Researchers test new gene therapy method in human cells
Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.
The research results are online Wednesday, Oct. 24, in the highly respected journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24'.
The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.
The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.
"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."
To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.
"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study only allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."
The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.
The second portion of the study, which was completed at ONPRC, is also considered an important achievement because egg cells only remain viable for a short period of time after they are harvested from a donor. Therefore, for this therapy to be a viable option in the clinic, preservation through freezing likely is necessary so that both the donor cell and a mother's cell are viable at the time of the procedure.
While this form of therapy has yet to be approved in the United States, the United Kingdom is seriously considering its use for treating human patients at risk for mitochondria-based disease. It's believed that this most recent breakthrough, combined with earlier animal studies, will help inform that decision-making process.
Because the research involved the use of human egg cells and there are restrictions to the use of federal funding for some work in human egg cells, private funding was obtained to accomplish the work.
In addition, researchers consulted with ethicists and other experts within OHSU's Institutional Review Board and the OHSU Stem Cell Research Oversight Committee (OSCRO) prior to embarking on this research. The OSCRO reviews research involving human embryonic stem cells at OHSU to ensure that all federal and state regulations governing the conduct of stem cell research are met and that all human embryonic stem cell research is conducted in accordance with the general principles expressed in the National Academies' Guidelines for Human Embryonic Stem Cell Research. The OHSU IRB reviews biomedical and behavioral research that involves humans in order to protect the rights and welfare of the research subjects.
Journal reference: Nature
Provided by Oregon Health & Science University
- Study shows how mitochondrial genes are passed from mother to child May 03, 2012 | not rated yet | 0
- World's first chimeric monkeys are born Jan 05, 2012 | not rated yet | 0
- New technique could eliminate inherited mitochondrial disease Aug 26, 2009 | not rated yet | 0
- Study: How stem cells become brain cells Dec 15, 2005 | not rated yet | 0
- Egg-like cells obtained in pig fetal skin Mar 28, 2006 | not rated yet | 0
- Motion perception revisited: High Phi effect challenges established motion perception assumptions Apr 23, 2013 | 3 / 5 (2) | 2
- Anything you can do I can do better: Neuromolecular foundations of the superiority illusion (Update) Apr 02, 2013 | 4.5 / 5 (11) | 5
- The visual system as economist: Neural resource allocation in visual adaptation Mar 30, 2013 | 5 / 5 (2) | 9
- Separate lives: Neuronal and organismal lifespans decoupled Mar 27, 2013 | 4.9 / 5 (8) | 0
- Sizing things up: The evolutionary neurobiology of scale invariance Feb 28, 2013 | 4.8 / 5 (10) | 14
How can there be a term called "intestinal metaplasia" of stomach
15 hours ago Hello everyone, Ok Stomach's normal epithelium is simple columnar, now in intestinal type of adenocarcinoma of stomach it undergoes "intestinal...
Pressure-volume curve: Elastic Recoil Pressure don't make sense
May 18, 2013 From pressure-volume curve of the lung and chest wall (attached photo), I don't understand why would the elastic recoil pressure of the lung is...
If you became brain-dead, would you want them to pull the plug?
May 17, 2013 I'd want the rest of me to stay alive. Sure it's a lousy way to live but it beats being all-the-way dead. Maybe if I make it 20 years they'll...
MRI bill question
May 15, 2013 Dear PFers, The hospital gave us a $12k bill for one MRI (head with contrast). The people I talked to at the hospital tell me that they do not...
Ratio of Hydrogen of Oxygen in Dessicated Animal Protein
May 13, 2013 As an experiment, for the past few months I've been consuming at least one portion of Jell-O or unflavored Knox gelatin per day. I'm 64, in very...
Alcohol and acetaminophen
May 13, 2013 Edit: sorry for the typo in the title , can't edit I looked around on google quite a bit and it's very hard to find precise information on the...
- More from Physics Forums - Medical Sciences
More news stories
Trends in Helicobacter pylori (H. pylori) and smoking explain a significant proportion of the decline of intestinal-type noncardia gastric adenocarcinoma (NCGA) incidence in US men between 1978 and 2008, and are estimated ...
Medical research 5 hours ago | not rated yet | 0
Widely available in pharmacies and health stores, phosphatidylserine is a natural food supplement produced from beef, oysters, and soy. Proven to improve cognition and slow memory loss, it's a popular treatment for older ...
Medical research 10 hours ago | 5 / 5 (1) | 0 |
Researchers at Emory University have identified a protein that stimulates a pair of "orphan receptors" found in the brain, solving a long-standing biological puzzle and possibly leading to future treatments for neurological ...
Medical research 10 hours ago | not rated yet | 0 |
Australian scientists have charted the path of insulin action in cells in precise detail like never before. This provides a comprehensive blueprint for understanding what goes wrong in diabetes.
Medical research 11 hours ago | 4.8 / 5 (4) | 0 |
Researchers at the University of Illinois at Chicago College of Medicine will study gender differences in how the heart uses and stores fat—its main energy source—and how changes in fat metabolism play ...
Medical research 14 hours ago | not rated yet | 0
(Medical Xpress)—Native peoples in regions where cameras are uncommon sometimes react with caution when their picture is taken. The fear that something must have been stolen from them to create the photo ...
11 hours ago | 4.2 / 5 (5) | 0 |
(Medical Xpress)—Despite spending billions of dollars on research and development, drug companies have been unable to come up with effective treatments for dementia and Alzheimer's Disease (AD). Now, A. ...
9 hours ago | 4.9 / 5 (8) | 0 |
An experimental sleeping pill from US drug company Merck is effective at helping people fall and stay asleep, according to reviewers at the US Food and Drug Administration, which could soon approve the new drug.
4 hours ago | 3 / 5 (2) | 0
Activating an enzyme known to play a role in the anti-aging benefits of calorie restriction delays the loss of brain cells and preserves cognitive function in mice, according to a study published in the May ...
5 hours ago | 5 / 5 (3) | 0 |
A drug commonly used to treat depression and anxiety may improve a stress-related heart condition in people with stable coronary heart disease, according to researchers at Duke Medicine.
6 hours ago | not rated yet | 0 |
Researchers at USC have found that a class of pharmaceuticals can both prevent and treat Alzheimer's Disease in mice.
8 hours ago | 5 / 5 (3) | 0 |