Genetics of Dravet Syndrome and its Targeted Therapy by Nanomedicine: A Roadmap for Future Treatment of Drug Resistant Seizures.

IF 2.4 4区 生物学 Q3 BIOCHEMISTRY & MOLECULAR BIOLOGY
Muhammad Ikram, Sufian Rasheed
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引用次数: 1

Abstract

According to the World Health Organization (WHO), epilepsy is the 4th most prevalent neurological disorder after migraine, stroke, and Alzheimer's disease. There are numerous types of epileptic syndrome that are reported in children; one of them is Dravet syndrome. It is a neurological disorder of infants' outset during the first year of life. Dravet syndrome is a genetically determined syndrome and the most studied form of genetic epilepsy. Nearly 70-80% of its cases are due to genetic alterations in the SCN1A gene, and almost 16% of cases are due to variations in the PCDH19 gene. Besides that, mutations in SCN1B, SCN2A, and GABRG2, including some novel genes, STXBP1, HCN1, and CDH2 have been observed in DS patients. It is a drug-resistant epileptic syndrome and its complete removal is still challenging. So, novel therapeutic techniques are being used to treat drug-resistant seizures. Recently, new strategies have been made to improve the neuron-specific targeting of AEDs encapsulated by nanocarriers. The nanocarriers will have a major contribution to nano-neuro medicines such as drug delivery, neuroimaging, neuroprotection, neurosurgery, and neuroregeneration. The nanotechnology-mediated techniques also have a fantastic success rate in gene therapy, as reported in recent years. The anti- epileptic drug delivery with the help of nanoparticles, at the targeted position, makes them applicable for the possible treatment of drug-resistant seizures and gives new hope to patients affected with it.

Dravet综合征的遗传学及其纳米医学靶向治疗:未来治疗耐药癫痫的路线图。
根据世界卫生组织(WHO)的数据,癫痫是继偏头痛、中风和阿尔茨海默病之后的第四大最常见的神经系统疾病。据报道,儿童有许多类型的癫痫综合征;其中之一是德拉韦特综合症。这是一种婴儿在生命的第一年开始出现的神经系统疾病。Dravet综合征是一种基因决定的综合征,也是研究最多的遗传性癫痫形式。近70-80%的病例是由于SCN1A基因的遗传改变,近16%的病例是由于PCDH19基因的变异。此外,在DS患者中还观察到SCN1B、SCN2A和GABRG2的突变,包括一些新的基因STXBP1、HCN1和CDH2。这是一种耐药癫痫综合征,完全去除它仍然具有挑战性。因此,新的治疗技术被用于治疗耐药性癫痫。近年来,人们提出了新的策略,以提高纳米载体封装的aed的神经元特异性靶向性。纳米载体将对纳米神经药物,如药物输送、神经成像、神经保护、神经外科和神经再生等方面做出重大贡献。据近年来的报道,纳米技术介导的技术在基因治疗中也有惊人的成功率。在纳米颗粒的帮助下,抗癫痫药物在靶向位置的传递,使它们适用于治疗耐药癫痫,并给受其影响的患者带来新的希望。
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来源期刊
Current molecular pharmacology
Current molecular pharmacology Pharmacology, Toxicology and Pharmaceutics-Drug Discovery
CiteScore
4.90
自引率
3.70%
发文量
112
期刊介绍: Current Molecular Pharmacology aims to publish the latest developments in cellular and molecular pharmacology with a major emphasis on the mechanism of action of novel drugs under development, innovative pharmacological technologies, cell signaling, transduction pathway analysis, genomics, proteomics, and metabonomics applications to drug action. An additional focus will be the way in which normal biological function is illuminated by knowledge of the action of drugs at the cellular and molecular level. The journal publishes full-length/mini reviews, original research articles and thematic issues on molecular pharmacology. Current Molecular Pharmacology is an essential journal for every scientist who is involved in drug design and discovery, target identification, target validation, preclinical and clinical development of drugs therapeutically useful in human disease.
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