Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases
The complexity and organization of the central nervous system (CNS) is widely modulated by the presence of the blood–brain barrier (BBB) and the blood–cerebrospinal fluid barrier (BCSFB), which both act as biochemical, dynamic obstacles impeding any type of undesirable exogenous exchanges. The disru...
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| Format: | Article |
| Language: | English |
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MDPI AG
2021-01-01
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| Series: | Pharmaceuticals |
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| Online Access: | https://www.mdpi.com/1424-8247/14/2/109 |
| _version_ | 1797405746852265984 |
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| author | Abdelfattah Faouzi Valérie Gaëlle Roullin |
| author_facet | Abdelfattah Faouzi Valérie Gaëlle Roullin |
| author_sort | Abdelfattah Faouzi |
| collection | DOAJ |
| description | The complexity and organization of the central nervous system (CNS) is widely modulated by the presence of the blood–brain barrier (BBB) and the blood–cerebrospinal fluid barrier (BCSFB), which both act as biochemical, dynamic obstacles impeding any type of undesirable exogenous exchanges. The disruption of these barriers is usually associated with the development of neuropathologies which can be the consequence of genetic disorders, local antigenic invasions, or autoimmune diseases. These disorders can take the shape of rare CNS-related diseases (other than Alzheimer’s and Parkinson’s) which a exhibit relatively low or moderate prevalence and could be part of a potential line of treatments from current nanotargeted therapies. Indeed, one of the most promising therapeutical alternatives in that field comes from the development of nanotechnologies which can be divided between drug delivery systems and diagnostic tools. Unfortunately, the number of studies dedicated to treating these rare diseases using nanotherapeutics is limited, which is mostly due to a lack of interest from industrial pharmaceutical companies. In the present review, we will provide an overview of some of these rare CNS diseases, discuss the physiopathology of these disorders, shed light on how nanotherapies could be of interest as a credible line of treatment, and finally address the major issues which can hinder the development of efficient therapies in that area. |
| first_indexed | 2024-03-09T03:15:36Z |
| format | Article |
| id | doaj.art-146a822c665d48789b6713b1ac857943 |
| institution | Directory Open Access Journal |
| issn | 1424-8247 |
| language | English |
| last_indexed | 2024-03-09T03:15:36Z |
| publishDate | 2021-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Pharmaceuticals |
| spelling | doaj.art-146a822c665d48789b6713b1ac8579432023-12-03T15:21:43ZengMDPI AGPharmaceuticals1424-82472021-01-0114210910.3390/ph14020109Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS DiseasesAbdelfattah Faouzi0Valérie Gaëlle Roullin1Center for Clinical Pharmacology, St. Louis College of Pharmacy and Washington University School of Medicine, St. Louis, MO 63131, USALaboratoire de Nanotechnologies Pharmaceutiques, Faculté de Pharmacie, Université de Montréal, Montréal, QC H3T 1J4, CanadaThe complexity and organization of the central nervous system (CNS) is widely modulated by the presence of the blood–brain barrier (BBB) and the blood–cerebrospinal fluid barrier (BCSFB), which both act as biochemical, dynamic obstacles impeding any type of undesirable exogenous exchanges. The disruption of these barriers is usually associated with the development of neuropathologies which can be the consequence of genetic disorders, local antigenic invasions, or autoimmune diseases. These disorders can take the shape of rare CNS-related diseases (other than Alzheimer’s and Parkinson’s) which a exhibit relatively low or moderate prevalence and could be part of a potential line of treatments from current nanotargeted therapies. Indeed, one of the most promising therapeutical alternatives in that field comes from the development of nanotechnologies which can be divided between drug delivery systems and diagnostic tools. Unfortunately, the number of studies dedicated to treating these rare diseases using nanotherapeutics is limited, which is mostly due to a lack of interest from industrial pharmaceutical companies. In the present review, we will provide an overview of some of these rare CNS diseases, discuss the physiopathology of these disorders, shed light on how nanotherapies could be of interest as a credible line of treatment, and finally address the major issues which can hinder the development of efficient therapies in that area.https://www.mdpi.com/1424-8247/14/2/109CNS disordersrare pathologiesorphan diseasesnanotechnologiesnanomedicinedrug delivery systems |
| spellingShingle | Abdelfattah Faouzi Valérie Gaëlle Roullin Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases Pharmaceuticals CNS disorders rare pathologies orphan diseases nanotechnologies nanomedicine drug delivery systems |
| title | Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases |
| title_full | Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases |
| title_fullStr | Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases |
| title_full_unstemmed | Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases |
| title_short | Think Big, Start Small: How Nanomedicine Could Alleviate the Burden of Rare CNS Diseases |
| title_sort | think big start small how nanomedicine could alleviate the burden of rare cns diseases |
| topic | CNS disorders rare pathologies orphan diseases nanotechnologies nanomedicine drug delivery systems |
| url | https://www.mdpi.com/1424-8247/14/2/109 |
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