Thursday, October 13, 2016

There May Be a Cure for Sickle Cell Anemia but It Requires Genetic Modification

Due to the way the cure is engineered, it's a distinct possibility you may not want it although sickle cell anemia is a terrible affliction. (Science Daily: Genome engineering paves way for sickle cell cure)

There's no comic aside on this one since it's serious disease affecting many people, mostly black when their heritage is sub-Saharan Africa.


If I'm in the situation and I know I'm a carrier of sickle cell, I've got one hell of a tough call in front of me.  Will I go with allowing scientists to use my sperm cells (or whatever source) to perform their genetic manipulation so I can have reasonable assurance my kid will be healthy.

I have no kids so I'm really not in a position to know how much I would have felt I needed to go with this solution.  Therefore, this goes to a toss-up question and if you don't do it then your kid may die.

Here's the intro to the science:

A team of physicians and laboratory scientists has taken a key step toward a cure for sickle cell disease, using CRISPR-Cas9 gene editing to fix the mutated gene responsible for the disease in stem cells from the blood of affected patients.

For the first time, they have corrected the mutation in a proportion of stem cells that is high enough to produce a substantial benefit in sickle cell patients.

- Science Daily


And one more on the background with sickle cell:

Sickle cell disease is a recessive genetic disorder caused by a single mutation in both copies of a gene coding for beta-globin, a protein that forms part of the oxygen-carrying molecule hemoglobin. This homozygous defect causes hemoglobin molecules to stick together, deforming red blood cells into a characteristic "sickle" shape.  These misshapen cells get stuck in blood vessels, causing blockages, anemia, pain, organ failure and significantly shortened lifespan.  Sickle cell disease is particularly prevalent in African Americans and the sub-Saharan African population, affecting hundreds of thousands of people worldwide.

- Science Daily

In English, if you carry the sickle cell gene and your wife does not then your kid will be a carrier.  It's a recessive gene so it must come from both parents (i.e. homozygosity) to take the full effect.  I'm not sure if it's fatal if the kid inherits the gene from both parents but it's not at all good.

It seems to me I have two choices with the first being a vasectomy and the second being to go through the genetic procedure.  It seems the procedure applies to the progeny rather than the sex cells of the parents which make the kid and that makes a whole different decision but still the situation is in my face because my wife is recessive, just as I am, so what is my move either way.

Again, this is all hypothetical but we sympathize / empathize greatly with anyone who has to make such a decision.

So there's your toss-up question:  what will you do?

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