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Amyotrophic Lateral Sclerosis (ALS), one of motor neuron diseases (MNDs), which impairs upper and lower motor neurons, resulting in gradual weakness and atrophy of muscles all over the body. The incidence rate is about 2.2/100,000 in Europe and America, but slightly higher in China (4/100,000) with 30,000-50,000 new patients every year in China. The aver age age of disease onset is 55 and the mean survival time from disease onset is typically 3 to 5 years. So far, no effective treatment has been developed for this devastating disease in the world. Recently, pathologies of RNA-binding proteins (RBPs), including TDP-43 and FUS, have been shown in over 95% of ALS patients, indicating that misregulation of RBPs may act as a common mechanism for ALS. Dr. Yichang Jia and his Tsinghua research group have successfully generated many ALS mutant RBP animal models. Based on these unique models, they have identified several novel therapeutic targets for the devastating disease. In the next 2-3 years, SineuGene will focus on translating these basic research achievements to the clinic to benefit the patients.

Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease (MJD), is an autosomal dominantly inherited neurodegenerative disorder, which is characterized by a cytosine-adenine-guanine (CAG) trinucleotide repeat expansion within the ATXN3?gene, leading to?an extended polyglutamine tract (poly(Q)).SCA3 is most prevalent?SCA subtype?in mainland China, with a well-documented inverse correlation between the CAG repeat size and age of onset, and a positive correlation between expanded repeats and disease severity. The primary?symptoms of SAC3 include spasticity, hyperreflexia, Babinski reflex, nystagmus, and swallowing difficulties. As of now, there are no approved drugs for SCA3. Clinical interventions primarily focus on symptom management and slowing disease progression. SineuGene is actively developing an antisense oligonucleotide (ASO) therapy, aiming to ively reduce mutant but not wild-type ATXN3 levels in affected cells.

Parkinson's disease (PD) has an estimated more than 10 million patients globally, the second most common neurodegenerative disease after Alzheimer's disease (AD). The pathological features of PD include loss of dopaminergic neurons in the substantia nigra and the formation of Lewy bodies in dopaminergic neurons. The patients exhibit a complicated clinical profile, including motor, sleep, cognitive, and affective abnormalities. Currently, available treatments, including levodopa and deep brain stimulation, can only partially alleviate the symptoms, but not reverse the neuron loss. Therefore, SineuGene basic research on anti-oxidation-based neuroprotection mechanisms and CNS highly efficient gene delivery tools may benefit the patients.

Alzheimer's disease (AD) accounts for about 70% of senile dementia, which manifests a progressive decline of mental functions, including learning and memory. In the United States, about 10% of people aged 65 and over have Alzheimer's disease. Genetic factors account for 5%-15% of AD. In addition to familial APP and Presenilin mutations, ApoE4 a single nucleotide polymorphism (SNP) is highly associated with AD. The AD pathological features include β-Amyloid deposition, senile plaques, neurofibrillary tangles, and tau pathology. Because current available drugs can only temporarily and limitedly improve the clinical symptoms of AD patients, therefore, a great number of medical needs are unmet. Recently, TDP-43 pathology has been identified in 40% AD patients, suggesting that dysfunctions of RNA binding proteins underlie disease mechanisms of AD. Therefore, our gene therapy approaches for ALS patients may also benefit a group of that of AD with similar pathological hall marker, like TDP-43 pathology.