{"title":"Clinical/surgical considerations.","authors":"Tom Foltynie, Ludvic Zrinzo","doi":"10.1016/B978-0-323-90120-8.00006-X","DOIUrl":null,"url":null,"abstract":"<p><p>While there are a diverse number of indications for the potential use of cell and gene therapies in people, many of the medical conditions being treated need to consider some general clinical and surgical issues, not only in designing clinical trials, but also in the eventual application of the therapy if shown to be successful. Such issues include the precision of the diagnosis and stage of disease, the presence of significant comorbidity, as well as the challenges that may be encountered in trial designs involving the neurosurgical delivery of these advanced interventions. This chapter discusses details of these issues that have arisen particularly in the field of Parkinson disease and other forms of neurodegeneration, although the general principles and considerations discussed may be equally relevant to other neurologic and nonneurologic conditions.</p>","PeriodicalId":12907,"journal":{"name":"Handbook of clinical neurology","volume":"205 ","pages":"101-110"},"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/B978-0-323-90120-8.00006-X","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
While there are a diverse number of indications for the potential use of cell and gene therapies in people, many of the medical conditions being treated need to consider some general clinical and surgical issues, not only in designing clinical trials, but also in the eventual application of the therapy if shown to be successful. Such issues include the precision of the diagnosis and stage of disease, the presence of significant comorbidity, as well as the challenges that may be encountered in trial designs involving the neurosurgical delivery of these advanced interventions. This chapter discusses details of these issues that have arisen particularly in the field of Parkinson disease and other forms of neurodegeneration, although the general principles and considerations discussed may be equally relevant to other neurologic and nonneurologic conditions.
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.