Dr. Steven Jonas, Jason Belling, and Paul Weiss — the study co-authors, Source: Reed Hutchinson/UCLA

At the moment, cells used for gene therapies spend months in specialized labs, and at a high cost: an individualized treatment from the cells can cost up to hundreds of thousands of dollars for just one patient.

The UCLA team hopes to change that. "We hope our method could be used in the future to prepare treatments that can be performed at the patient's bedside," Weiss said.

Their method could be used with CRISPR, which would minimize costs, time, and safety in medical therapies. 

The technique the team has developed uses high-frequency acoustic waves along with millions of cells that flow through a device in a cell culture liquid. This procedure opens up pores alone the cells' membranes, which in turn enables DNA and other biological cargo to enter these cells without damaging the cells by contacting them directly. 

"When combined with new gene-editing approaches, the method enables us to correct a DNA sequence that is miscoded in a disease," said Weiss. "The viability is very high compared with other techniques, but we still want higher efficiencies and are working toward that."

New Method to Help Treat Cancer and Genetic Disorders through Gene Therapy
A prototype of the device, Source: Reed Hutchinson/UCLA

Dr. Steven Jonas, the study's co-senior author and UCLA clinical instructor in pediatrics explained "If the delivery works, and it seems to, this research is an important step toward bringing new therapies more broadly to the patients who need them."

"Traditionally, we have treated cancers with chemotherapy, surgery, radiation, and bone marrow transplantations. Now, we're at an amazing era of medicine, where we can use different types of gene therapies that can train the immune system to fight cancer."

The new approach is still under research and not yet available to treat humans. 

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