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New gene therapy method replaces mitochondrial DNA, study says

“Scientists have demonstrated a new type of gene therapy that would – in principle – allow mothers to avoid saddling their children with rare diseases that could result in heart problems, dementia, diabetes, deafness and other significant health issues.”

By extracting egg cells from volunteer donors, scientists were able to remove their cells’ chromosomes, and replace it with the mother’s chromosomes. The mitochondrial DNA however, remained intact within the cell. Thus, if these cells were to be injected back into the mother’s womb, her children will still resemble her characteristics (inherited from her chromosomes), yet her infected or mutated mitochondria will not be given to her children.

Read more about how these scientists did this: http://articles.latimes.com/2012/oct/24/news/la-heb-gene-therapy-mitochondrial-dna-20121024

New gene therapy for smoking kills the pleasure of nicotine

“A vaccine may one day help by preventing nicotine from reaching its target in the brain, according to research published [recently].”             Since most smoking therapies do a poor job of stopping the habit, scientists are trying out a new approach—a vaccine. The smoker would inject the vaccine like a shot, which in turn would create nicotine antibodies which would capture the nicotine from the bloodstream and trap it before reaching the brain and giving off its addictive attribute. Although vaccines have failed in the past, scientist are trying to solve this problem via gene therapy, in which a new gene is inserted into the body to do a particular job. The experiment was carried out in mice, where the insertion of gene that produces a nicotine antibody was taken into the mice’s livers, and the production of the antibody began. The vaccine was shown effective when nicotine was injected into the mice, and the antibodies were able to trap it before reaching the brain. Although the technology is not yet advanced for such procedures to be carried out on humans, it may not be too long before addiction therapies are replaced with this vaccine. 

Read more about how these scientist did it: http://articles.latimes.com/2012/jun/29/news/la-heb-new-gene-therapy-for-smoking-kills-the-pleasure-of-nicotine-20120629

Gene therapy makes major stride in 'Lorenzo's Oil' disease

The neurological fatal disorder known as adrenoleukodystrophy, or ADL has been stabilized in two 7 year old boys who have been treated for the disease by gene therapy. The disease is known to never stabilize, and “the fact that they [the scientists] were able to achieve that means they are getting a therapeutic effect” says Dr. Katherine A High of the Children’s Hospital of Philadelphia. This disorder causes the brain to start deteriorating in children around the age of 5, and may leave them dead within a year or two. If the disorder is identified before brain deterioration begins, the use of “Lorenzo’s Oil” may help hinder the diseases progression. However, if the deterioration has already begun, scientists can only treat the disease by a means of bone marrow transplant.  If the donor is a close relative, the disease may be ceased, however, if the donor is less closely related, the treatment may still be successful, but side effects may also occur, and sometimes they are severe.

Scientists have taken the normal ADL gene and inserted in into HIV that has been deemed un-harmful. HIV, which comes from the lentivirus family, may also be safer than the conventional mouse retroviruses that have been used in previous gene therapy treatments because  they are less likely to turn on unwanted genes once inserted. The bone marrow transplant was deemed successful in arresting the disease, and about 15% of the cells have begun producing the desired protein, which is great news, according to Dr. Donald Kohn.

Read more about the use of the lentivirus in the treatment of ADL and other related diseases: http://articles.latimes.com/2009/nov/06/science/sci-gene-therapy6

Successes of Gene Therapy

“An experimental gene therapy used in critically ill heart patients has shown significant potential for restoring blood flow in clogged cardiac vessels, reducing chest pain and enhancing quality of life. These successes, which have been demonstrated in a handful of small research studies so far, are drawing attention to a technique that appears to stimulate growth of new blood vessels. If the results are duplicated in larger studies, gene therapy may one day become an important tool in the fight against coronary artery disease.”

Here how to gene therapy helped two patients recover from their heart defects: http://articles.latimes.com/2001/nov/19/health/he-5906

Find more articles at:

http://articles.latimes.com/keyword/gene-therapy

http://www.sciencedaily.com/articles/g/gene_therapy.htm

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