Treating congenital defects with gene editing (Introduction)

by David Turell @, Saturday, June 13, 2020, 21:27 (106 days ago)

Of general interest, we now can alter genetics:

https://www.newscientist.com/article/2246020-three-people-with-inherited-diseases-succe...

"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."

Comment: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

Treating congenital defects with gene editing

by dhw, Sunday, June 14, 2020, 11:04 (105 days ago) @ David Turell

DAVID: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

Disregarding the problems that our fellow organisms and our not-so-clever ancestors have had to face over the last 3.8 billion years, why do you think he wanted to set us problems in the first place?

Treating congenital defects with gene editing

by David Turell @, Sunday, June 14, 2020, 16:10 (105 days ago) @ dhw

DAVID: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

dhw: Disregarding the problems that our fellow organisms and our not-so-clever ancestors have had to face over the last 3.8 billion years, why do you think he wanted to set us problems in the first place?

Opening up to lots of guesswork. Viruses were with us from close to the beginning of life, and there is important evidence they actually helped in the process of evolution. That doesn't explain the bad ones, but that means their evolution was not tightly controlled by God; we don't know the reason. Didn't worry about it, didn't care, or let it go on purposely. Take your pick. As for biological mistakes, the complexity of life has many safeguards built in, but it is obvious that mistakes can happen and do. We seem to be able to solve some from our brain power and that applies to creating vaccines also. We do have the brains.

Treating congenital defects with gene editing

by dhw, Monday, June 15, 2020, 11:02 (104 days ago) @ David Turell

DAVID: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

dhw: Disregarding the problems that our fellow organisms and our not-so-clever ancestors have had to face over the last 3.8 billion years, why do you think he wanted to set us problems in the first place?

DAVID: Opening up to lots of guesswork. Viruses were with us from close to the beginning of life, and there is important evidence they actually helped in the process of evolution. That doesn't explain the bad ones, but that means their evolution was not tightly controlled by God; we don't know the reason. Didn't worry about it, didn't care, or let it go on purposely. Take your pick.

Nobody “knows” your God’s reasons for anything! But it appears from this that God might NOT always be in tight control of evolution. And if you accept that as a POSSIBILITY, you can hardly reject it as a possible explanation for the vast variety of life forms resulting from his not WISHING to tightly control evolution, i.e. that he let evolution ”go on purposely”.

DAVID: As for biological mistakes, the complexity of life has many safeguards built in, but it is obvious that mistakes can happen and do. We seem to be able to solve some from our brain power and that applies to creating vaccines also. We do have the brains.

My question was why you think he wanted to set us problems in the first place. If you argue that all the problems he set us may have been the result of his letting evolution “go on purposely”, you are supporting the theory that he set it in motion and did not control it. Or if he made mistakes, you are committing what you seem to regard as almost blasphemous, i.e. that your God is humanly fallible. As an “answer for theodicy”, it works quite nicely, though – evil is the result of God not being in control, or being humanly fallible.

Treating congenital defects with gene editing

by David Turell @, Monday, June 15, 2020, 17:49 (104 days ago) @ dhw

DAVID: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

dhw: Disregarding the problems that our fellow organisms and our not-so-clever ancestors have had to face over the last 3.8 billion years, why do you think he wanted to set us problems in the first place?

DAVID: Opening up to lots of guesswork. Viruses were with us from close to the beginning of life, and there is important evidence they actually helped in the process of evolution. That doesn't explain the bad ones, but that means their evolution was not tightly controlled by God; we don't know the reason. Didn't worry about it, didn't care, or let it go on purposely. Take your pick.

dhw: Nobody “knows” your God’s reasons for anything! But it appears from this that God might NOT always be in tight control of evolution. And if you accept that as a POSSIBILITY, you can hardly reject it as a possible explanation for the vast variety of life forms resulting from his not WISHING to tightly control evolution, i.e. that he let evolution ”go on purposely”.

Or His tight control allowed the mistakes to happen because He anticipated our giant brain would solve the problems that appeared!


DAVID: As for biological mistakes, the complexity of life has many safeguards built in, but it is obvious that mistakes can happen and do. We seem to be able to solve some from our brain power and that applies to creating vaccines also. We do have the brains.

dhw: My question was why you think he wanted to set us problems in the first place. If you argue that all the problems he set us may have been the result of his letting evolution “go on purposely”, you are supporting the theory that he set it in motion and did not control it. Or if he made mistakes, you are committing what you seem to regard as almost blasphemous, i.e. that your God is humanly fallible. As an “answer for theodicy”, it works quite nicely, though – evil is the result of God not being in control, or being humanly fallible.

Same reply: Or His tight control allowed the mistakes to happen because He anticipated our giant brain would solve the problems that appeared! All we know is that there are biological errors in any living system operating at such high speed, and controls designed into it cannot stop everyone of them. That leaves us with: God did the best He could, and any better is impossible considering the necessary complexity of living organisms. I'll accept that.

Treating congenital defects with gene editing

by dhw, Tuesday, June 16, 2020, 11:12 (103 days ago) @ David Turell

DAVID: Thank goodness, our advanced human brain can find solutions for biological problems. It is the answer I have for theodicy. God gave us a brain that can solve problems that arise.

dhw: Disregarding the problems that our fellow organisms and our not-so-clever ancestors have had to face over the last 3.8 billion years, why do you think he wanted to set us problems in the first place?

DAVID: Opening up to lots of guesswork. Viruses were with us from close to the beginning of life, and there is important evidence they actually helped in the process of evolution. That doesn't explain the bad ones, but that means their evolution was not tightly controlled by God; we don't know the reason. Didn't worry about it, didn't care, or let it go on purposely. Take your pick.

dhw: Nobody “knows” your God’s reasons for anything! But it appears from this that God might NOT always be in tight control of evolution. And if you accept that as a POSSIBILITY, you can hardly reject it as a possible explanation for the vast variety of life forms resulting from his not WISHING to tightly control evolution, i.e. that he let evolution ”go on purposely”.

DAVID: Or His tight control allowed the mistakes to happen because He anticipated our giant brain would solve the problems that appeared!

As usual, you seem to think life began with humans. And I really don’t see how “allowing mistakes to happen” ties in with tight control – but I’m not going to quarrel with your proposal that that he might not have tightly controlled evolution and might have “let it go on purposely”. That is one of the various explanations I have offered for the higgledy-piggledy history of evolution.

.dhw: […] Or if he made mistakes, you are committing what you seem to regard as almost blasphemous, i.e. that your God is humanly fallible. As an “answer for theodicy”, it works quite nicely, though – evil is the result of God not being in control, or being humanly fallible.

DAVID: […] All we know is that there are biological errors in any living system operating at such high speed, and controls designed into it cannot stop everyone of them. That leaves us with: God did the best He could, and any better is impossible considering the necessary complexity of living organisms. I'll accept that.

Well, if you can accept that God did the best he could but he couldn’t avoid making mistakes (how extraordinarily human of him), I don’t see why you can’t accept the possibility that life’s bush was the product of his experiments, or H. sapiens came late on in his thinking. Why is that more “human” than making mistakes?

Treating congenital defects with gene editing

by David Turell @, Tuesday, June 16, 2020, 19:15 (103 days ago) @ dhw

DAVID: Opening up to lots of guesswork. Viruses were with us from close to the beginning of life, and there is important evidence they actually helped in the process of evolution. That doesn't explain the bad ones, but that means their evolution was not tightly controlled by God; we don't know the reason. Didn't worry about it, didn't care, or let it go on purposely. Take your pick.

dhw: Nobody “knows” your God’s reasons for anything! But it appears from this that God might NOT always be in tight control of evolution. And if you accept that as a POSSIBILITY, you can hardly reject it as a possible explanation for the vast variety of life forms resulting from his not WISHING to tightly control evolution, i.e. that he let evolution ”go on purposely”.

DAVID: Or His tight control allowed the mistakes to happen because He anticipated our giant brain would solve the problems that appeared!

dhw: As usual, you seem to think life began with humans. And I really don’t see how “allowing mistakes to happen” ties in with tight control – but I’m not going to quarrel with your proposal that that he might not have tightly controlled evolution and might have “let it go on purposely”. That is one of the various explanations I have offered for the higgledy-piggledy history of evolution.

It is easiest to discuss our problems which we know as current events. You are right the history is higgledy-piggledy, but I see purpose in creating the necessary econiches and your god is usually not that purposeful.


.dhw: […] Or if he made mistakes, you are committing what you seem to regard as almost blasphemous, i.e. that your God is humanly fallible. As an “answer for theodicy”, it works quite nicely, though – evil is the result of God not being in control, or being humanly fallible.

DAVID: […] All we know is that there are biological errors in any living system operating at such high speed, and controls designed into it cannot stop everyone of them. That leaves us with: God did the best He could, and any better is impossible considering the necessary complexity of living organisms. I'll accept that.

dhw: Well, if you can accept that God did the best he could but he couldn’t avoid making mistakes (how extraordinarily human of him), I don’t see why you can’t accept the possibility that life’s bush was the product of his experiments, or H. sapiens came late on in his thinking. Why is that more “human” than making mistakes?

I didn't say the biological errors were God's mistakes, but implied it is probably impossible for a high speed biochemical system to always be perfect. God cannot achieve that result which requires perfect molecular reactions at all times.

Treating congenital defects with gene editing

by Balance_Maintained @, U.S.A., Sunday, July 19, 2020, 06:46 (71 days ago) @ David Turell

This is a pretty amazing breakthrough. I really love it when science can solve problems like this. My major concern with it is, and always has been, that humans have a tendency to play with things they don't understand. Our biology is complex beyond compare, and in complex systems it is very, very difficult to understand how one change can impact seemingly unrelated systems. Just ask any programmer.

--
What is the purpose of living? How about, 'to reduce needless suffering. It seems to me to be a worthy purpose.

Treating congenital defects with gene editing

by David Turell @, Sunday, July 19, 2020, 20:18 (70 days ago) @ Balance_Maintained

Tony: This is a pretty amazing breakthrough. I really love it when science can solve problems like this. My major concern with it is, and always has been, that humans have a tendency to play with things they don't understand. Our biology is complex beyond compare, and in complex systems it is very, very difficult to understand how one change can impact seemingly unrelated systems. Just ask any programmer.

Looks safe so far. We don't understand a lot about genomes. Right to be careful.

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