SCI transl Med: scientists are expected to develop new therapies for Parkinson's disease

November 3, 2019 / BIOON / -- recently, a report was published in the international journal Science Translational Medicine In the previous report, scientists from Northwestern University in the United States developed and tested a new Parkinson's disease therapy by using neurons derived from patients' bodies, which can improve the treatment of Parkinson's patients by slowing down the effect of harmful gene mutations Image source: Wikipedia some for genetics The experimental therapy of sexual disorders can target the mutated proteins or enzymes, but in this study, researchers use a completely different method to replace the damaged enzymes Researchers enlarge the function of healthy enzymes, so as to study human brain cells and mouse models, and successfully alleviate the symptoms of Parkinson's disease patients The researchers point out that the activation of wild-type glucocerebrosidase (gcase) may be a potential therapeutic target for many forms of Parkinson's disease Parkinson's disease is the second most common neurodegenerative disease It mainly affects the function of neurons in the substantia nigra area of the brain These neurons are mainly responsible for the production of dopamine, a chemical messenger used to transmit signals through the brain At the same time, these neurons can also transmit information related to planning and controlling the movement of the body Only the mutation of gba1 can represent the most common genetic risk factor of Parkinson's disease Gba1 can encode a special enzyme called glucocerebrosidase (gcase), which is very important for the function of neurons Mutations related to Parkinson's disease can promote the deactivation of gba1 and the production of abnormal gcase enzymes, which lead to the accumulation of dopamine producing neuronal toxic proteins With the death of neurons, patients will have symptoms such as tremor and tardiness Because some treatments can improve the symptoms of patients, but there is no treatment to block the disease at present The researchers point out that drug development for gba1 related Parkinson's disease focuses on the function of stable mutant gcase and limits its harmful effects, however, these therapies can only play a role in a small number of Parkinson's disease patients; on the contrary, activation of wild-type gcase may be related to multiple forms of Parkinson's disease with reduced activity of wild-type gcase In the current research, researchers have developed a series of new chemical activators, which can stabilize and amplify the activity of normal gcase These small molecular activators that can combine with gcase can improve the cell dysfunction related to Parkinson's disease; more importantly, they can also play a role in many Parkinson's diseases Researcher Krainc said that this study points out the potential of regulating wild type GCase activity and protein levels in genetic and idiopathic Parkinson disease, and highlights the importance of individualized or precise neuropathy therapy development In 2017, researcher krainc and his colleagues pointed out in the journal Science that some key pathological features of Parkinson's disease can only be observed in human neurons, not in mouse models; this emphasized the importance and value of patient derived neurons in the development of Parkinson's disease drugs The researchers hope to conduct more in-depth analysis and detection of human neurons to develop candidate drugs that can effectively target dopaminergic neurons in patients with Parkinson's disease Original sources: Lena f burbula, Sohee Jeon, Jianbin Zheng, et al A Modulor of wild type glycorebrosidase improvements pathogic cryptotypes in dopaminergic national models of Parkinson's disease, Science Translational Medicine (2019) Doi: 10.1126/scitranslmed.aau6870