{"title":"Viral and nonviral approaches.","authors":"Mikko Airavaara, Mart Saarma","doi":"10.1016/B978-0-323-90120-8.00008-3","DOIUrl":null,"url":null,"abstract":"<p><p>Neurodegenerative diseases pose a substantial unmet medical need, and no disease-modifying treatments exist. Neurotrophic factors have been studied for decades as a therapy to slow down or stop the progression of these diseases. In this chapter, we focus on Parkinson disease, the second most common neurodegenerative disorder, and on studies carried out with neurotrophic factors. We explore the routes of administration, how the invasive intracranial administration is the challenge, and different ways to deliver the therapeutic proteins, for example, gene therapy and protein therapy. This therapy concept has been developed to mostly work on the restoration of the lost nigrostriatal dopaminergic neuronal connectivity in the brain. However, in recent years, the center of attention of neurotrophic factors has been on maintaining proteostasis and dissolving and preventing protein inclusions called Lewy bodies. We describe the most studied neurotrophic factor families and compare different preclinical experiments that have been carried out. We also analyze several clinical trials and describe their challenges and breakthroughs and discuss the prospects and challenges of neurotrophic support as a therapy for neurodegenerative diseases. In this chapter, we discuss why they still do and why it is essential to continue to work with this area of neurorestorative research around neurotrophic factors.</p>","PeriodicalId":12907,"journal":{"name":"Handbook of clinical neurology","volume":"205 ","pages":"83-97"},"PeriodicalIF":0.0000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Handbook of clinical neurology","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1016/B978-0-323-90120-8.00008-3","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"Medicine","Score":null,"Total":0}
引用次数: 0
Abstract
Neurodegenerative diseases pose a substantial unmet medical need, and no disease-modifying treatments exist. Neurotrophic factors have been studied for decades as a therapy to slow down or stop the progression of these diseases. In this chapter, we focus on Parkinson disease, the second most common neurodegenerative disorder, and on studies carried out with neurotrophic factors. We explore the routes of administration, how the invasive intracranial administration is the challenge, and different ways to deliver the therapeutic proteins, for example, gene therapy and protein therapy. This therapy concept has been developed to mostly work on the restoration of the lost nigrostriatal dopaminergic neuronal connectivity in the brain. However, in recent years, the center of attention of neurotrophic factors has been on maintaining proteostasis and dissolving and preventing protein inclusions called Lewy bodies. We describe the most studied neurotrophic factor families and compare different preclinical experiments that have been carried out. We also analyze several clinical trials and describe their challenges and breakthroughs and discuss the prospects and challenges of neurotrophic support as a therapy for neurodegenerative diseases. In this chapter, we discuss why they still do and why it is essential to continue to work with this area of neurorestorative research around neurotrophic factors.
期刊介绍：
The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.
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