Gene editing for autism: a new form of lobotomy?

This article from Newsweek appeared in my inbox and I read it with great surprise -- and alarm:


Scientists have figured how to use a form of the powerful gene-editing tool CRISPR to erase genetic traits normally associated with autism. It is technology that could one day revolutionize the therapies that treat autism and improve the lives of thousands of people who suffer from the developmental disorder, the researchers say.

CRISPR has been a game changer in the biomedical research world because of the ease and precision with which it can be used to alter the genetic code. A team of scientists in Texas used it to edit out genes associated with autism traits in mice and observed clear results: The animals stopped digging obsessively, their erratic jumping around the cages slowed to a halt and they became more calm, according to the study published in the monthly journal Nature Biomedical Engineering.

This technology might be years away from being tested on humans, but the results are promising, lead author of the study neuroscientist Hye Young Lee told Newsweek.

Lee, an autism researcher at the University of Texas Health Science Center at San Antonio, used a very thin needle to inject CRISPR-Cas9 into the brain tissue of mice, targeting the striatum, a region in the brain known to mediate habit formation.

CRISPR-Cas9 is made of just a few ingredients. The RNA, a nucleic acid present in all living cells, is like a messenger, a guide that allows a specific site on the genome to be targeted. It is used along with a bacterial enzyme, called Cas9, which acts like  molecular scissors, cutting the DNA at an exact point……

Thin NEEDLE INJECTED into the BRAIN -- SCISSORS --- CUTTING?  That does have hints of this:

The lobotomy procedure was first done by the Portuguese neurologist, Egas Moniz, who believed that patients with obsessive behavior were suffering from fixed circuits in the brain. His original technique was then adapted by others, with similar procedures. Surgeons would drill a pair of holes into the skull, either at the side or top, and push a sharp instrument - a leucotome - into the brain and then the surgeon would sweep this from side to side, to cut the connections between the frontal lobes and the rest of the brain. By the 1940's it was being seen as a miracle cure, used to treat a range of illnesses, from schizophrenia to depression and compulsive disorders. Starting in the mid-1950s, it rapidly fell out of favor, partly because of poor results and partly because of the introduction of the first wave of effective psychiatric drugs, which were safer and more effective.

"I got increasingly conservative about it because I don't think any of us were ever really happy about putting in a brain needle and stirring the works," he says. "Not a nice thought."

                             - Psychiatrist Dr John Pippard after turning against lobotomies


Is this really supposed to help autism?  I read studies and much research,  plus also write about Megan and her many health issues  .  Of course there is the behavioral aspects of autism as it is a gut-brain disease we are talking about.  It includes the immune system  , that hugely important bacterial and viral stew that is turning out to be the driver   in many of these autoimmune diseases that affect the brain  . Autism is NOT a genetic condition, though it if often spun like it is so elaborate and expensive research can keep the cash cow happy.  It is a GUT-IMMUNE-BRAIN disease .

Further research brought these issues to light:

■ CRISPR could use gold nanoparticles to edit your Brain - In this study, the researchers used CRISPR-Gold to edit the brains of mice with fragile X syndrome, a genetic condition that is correlated with autism spectrum disorder (ASD). While fragile X syndrome is a genetic disorder that can be diagnosed in genetic testing, clinicians diagnose ASD based behavioral criteria like repetitive behaviors. That means that it’s hard to discuss the idea of a “cure” for autism, but it may be possible, using CRISPR-Gold, to treat the fragile X syndrome that causes ASD-correlated behaviors in some individuals.

The virus DNA in the brain can have side effects, because it doesn’t go away once the CRISPR operation is done. Using the gold nanoparticles removes the issue of foreign DNA that’s producing damaging proteins, one of the central issues of CRISPR. Still, leaving bits of gold in the brain isn’t good either,...

I want to emphasize that this is not the solution,” says Lee. She says it might be possible, using this technology, to allow autistic people to treat unwanted symptoms such as repetitive behaviors just like they might with medicine—with the difference that CRISPR changes would likely be permanent .

SO virus DNA or gold could be permanently left in the brain.  That this is aimed for the “genetic disorder, Fragile X” (2%-3% of all cases of asd), but then below it seems to include ALL of the autism spectrum. Plus, it’s not “the solution” yet it could be permanent. Sounds confusing!

■ Autism patients also tend to engage in repetitive behavior, like rocking back and forth and flapping their arms. The study suggests that these behaviors could be remedied through gene editing. These results could also open the door for further research on how to alter genes related to the other autism traits, said Lee. “I really want to give hope for patients and families. It is such a social burden on them ……. 

I must say that sounds offensive to me.  I am not looking to help my daughter because she is a SOCIAL BURDEN.  Megan has behaviors related to antibodies that will not let her brain rest.  LIke most with an asd diagnosis, the gut bacteria are too pathogenic with beneficial bacteria lacking. link She has OCD and tics with her gut and immune system as the source, and it can be exhausting.  She can have physical pain. She has immune dysfunction.  She is nonverbal. She has sensory overload.  She has had severe seizures. Diet, probiotics and supplements aimed at her immune system can help. Research is showing that immune treatments, like high-dose intravenous immunoglobulin  are proving to be very effective for autism. That makes sense and we need these asap to help so many very ill asd patients, but editing their brains ------ sounds like a sci-fi experiment.

■ People with autism demonstrate repetitive behaviors and impaired habit learning. Yet the neural circuits underlying these behaviors remain unclear. The dorsolateral striatum is a region of the brain that has been implicated in habit formation and repetitive behavior. Nevertheless, the link between dysfunction of this structure and autism-related repetitive behavior remains elusive. The striatum, the major input structure of the basal ganglia area of the brain,...........

■ Autism spectrum disorders (ASD) may be associated with neuropsychiatric symptoms such as tics, obsessive-compulsive behaviors, and other symptoms characteristic of pediatric autoimmune neuropsychiatric disorder associated with streptococcal infections (PANDAS). Abnormal anti-neuronal autoantibody responses against the basal ganglia are elevated in neuropsychiatric disorders such as PANDAS. Our study suggests that anti-neuronal autoantibodies in ASD may be associated with the presence of PANDAS and investigates microbial polysaccharides as potential inducers of anti-neuronal autoantibodies in children with confirmed ASD or ASD/PANDAS.

■ The development of efficient and reliable ways to make precise, targeted changes link  to the genome of living cells is a long-standing goal for biomedical researchers. Recently, a new tool based on a bacterial CRISPR-associated protein-9 nuclease (Cas9) from Streptococcus pyogenes has generated considerable excitement…….

Wow.  The last thing any of these children and young adults need entering their bodies are pathogenic BACTERIA or VIRUSES.

 Many of the proposed applications involve editing the genomes of somatic (non-reproductive) cells, but there has been interest in and debate about the potential to edit reproductive cells. However, any changes made in these cells will be passed on from generation to generation and, as such, have important ethical implications. ...CRISPRCas9 procedures on reproductive cells during early stages of pregnancy could be passed on to subsequent generations with unanticipated consequences…...

“Editing reproductive cells” sounds reminiscent of this:


 Human trials are expected to get underway in Western Countries this year, and China has been experimenting with CRISPR-Cas9 for a while. Now, however, a new study by Stanford University researchers has found that a high percentage of people may contain immune responses that could prevent CRISPR-Cas9’s from working, or even be dangerous.

The study analyzed blood for antibodies to two bacteria from which Cas9 is derived: Streptococcus pyogenes and Staphylococcus aureus. The researchers’ concern stemmed from the fact that these bacteria frequently cause infections in humans…..Of more serious concern is that the researchers also found T-cells that could attack cells producing Cas9 proteins in six out of 13 donors, because “The existence of the pre-existing cellular immunity would likely both limit the effectiveness of and create safety concerns for Cas9-based genome editing.

Stanford’s finding definitely puts a bit of a cloud over the future of CRISPR-Cas9

As we can see, it’s very important to do RESEARCH on the research.  Editing genes now or erasing them for future generations seems to have numerous negatives.  My daughter has an autism diagnosis that is related to her autoimmune diagnosis.  We are learning more and more about the gut-brain connection in autism  . Cutting and pasting DNA seems to be an expensive, money-maker with much danger associated to it.  Who is this really helping? Our children deserve better.


By Teresa Conrick / Science Editor to Age of Autism
(Source:; July 4, 2018;
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