Importance of silent genes

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Offline Manal

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Importance of silent genes
« on: December 02, 2006, 01:37:58 AM »
Dear all

One type of the researches done in thal is to switch on the gamma gloin genes responsible for producing the HB F.  These studies involve hydroxyurea, butyrate compounds, others to be discovered..............etc.  My two concerns are:

1) Maybe the reason that God switched these genes is to enable other activities to start, it could be development, certain cell divisions, other gene activity,......etc and switching on these ''normally'' silent  genes may interfers with other body activites which may depend on the switch off of those genes.

2) Is it calculated where are these medications targeting?? I mean these medecines may have the desired effect on the specific gene or genes involved, it can also have an undesirable effect by turning on the wrong genes

What do you think, do i make sense or am i over-reacting. Andy can you shed any light on this.

Hope i am understood

Take care



Offline Christine Mary

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Re: Importance of silent genes
« Reply #1 on: December 02, 2006, 01:47:11 AM »

From what I've read recently, this is exactly what scientists are working on regarding gene therapy. They are trying to figure out how to avoid turning on certain cancer causing genes called oncogenes.
Here is part of the article......

Gene Transfer

While stem cell transplantation has a high success rate among young, HLA-matched patients, researchers hope to find a more universally available cure. Gene transfer may be the solution. "The goal is to introduce a gene to restore normal function in red blood cells, removing the transfusion requirement," said Michel Sadelain, a researcher at Memorial Sloan-Kettering Cancer Center.

"The goal is to introduce a gene to restore normal function in red blood cells."
The process involves removing some of the patient's stem cells, inserting a gene that codes for the healthy globins, and then re-injecting the cells into the body. To introduce the new gene, Sadelain and his team have created a vector based on a lentivirus that is capable of introducing β-hemoglobin genes into the cells. They demonstrated the concept could work in thalassemic mice in 2003. "These mice remained alive only due to transplanted gene," said Sadelain. In the laboratory, they've shown that the gene vector works at least as well in human cells as it does in mouse cells, yielding a level of expression of about 55%.

A clinical trial of gene therapy to cure thalassemia is planned to begin in the next year in France, reported Arthur Bank, a physician and geneticist at Columbia University in New York and Philippe Leboulch at Harvard Medical School and Brigham & Women's Hospital in Boston. The first phase will enroll five patients with β thalassemia and look primarily at safety.

Gene therapy: not without risks

Gene transfer is not without risks, however. Inserting new genes into a person's DNA can inadvertently activate certain cancer-causing genes called oncogenes. To reduce the risk of this happening, researchers have devised a way to confine the gene's expression to maturing red blood cells. In other words, all of the blood-forming cells will carry the gene but only a subset, the red blood cells, will carry the switch to turn it on. The lentivirus vector also contains bulky insulating molecules made of a protein called chromatin that physically block molecules called transcription factors from binding to the wrong site on the patient's DNA and turning on the wrong genes.

A three-dimensional structure brings DNA's regulatory region close to globin genes.
Inserting new pieces of DNA into precisely the right location is made even more difficult by the fact that the globin genes can fold into a three-dimensional structure of DNA and its surrounding chromatin. The three-dimensional structure brings the regulatory region of the DNA close to globin genes. The shape of this three-dimensional complex controls which genes are expressed, and it presents a challenge for researchers who are developing gene vectors. "If you don't control the insertion of new genes precisely, the regulatory elements on the imported section of DNA may start controlling the existing genes in the locus," said Frank Grosveld of the Erasmus Medical Center in Rotterdam, The Netherlands.

Another option for introducing healthy copies of the β-globin gene into thalassemia patients is via somatic cell nuclear transfer, sometimes called therapeutic cloning, said Tim Townes of the University of Alabama. Cells produced this way would be identical to the patient and would presumably not elicit an immune response when implanted into the patient. Townes described a scheme for using this approach.

Lauryn's Mom


Offline Andy Battaglia

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Re: Importance of silent genes
« Reply #2 on: December 02, 2006, 10:14:53 PM »
HI Manal,

Your questions are very interesting.

If you get into a strict interpretation of what God wants, you might not even be able to justify treating any illness. I don't believe we can take that approach. God has given us great brains and great abilities and my belief is that God has given us knowledge to change our world for the better and if we are to fully realize God's plans, we must try to make the best of our abilities and do everything we can to better our world.

The human body uses fetal hemoglobin in the womb. HbF is best suited for the needs in the womb, but at birth, there is a transition to adult Hb which is more suited to the needs after birth. However, fetal hemoglobin does work and its production is affected by even natural foods like grapes and wheatgrass so I cannot see any reason to say God meant for that gene to shut off for good because it isn't shut off for good. It may be in fact, that we need to discover the natural diet that will do the most to turn on this fetal HB. Perhaps that is part of God's plan. There is no danger to activating the gene. It is something done naturally.

I do also share your questions about what else things like hydroxyurea may do in the body but that also holds for any medicine. I do take comfort in seeing things like this from studies...


No side effects of hydroxyurea have been observed.

The doctors and researchers are doing their best. We need more money for research but please take advantage of what they have learned to help thals.


All we are saying is give thals a chance.


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