Recent important achievements in gene therapy research field!

In this paper, I have compiled many research results, and jointly interpreted the new achievements of scientists in the field of gene therapy research, and shared them with you! Photo source: www.pixabay.com [1] JCI: new technology improves the effect of gene therapy on vision doi: 10.1172/jci129085 in the experiment with rats, pigs and monkeys as models, researchers from Johns Hopkins University School of medicine developed a gene therapy to save vision If it is proved to be safe and effective for human beings, the technology can provide a new and more durable treatment option for patients with common diseases such as wet age-related macular degeneration (AMD), and it may replace the defective genes in patients with hereditary retinopathy This new method was published in the Journal of clinical investigation recently The method uses a small needle to inject harmless, genetically engineered virus particles into the space between the whites of the eyes and the vascular layer of the eyes (known as the suprachoroidal cavity) From there, the virus can transfer the therapeutic gene to retinal cells Although currently only tested in animals, the new suprachoroidal injection technology is less invasive because it does not involve the separation of retina, and can be carried out in the outpatient clinic in theory, which marks the advent of a new gene therapy for vision 【2】 Science: phase I clinical trials show that drug-induced local IL-12 gene therapy is promising for the treatment of recurrent glioblastoma patients Doi: 10.1126/scitranslmed.aaw5680 in a new clinical study, an inducible tumor targeting gene therapy has been tested for the first time in glioblastoma patients This two-part approach involves injecting a gene encoding an immune activator into a brain tumor and taking a drug that activates the gene, leading to the production of the immune activator, interleukin-12 (IL-12), and the infiltration of immune cells into the tumor tissue These results also suggest that this treatment may prolong the survival of patients, and the relevant research results are published in the Journal of Science Translational Medicine According to researcher Frederick Lang, all the recent evidence suggests that if you can really let the immune system attack tumors, then you have the potential to increase the cure of tumors, and this [research] is moving in this direction I think it's exciting Glioblastoma is an aggressive and fatal form of brain cancer with an average survival of only 6 to 9 months after diagnosis Antonio chiocca, a neurosurgeon at Harvard Medical School who led the study, said that even with standard treatment (surgical removal of all or part of the tumor followed by six weeks of chemoradiotherapy), "on average.. The tumor relapsed within seven months." 【3】 SCI Rep: in-depth analysis of the function of microRNAs molecules or the development of new disease gene therapy doi: 10.1038/s41598-019-46841-1, published in the international journal scientific In the Research Report on reports, scientists from the University of eastern Finland and Oxford University found that microRNA, a naturally occurring microRNA molecule in cells, may also exist in large quantities in the nucleus Previously, microRNAs were thought to exist mainly in the cytoplasm, but the researchers found that the concentration of microRNA in the nucleus would change due to hypoxia, and microRNA It plays a key role in the expression of nuclear genes, and the relevant research results are very important for the development of new gene therapy In this paper, the researchers analyzed the characteristics of microRNAs molecules in different parts of endothelial cells, and found that most of microRNAs would be enriched in the nucleus When the researchers exposed the cell culture to hypoxia, they found that the change of single microRNA concentration mainly occurred in the cytoplasm or nucleus, and the role of microRNA in regulating gene expression in cells For example, the researchers found that a molecule, microrna-210, which was previously thought to be related to hypoxia, is actually more abundant in the nucleus, which may reveal the molecular mechanism of cell adaptation to hypoxia 【4】 Circulation: using gene therapy to inhibit CaMKII is expected to treat CPVT hereditary arrhythmia doi: 10.1161/circulationaha.118.038514 in a new study, researchers from Boston Children's Hospital in the United States reported that gene therapy can inhibit the disease in the model of hereditary arrhythmia in mice These findings provide the possibility for the development of single dose gene therapy for hereditary arrhythmias, and also for the treatment of more common arrhythmias (such as atrial fibrillation) The relevant research results were recently published in the Circulation Journal Dr VASSILIOS bezzerides, the researcher, said, "we hope to provide a single dose gene therapy that will work in the long term Our research provides a conceptual validation for the development of transformable gene therapy strategies for the treatment of hereditary arrhythmias This study focuses on catecholaminergic pleomorphic ventricular tachycardia (CPVT), which is the main cause of sudden death in children and young people This type of arrhythmia is usually caused by exercise or mental stress and occurs for the first time at an average age of 12, usually with sudden loss of consciousness 【5】 SCI trans Med: gene therapy - a new way to treat vision doi: 10.1126/scitraslmed.aax2324 in a recent article, the author summarized the recent treatment strategies for blinding diseases Gene replacement or gene editing strategies may potentially reverse vision loss In the early stage of retinal degeneration, early intervention is particularly promising when photoreceptor cells (rod cells and cone cells) are still intact The first gene therapy approved for Leber congenital amaurosis (LCA) confirmed the mutation of the double allele RPE65, paving the way for more than 30 gene substitution trials under other conditions in the world The gene independent strategy aims to prevent or slow down the progressive degeneration of photoreceptor cells with a broad-spectrum neuroprotective agent for retinal dystrophy Neuroprotective strategies, especially the preservation of cones, are the best way to treat diseases in which photoreceptor degeneration is taking place Stem cell therapy, optogenetic therapy and retinal prosthesis are used to restore vision in the later stage of retinal degeneration These methods can be used independently of causal mutations and are expected to restore low vision in blind patients Stem cell therapy for the replacement of degenerated cells to restore vision is being developed or clinically evaluated in a wide range of retinal degenerative diseases Photo source: Nature [6] nature: gene therapy promotes cardiac regeneration doi: 10.1038/s41586-019-1191-6 researchers from King's College London found that one therapy can induce cardiac cell regeneration after a heart attack According to the data of the World Health Organization, myocardial infarction is the main cause of heart failure, which is usually called heart attack It is caused by the sudden blockage of the coronary artery At present, more than 23 million people are affected by this disease At present, when a patient survives a heart attack, their heart will leave permanent structural damage, forming scars, which may lead to future heart failure In sharp contrast to fish and salamanders, salamander hearts can regenerate in their lifetime; in the study recently published in nature, the team implanted a small piece of genetic material called microrna-199 into the heart of pigs This gene can promote almost complete recovery of heart function in pigs one month after myocardial infarction 【7】 PNAs: gene therapy can treat congenital deafness in mice doi: 10.1073/pnas.1817537116 in collaboration with universities in Miami, Colombia and San Francisco, scientists from Pasteur Institute, INSERM, CNRS, French College, Sorbonne University and Claremont Auvergne University have successfully restored the hearing of dfnb9 deaf mice, which is the most common congenital inheritance One of the cases of sexual deafness Individuals with dfnb9 deafness lack the gene encoding otoferlin, a protein essential for the transmission of sound information in the synapses of auditory and sensory cells By injecting the gene into the cochlea of an adult dfnb9 mouse model, the scientists successfully restored the auditory synaptic function and hearing threshold to near normal levels These findings, published in PNAS, open up a new way for future gene therapy trials in patients with dfnb9 More than half of the nonsyndromic congenital deafness patients have genetic causes, most of them (about 80%) are caused by autosomal recessive hereditary deafness (dfnb) Cochlear implant is the only choice to restore the hearing of these patients Dfnb9 deafness is caused by mutations in the gene encoding otoferlin, a key protein in the synaptic transmission of sound information in inner hair cells Although there are no detectable defects in the sensory epithelium, these synapses are unable to release neurotransmitters in response to sound stimulation, so the mutant mice lacking ear ferritin are very deaf Therefore, dfnb9 mice constitute a suitable model for testing the efficacy of viral gene therapy in late application 【8】 Brain: gene therapy can promote the regeneration of neurons doi: 10.1093/brain/awy340 researchers from the Netherlands Institute of Neuroscience (NIN) and the University of Leiden Medical Center (lumc) have shown that the use of gene therapy can recover faster after nerve injury By combining surgical repair procedures with gene therapy, the survival of nerve cells and the regeneration of long-distance nerve fibers were stimulated for the first time The findings, published in the journal Brain, are an important step toward developing new treatments for patients with neurological injury After birth or a traffic accident, the nerves in the neck may be torn from the spinal cord As a result, these patients lost arm function and were unable to perform daily activities At present, surgical repair is the only available treatment for patients with this kind of nerve injury "After surgery, nerve fibers must bridge several centimeters before reaching muscles and nerve cells, and the new fibers need to regenerate a large number of nerve cells lost Most regenerated nerve fibers do not reach muscles So the recovery of arm function is disappointing and incomplete, "the authors said 【9】 Cell stem cell: gene therapy can be used to treat the fatal autoimmune disease doi: 10.1016/j.stem.2018.12.003 recently, UCLA researchers created a method to repair gene mutations in blood stem cells, in order to reverse the occurrence of life-threatening autoimmune syndrome (called IPEX) This work is published in the latest cell stem cell magazine IPEX is an autoimmune disease caused by a mutation The mutation blocks the expression of a gene called Foxp3, which prevents the production of blood stem cells to differentiate into regulatory T cells Regulatory T cells can negatively control the activity of the body's immune system Without such cells, the immune system will attack the body's own tissues and organs, and eventually lead to the occurrence of autoimmune diseases 【10】 SCI adv: new gene therapy is expected to conquer multiple blood diseases doi: 10.1126/sciadv.aau6762 gene therapy has great potential in the field of medical research, if we can