Can gene therapy be used to treat autism?
Qiu Zilong, a researcher at the Institute of Neuroscience of the Chinese Academy of Sciences (self-proclaimed “Qiu Dao” on the Internet), recently gave a speech entitled “Gene Therapy: A Glimmering in the Autism Fog”, when the shell net published its speech I used a scary headline "I want to use genetic therapy to solve autism and let the world come to China to cure diseases." In fact, most of the speeches of Qiu Zilong are about gene sequencing and gene therapy for other diseases. What is related to autism is that he simply mentioned that he is studying Reiter syndrome and hopes to treat it with gene therapy in the future. He said that this is a kind of autism spectrum disorder, so he became a gene therapy for autism.
Although the early symptoms of Reiter's syndrome are similar to autism, they are not autistic and are not a form of autism spectrum disorder. Previously, the American Psychiatric Association classified Rett syndrome as a generalized developmental disorder with autism, Asperger's syndrome, childhood disintegration disorders, and unspecified generalized developmental disorders. However, in the Fifth Edition of the Diagnostic and Statistical Manual of Mental Disorders (DSM-5), which was published in 2013, the Society changed the generalized developmental disorder to autism spectrum disorder, but removed the Reiter syndrome from the manual. Therefore, Reiter syndrome has never been classified as an autism spectrum. This is because the Reiter syndrome has long been found to be caused by a mutation in the gene called MECP2, which is a single-gene genetic disease, and even mental illness is not considered.
Conversely, the genetic factors of the autism spectrum are very complex, and there are more than one hundred genes involved. Moreover, the occurrence of autism is not entirely caused by genes, but also environmental factors and epigenetic factors. In an interview, Qiu Zilong asserted that autism is completely genetic, and that there are no non-genetic factors, indicating that he is very unfamiliar with the research progress of autism.
For single-gene genetic diseases like Reiter syndrome, it is possible to treat with gene therapy. The United States AveXis (later acquired by Novartis Pharmaceuticals) began research a few years ago, and announced in August this year that it has completed preclinical studies and is preparing to apply for clinical trials. And Qi Zilong’s gene therapy is still only on his lips, but he is imagining that the whole world is coming to him for treatment.
But for autism spectrum disorders involving many genes, gene therapy is currently not available. Qiu Zilong retorted: "Although there are hundreds of genes that cause autism, there is usually only one important genetic mutation in a patient. Why can't gene therapy? Reiter syndrome can be gene therapy, and other autism can of course." Qiu Zilong does not seem to know how many genes are inherited. At present, only a small number of autism spectrum disorders are known to involve single gene mutations, and most involve multiple genes. In a patient, there are multiple gene mutations related to autism. How to treat them? The inheritance of autism is very complicated, beyond the imagination of the child.
Reiter syndrome is a rare genetic disease that occurs in about 10,000 people, and autism spectrum disorders are very common, ranging from 1% to 2% in the population. Therefore, it is not difficult to understand why Qiu Zilong has to say that Reiter syndrome is autism, which is to raise the importance of its research. If you only say Reiter's syndrome, how many people have heard of this rare genetic disease? It is well known that it is autism, and it is easy to apply for funds and sell yourself.
At the beginning, Han Chunyu’s gene editing technology of Hebei University of Science and Technology was questioned. When many people could not repeat the results, Qiu Zilong announced that he repeated the results of Han Chunyu, the only one in the world that claimed to repeat Han Chunyu’s results. of. Does Qiu Zilong's research on Reiter syndrome be as reliable and credible as his research on Han Chunyu's gene editing? Or, just to apply for funding and sell yourself, just say that? Of course, we can't rule out that Qiu Zilong got the true biography from Han Chunyu. His research on Reiter syndrome, like Han Chunyu's research on gene editing, has "academic secrets" that foreigners cannot know.