CRISPR Gene Editing Fixes Muscular Dystrophy In Dogs, Humans Could Be Next (time.com)

schwit1 shares a report from Time: In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular biology at UT Southwestern Medical Center, reported that he and his team successfully used CRISPR to correct the genetic defect responsible for Duchenne muscular dystrophy in four beagles bred with the disease-causing gene. It's the first use of CRISPR to treat muscular dystrophy in a large animal. (Previous studies had tested the technology on rodents.) In varying degrees, the genetic therapy halted the muscle degradation associated with the disease. Duchenne is caused by mutations in the dystrophin gene, which codes for a protein essential for normal muscle function. People born with the disease are often eventually confined to wheelchairs as their muscles continue to weaken, and in the later stages, many rely on ventilators to breathe as their diaphragm muscles stop working. Eventually, they develop heart and respiratory failure.

Olson and his team "fixed" the mutated dystrophin gene in four dogs by splicing out an offending section of the gene using CRISPR. The gene editing technology, discovered in 2012, can cut out sections of DNA at precise locations (and also potentially introduce new DNA as well). In the case of Duchenne, says Olson, simply snipping out a section of the mutated dystrophin gene allows the gene to make enough of the proper protein that muscles need to function. The hope is that if those animal studies and human trials prove this technique is safe and effective, CRISPR could potentially lead to a cure for Duchenne, Olson says. "We are going for a cure, not a treatment," he says. "All of the other therapies so far for Duchenne muscular dystrophy have treated the symptoms and consequences of the disease. This is going right at the root cause of the genetic mutation."

Comments

Bill Hamilton would be optimistic now

[SurenEnfiajyan]

Genetic engineering improved beyond everything Hamilton could have dreamed of.

Re:Humanity 2.0

[klingens]

Wrong. Medical genetic engineering and GMOs are very different.Genetic engineering as up to now means "more poison" pretty much. All the Roundup ready poison, the Bt-corn poison,etc. The only exception there is golden rice from Asia. All our western corporations only want to put more poison into the environment and our bodies. So being against GMO is very much a no brainer. The mainly asian governments want to prevent blindness in poor people instead. While there hsa been some resistance against golden rice, it's mainly "but the people only need to eat a little more vegetables". Which comes from dumb rich western organizations who cannot comprehend that the poor asians simply don't have the money to buy this. There are no walmarts or rather Whole Foods supremarkets in rural Asia.

Medical genetic engineering is different. There you have insulin for diabetics and erythropoetin for anemic people. Granted, most of the erythropoetin is used for doping in sports, but it's still a godsend for anemic people on dialysis: no more 16h a day sleep. And for diabetics insulin a literal live saver.
Both of these drugs are wholly accepted in any society, even with the doping problem. So another genetic tech which saves lives will be accepted too

Will this repair the genes in the gametes?

[Solandri]

Gametes = sperm and egg cells. If you're not fixing the genetic defect in those, then this will actually make the situation worse. Previously, people with MD were less likely to reproduce because of the disease (it usually manifests between age 2-15, with most afflicted persons dying by their 20s). That kept the damaged gene sequence relatively rare in the population's gene pool. If we now use gene therapy to remove the negative symptoms of the disease, but without repairing the damaged gene sequence in the gametes, parents with MD will end up passing the disease on to their children. And eventually that sequence will end up spreading throughout the entire population. And we'll end up with a world where the a large percentage of people need this therapy just to have a normal life. According to TFA, this treatment has only been applied to muscle tissue (where the bad gene sequence is needed by the muscles).

People equate death = bad. But if the death results from bad genes, the death is actually good (for the species) because it's functioning to reduce the prevalence of the bad genes from the population's gene pool. What's bad for the individual may be good for the species.

An alternative is to require people receiving this treatment to consent to forced sterilization (there are plenty of kids who need adopting anyway). But sterilization is a touchy subject which encroaches on the abortion debate (you're saying society can override an individual's right to control their own body).

Re:Humanity 2.0

[morethanapapercert]

You're right. My son has DMD, the very disease this treatment is aimed at. I'd volunteer my son for this in a fucking heartbeat because the alternative is seeing him wither away and spend years struggling to even breathe before dying in his late 20's from cardiac or respiratory failure. Unfortunately, my son is already permanently wheelchair bound. He is already considered to have declined to far to be a viable test subject. That is a heart breaking disappointment we've had to swallow several times now as he keeps falling outside the study requirements for several different life extending trial therapies even as those trials accept worse and worse patients.

I've been following CRISPR with intense interest as a result of my son's condition and it really does look like a golden bullet for curing DMD. But early detection, preferably in-uterine detection, will be key. The reason being is that this treatment would essentially freeze the boys level of muscular competence. If you treat a child who has yet to show any symptoms, then he will likely never experience any symptoms. But if you treat a wheelchair bound 10 year old, he is not going to recover the ability to walk, he is going to be wheelchair bound for the rest of his life. The good news would be that this would greatly extend his life expectancy.