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Thalassemia Patients and Friends and thalpal Ā© A. Battaglia 2018





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Author Topic: Thalassemia major cure by Chinese scientists.  (Read 2580 times)
Pratik
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« on: October 01, 2017, 05:05:06 AM »

Good day friends. Today I have some excellent news to share. Some Chinese scientist have finally develop and found the cure for our Thalassemia major disease. It's Sachin auspicious day in first day of quarter 4 to receive this news. Our cure and treatment is now not far away. Maybe at most by the end of next year we will be leaving of your life without any disease without being called a Thalassemia major child and person and people will stop making fun of us and calling is disabled or weak.

Check out this article below that I found on Times of India.

http://m.timesofindia.com/home/science/chinese-repair-genetic-defect-causing-thalassemia/articleshow/60897046.cms

When I read this news this morning I jumped from my sofa ROM happiness. I mean immense immense happiness. I almost cried for all those thousands of people and gifts that suffer from this disease and are now going to be cured. :dunn
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sofear
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« Reply #1 on: October 01, 2017, 01:50:45 PM »

They did this in in embryos and I read a comment somewhere that it would still be a long way to apply this to other groups.

I can't judge it without any medical background, but isn't what companies like bluebird bio do much more advanced, as in: they work towards curing thalassemia in childs and adults and not just embryos?

I mean, if people just did a PID and checked for thalassemia, they could guarantee that the child is born without thalassemia, so the most interesting research is that done on people that are already born with it.
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zahra
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« Reply #2 on: October 02, 2017, 02:18:25 AM »

Hi Pratik,

Someone else has posted about this in 'working towards a cure' based on a bbc article. That article clearly states the embryo's created were not implanted. Also that there are many ethical issues to resolve before it could be so. I'm not expecting anything of real value anytime soon. At this time no one knows the effect of gene editing at this early stage of development over a whole life
They must make sure this editing did not change , activate or put to sleep any other genes too because if it did that would open a whole new pandora's box.

Zahra

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Andy Battaglia
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« Reply #3 on: October 06, 2017, 04:35:11 PM »

There are many potential problems with CRSPR and I am not enthusiastic about it, as it requires making genetic changes that can be passed on to children. They do not understand the interactions between genes well enough to be able to declare it as safe practice. As Zahra said, it opens Pandora's box and I think we need to proceed with great caution concerning CRSPR.

However, another approach to gene therapy using one's own stem cells was just announced. I believe this will be a hot topic at the upcoming TIF conference. I wish I could go, but the schedule and lack of funds make it impossible for me.
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Andy

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Sharmin
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« Reply #4 on: October 18, 2017, 08:52:20 PM »

Hello Andy,

I am eager to learn about the new approach using the patient's own stem sells. Is it the Sangamo gene therapy?

Thank you,

Sharmin
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Sharmin
Andy Battaglia
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« Reply #5 on: October 25, 2017, 05:17:01 PM »

I think it is Sangamo, but I am not certain.
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Andy

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Pratik
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« Reply #6 on: December 01, 2017, 08:27:01 AM »

Hopefully this treatment will be available next year.
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Andy Battaglia
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« Reply #7 on: December 30, 2017, 04:03:56 PM »

Sofear,

Gene therapy involves good globin genes being inserted into a patient where they can reproduce and allow them to make their own red blood cells, but this is not inheritable, and that is where the focus of concern about CRSPR has always been. The long term consequences of changing the inheritable genome are unknown. We see with thalassemia that it is most often affected by more genes than the globin genes. This interaction between genes is not well understood and until it is, gene editing will not progress in humans as quickly as people hope for.
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Andy

All we are saying is give thals a chance.
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