Literature DB >> 32557526

Advancing gene therapies, methods, and technologies for Parkinson's Disease and other neurological disorders.

Massimo S Fiandaca1, Russell R Lonser2, J Bradley Elder2, Mirosław Ząbek3, Krystof S Bankiewicz4,5,6.   

Abstract

INTRODUCTION: Vector-based intracerebral gene therapies are being used to treat specific neurodegenerative conditions such as Parkinson's Disease (PD). This review presents a basis for central nervous system (CNS) gene therapy treatments of neurodegenerative diseases such as PD, as well as the need for novel skill sets and health delivery strategies within the clinical neurosciences (neurology and neurosurgery) to meet future demand for such therapies. STATE OF THE ART: Preclinical vector-based gene therapy approaches have been translated into clinical trials for PD and other neurodegenerative conditions. Unfortunately, such trials, and parallel efforts using other therapeutics, have yet to provide a breakthrough. Image-guided convection enhanced delivery (CED) optimises the parenchymal distribution of gene therapies applied within the CNS, and may ultimately provide such a breakthrough. CLINICAL IMPLICATIONS: Currently, image-guided CED and gene therapy are not part of training programmes for most neurosurgeons and neurologists. As a result, few medical centres and hospitals have sufficiently experienced teams to participate in gene transfer clinical trials for PD or other neurological conditions. If CNS gene therapies prove to be efficacious for PD and/or other conditions, the demand for such treatments will overwhelm the available number of experienced clinical neuroscience teams and treatment centres. FUTURE DIRECTIONS: Expanded indications and demand for CNS gene therapies will require a worldwide educational effort to supplement the training of clinical neuroscience practitioners. Initially, a limited number of Centres of Excellence will need to establish relevant educational training requirements and best practice for such therapeutic approaches. Advanced technologies, including robotics and artificial intelligence, are especially germane in this regard, and will expand the treatment team's capabilities while assisting in the safe and timely care of those afflicted.

Entities:  

Keywords:  Parkinson’s Disease; artificial intelligence; clinical neuroscience education; convection-enhanced delivery; gene therapy; robotics

Mesh:

Year:  2020        PMID: 32557526     DOI: 10.5603/PJNNS.a2020.0046

Source DB:  PubMed          Journal:  Neurol Neurochir Pol        ISSN: 0028-3843            Impact factor:   1.621


  5 in total

Review 1.  Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities.

Authors:  Sarah J Tabrizi; Carlos Estevez-Fraga; Willeke M C van Roon-Mom; Michael D Flower; Rachael I Scahill; Edward J Wild; Ignacio Muñoz-Sanjuan; Cristina Sampaio; Anne E Rosser; Blair R Leavitt
Journal:  Lancet Neurol       Date:  2022-07       Impact factor: 59.935

Review 2.  Schizophrenia and Alarmins.

Authors:  Huan Ma; Ning Cheng; Caiyi Zhang
Journal:  Medicina (Kaunas)       Date:  2022-05-24       Impact factor: 2.948

3.  Frequency of mutations in PRKN, PINK1, and DJ1 in Patients With Early-Onset Parkinson Disease from neighboring countries in Central Europe.

Authors:  Łukasz M Milanowski; Jennifer A Lindemann; Dorota Hoffman-Zacharska; Alexandra I Soto-Beasley; Maria Barcikowska; Magdalena Boczarska-Jedynak; Angela Deutschlander; Gabriela Kłodowska; Jarosław Dulski; Lyuda Fedoryshyn; Andrzej Friedman; Zygmunt Jamrozik; Piotr Janik; Katherine Karpinsky; Dariusz Koziorowski; Anna Krygowska-Wajs; Barbara Jasińska-Myga; Grzegorz Opala; Anna Potulska-Chromik; Aleksander Pulyk; Irena Rektorova; Yanosh Sanotsky; Joanna Siuda; Jarosław Sławek; Katarzyna Śmiłowska; Lech Szczechowski; Monika Rudzińska-Bar; Ronald L Walton; Owen A Ross; Zbigniew K Wszolek
Journal:  Parkinsonism Relat Disord       Date:  2021-04-02       Impact factor: 4.891

4.  Parkinson's Disease Etiology: Insights and Associations with Phosphate Toxicity.

Authors:  Ronald B Brown
Journal:  Int J Mol Sci       Date:  2022-07-22       Impact factor: 6.208

Review 5.  Clinical, pathological and genetic characteristics of Perry disease-new cases and literature review.

Authors:  Jarosław Dulski; Catalina Cerquera-Cleves; Lukasz Milanowski; Alexa Kidd; Emilia J Sitek; Audrey Strongosky; Ana María Vanegas Monroy; Dennis W Dickson; Owen A Ross; Jolanta Pentela-Nowicka; Jarosław Sławek; Zbigniew K Wszolek
Journal:  Eur J Neurol       Date:  2021-08-26       Impact factor: 6.288

  5 in total

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