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Three people with inherited diseases successfully treated with CRISPR

crispr aging

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#1 gamesguru

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Posted 14 June 2020 - 02:10 PM


Encouraging fronts on CRISPR research in treating genetic diseases :)

 

Three people with inherited diseases successfully treated with CRISPR
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Sickle cell disease can distort red blood cells

Stocktrek Images, Inc/Alamy

Two people with beta thalassaemia and one with sickle cell disease no longer require blood transfusions, which are normally used to treat severe forms of these inherited diseases, after their bone marrow stem cells were gene-edited with CRISPR.

 

Result of the ongoing trial, which is the first to use CRISPR to treat inherited genetic disorders, were announced today at a virtual meeting of the European Hematology Association.

 

“The preliminary results… demonstrate, in essence, a functional cure for patients with beta thalassaemia and sickle cell disease,” team member Haydar Frangoul at Sarah Cannon Research Institute in Nashville, Tennessee, said in a statement.

 

Beta thalassaemia and sickle cell are diseases caused by mutations that affect haemoglobin, the protein that carries oxygen in red blood cells. Those with severe forms require regular blood transfusions.

 

However, a few people with the disease-causing mutations never show any symptoms, because they keep producing fetal haemoglobin in adulthood. Normally, fetal haemoglobin stops being produced soon after birth.

 

This discovery has inspired the development of treatments based on boosting fetal haemoglobin. In this trial, run by collaborating companies CRISPR Therapeutics and Vertex, bone marrow stem cells are removed from people and the gene that turns off fetal haemoglobin production is disabled with CRISPR.

 

The remaining bone marrow cells are killed by chemotherapy, then replaced by the edited cells. This is done to ensure new blood cells are produced by the edited stem cells, but the chemotherapy can have serious side effects including infertility.

 

The first two patients with beta thalassaemia no longer need blood transfusions since being treated 15 and 5 months ago. Nor does the patient with sickle cell disease, 9 months after treatment.

 

The results are excellent, says Marina Cavazzana at the Necker-Enfants Malades Hospital in Paris, France, whose team has treated a 13-year-old boy with sickle cell disease using a different approach.

 

While the three patients did suffer some adverse effects due to the chemotherapy, the CRISPR gene editing appears safe. However, the patients may need to be monitored for the rest of their lives to be sure it has no adverse effects, says Cavazzana.

 

Altogether five people have now been treated. The trial was put on hold because of the coronavirus pandemic, but has now resumed.

 



#2 MichaelFocus22

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Posted 14 June 2020 - 05:58 PM

Perfect, that we are finally seeing the greatness of crispr at work. We will cure all diseases and inherited genetic conditions.



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#3 Believer

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Posted 15 June 2020 - 06:24 PM

Perfect, that we are finally seeing the greatness of crispr at work. We will cure all diseases and inherited genetic conditions.

Unfortunately the political elite will label it was "unethical" like stem cell therapy and so many other safe therapies, and they will regulate gene therapy to death making it almost impossible to use it effectively to treat diseases.

 



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#4 gamesguru

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Posted 16 June 2020 - 12:52 AM

i mean inviting effective treatments to disease and aging generally could contribute to overpopulation.  We are already on track to have 10 billion soon, and some people feel that is far too many.  There would have to be some difficult to regulate restrictions, like you can't treat aging diseases with XYZ advanced technologies UNLESS you sequester your right to reproduce and have kids who could also stick around much longer than typical and potentially have kids of their own and so on..  There is a population crisis when you just naively introduce effective aging treatments, not just the rich will want it


  • Dangerous, Irresponsible x 1





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