Dave Farber
2018-07-12 10:00:14 UTC
Subject: [Dewayne-Net] Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
Date: July 12, 2018 at 6:52:26 PM GMT+9
Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
Scientists report that they have discovered a way to tweak genes in the bodyâs immune cells by using electrical fields.
By Gina Kolata
Jul 11 2018
<https://www.nytimes.com/2018/07/11/health/gene-editing-cancer.html>
For the first time, scientists have found a way to efficiently and precisely remove genes from white blood cells of the immune system and to insert beneficial replacements, all in far less time than it normally takes to edit genes.
If the technique can be replicated in other labs, experts said, it may open up profound new possibilities for treating an array of diseases, including cancer, infections like H.I.V. and autoimmune conditions like lupus and rheumatoid arthritis.
The new work, published on Wednesday in the journal Nature, âis a major advance,â said Dr. John Wherry, director of the Institute of Immunology at the University of Pennsylvania, who was not involved in the study.
But because the technique is so new, no patients have yet been treated with white blood cells engineered with it.
âThe proof will be when this technology is used to develop a new therapeutic product,â cautioned Dr. Marcela Maus, director of cellular immunotherapy at Massachusetts General Hospital.
That test may not be far away. The researchers have already used the method in the laboratory to alter the abnormal immune cells of children with a rare genetic condition. They plan to return the altered cells to the children in an effort to cure them.
Currently, scientists attempting to edit the genome often must rely on modified viruses to slice open DNA in a cell and to deliver new genes into the cell. The method is time-consuming and difficult, limiting its use.
Despite the drawbacks, the virus method has had some success. Patients with a few rare blood cancers can be treated with engineered white blood cells â the immune systemâs T-cells â that go directly to the tumors and kill them.
This type of treatment with engineered white cells, called immunotherapy, has been limited because of the difficulty of making viruses to carry the genetic material and the time needed to create them.
But researchers now say they have a found a way to use electrical fields, not viruses, to deliver both gene-editing tools and new genetic material into the cell. By speeding the process, in theory a treatment could be available to patients with almost any type of cancer.
âWhat takes months or even a year may now take a couple weeks using this new technology,â said Fred Ramsdell, vice president of research at the Parker Institute for Cancer Immunotherapy in San Francisco. âIf you are a cancer patient, weeks versus months could make a huge difference.â
âI think itâs going to be a huge breakthrough,â he added.
The Parker Institute already is working with the authors of the new paper, led by Dr. Alexander Marson, scientific director of biomedicine at the Innovative Genomics Institute â a partnership between University of California, San Francisco and the University of California, Berkeley â to make engineered cells to treat a variety of cancers.
In the new study, Dr. Marson and his colleagues engineered T-cells to recognize human melanoma cells. In mice carrying the human cancer cells, the modified T-cells went right to the cancer, attacking it.
The researchers also corrected â in the lab â the T-cells of three children with a rare mutation that caused autoimmune diseases. The plan now is to return these corrected cells to the children, where they should function normally and suppress the defective immune cells, curing the children.
The technique may also hold great promise for treating H.I.V., Dr. Wherry said.
The H.I.V. virus infects T-cells. If they can be engineered so that the virus cannot enter the T-cells, a person infected with H.I.V. should not progress to AIDS. Those T-cells already infected would die, and the engineered cells would replace them.
Previous research has shown it might be possible to treat H.I.V. in this way. âBut now there is a really efficient strategy to do this,â Dr. Wherry said.
[snip]
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-------------------------------------------Date: July 12, 2018 at 6:52:26 PM GMT+9
Swift Gene-Editing Method May Revolutionize Treatments for Cancer and Infectious Diseases
Scientists report that they have discovered a way to tweak genes in the bodyâs immune cells by using electrical fields.
By Gina Kolata
Jul 11 2018
<https://www.nytimes.com/2018/07/11/health/gene-editing-cancer.html>
For the first time, scientists have found a way to efficiently and precisely remove genes from white blood cells of the immune system and to insert beneficial replacements, all in far less time than it normally takes to edit genes.
If the technique can be replicated in other labs, experts said, it may open up profound new possibilities for treating an array of diseases, including cancer, infections like H.I.V. and autoimmune conditions like lupus and rheumatoid arthritis.
The new work, published on Wednesday in the journal Nature, âis a major advance,â said Dr. John Wherry, director of the Institute of Immunology at the University of Pennsylvania, who was not involved in the study.
But because the technique is so new, no patients have yet been treated with white blood cells engineered with it.
âThe proof will be when this technology is used to develop a new therapeutic product,â cautioned Dr. Marcela Maus, director of cellular immunotherapy at Massachusetts General Hospital.
That test may not be far away. The researchers have already used the method in the laboratory to alter the abnormal immune cells of children with a rare genetic condition. They plan to return the altered cells to the children in an effort to cure them.
Currently, scientists attempting to edit the genome often must rely on modified viruses to slice open DNA in a cell and to deliver new genes into the cell. The method is time-consuming and difficult, limiting its use.
Despite the drawbacks, the virus method has had some success. Patients with a few rare blood cancers can be treated with engineered white blood cells â the immune systemâs T-cells â that go directly to the tumors and kill them.
This type of treatment with engineered white cells, called immunotherapy, has been limited because of the difficulty of making viruses to carry the genetic material and the time needed to create them.
But researchers now say they have a found a way to use electrical fields, not viruses, to deliver both gene-editing tools and new genetic material into the cell. By speeding the process, in theory a treatment could be available to patients with almost any type of cancer.
âWhat takes months or even a year may now take a couple weeks using this new technology,â said Fred Ramsdell, vice president of research at the Parker Institute for Cancer Immunotherapy in San Francisco. âIf you are a cancer patient, weeks versus months could make a huge difference.â
âI think itâs going to be a huge breakthrough,â he added.
The Parker Institute already is working with the authors of the new paper, led by Dr. Alexander Marson, scientific director of biomedicine at the Innovative Genomics Institute â a partnership between University of California, San Francisco and the University of California, Berkeley â to make engineered cells to treat a variety of cancers.
In the new study, Dr. Marson and his colleagues engineered T-cells to recognize human melanoma cells. In mice carrying the human cancer cells, the modified T-cells went right to the cancer, attacking it.
The researchers also corrected â in the lab â the T-cells of three children with a rare mutation that caused autoimmune diseases. The plan now is to return these corrected cells to the children, where they should function normally and suppress the defective immune cells, curing the children.
The technique may also hold great promise for treating H.I.V., Dr. Wherry said.
The H.I.V. virus infects T-cells. If they can be engineered so that the virus cannot enter the T-cells, a person infected with H.I.V. should not progress to AIDS. Those T-cells already infected would die, and the engineered cells would replace them.
Previous research has shown it might be possible to treat H.I.V. in this way. âBut now there is a really efficient strategy to do this,â Dr. Wherry said.
[snip]
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