Oligodendrocytes, the Forgotten Target of Gene Therapy DOI Creative Commons
Yasemin Güneş, Catherine Le Stunff, Pierre Bougnères

и другие.

Cells, Год журнала: 2024, Номер 13(23), С. 1973 - 1973

Опубликована: Ноя. 28, 2024

If the billions of oligodendrocytes (OLs) populating central nervous system (CNS) patients could express their feelings, they would undoubtedly tell gene therapists about frustration with other neural cell populations, neurons, microglia, or astrocytes, which have been favorite targets transfer experiments. This review questions why OLs left out most therapy attempts. The first explanation is that pathogenic role still discussed in CNS diseases. Another reason so-called ubiquitous CAG, CBA, CBh, CMV promoters—widely used studies—are unable poorly able to activate transcription episomal transgene copies brought by adeno-associated virus (AAV) vectors OLs. Accordingly, expression has either not found evaluated studies rodents non-human primates. aims current are give rightful place among cells future target and encourage researchers test effect OL transduction various

Язык: Английский

Lipid nanoparticle (LNP) mediated mRNA delivery in neurodegenerative diseases DOI

Seyedeh Ghazal Moosavi,

Niloufar Rahiman, Mahmoud Reza Jaafari

и другие.

Journal of Controlled Release, Год журнала: 2025, Номер unknown, С. 113641 - 113641

Опубликована: Март 1, 2025

Язык: Английский

Процитировано

0

Advances in clinical neurorestorative treatments of Parkinson’s disease DOI Creative Commons

Yixuan Yin,

Dongning Su,

Joyce S. T. Lam

и другие.

Journal of Neurorestoratology, Год журнала: 2025, Номер unknown, С. 100204 - 100204

Опубликована: Март 1, 2025

Язык: Английский

Процитировано

0

A Comprehensive Overview of the Current Status and Advancements in Various Treatment Strategies against Epilepsy DOI Creative Commons
Abdul Waris, Muhammad Siraj, Ayyaz Ali Khan

и другие.

ACS Pharmacology & Translational Science, Год журнала: 2024, Номер 7(12), С. 3729 - 3757

Опубликована: Ноя. 1, 2024

Epilepsy affects more than 70 million individuals of all ages worldwide and remains one the most severe chronic noncommunicable neurological diseases globally. Several neurotransmitters, membrane protein channels, receptors, enzymes, and, recently noted, various pathways, such as inflammatory mTORC complexes, play significant roles in initiation propagation seizures. Over past two decades, developments have been made diagnosis treatment epilepsy. Various pharmacological drugs with diverse mechanisms action other options developed to control seizures treat These include surgical treatment, nanomedicine, gene therapy, natural products, nervous stimulation, a ketogenic diet, gut microbiota, etc., which are developmental stages. Despite plethora options, one-third affected resistant current medications, while majority approved side effects, changes can occur, pharmacoresistance, effects on cognition, long-term problems, drug interactions, risks poor adherence, specific for certain psychological complications. Therefore, development new that no or minimal adverse is needed combat this deadly disease. In Review, we comprehensively summarize explain stages epilepsy well their status clinical trials advancements.

Язык: Английский

Процитировано

2

Adeno-Associated Virus Therapies: Pioneering Solutions for Human Genetic Diseases DOI Creative Commons
Dequan Liu, Li Tian, Lei Liu

и другие.

Cytokine & Growth Factor Reviews, Год журнала: 2024, Номер unknown

Опубликована: Сен. 1, 2024

Язык: Английский

Процитировано

2

Oligodendrocytes, the Forgotten Target of Gene Therapy DOI Creative Commons
Yasemin Güneş, Catherine Le Stunff, Pierre Bougnères

и другие.

Cells, Год журнала: 2024, Номер 13(23), С. 1973 - 1973

Опубликована: Ноя. 28, 2024

If the billions of oligodendrocytes (OLs) populating central nervous system (CNS) patients could express their feelings, they would undoubtedly tell gene therapists about frustration with other neural cell populations, neurons, microglia, or astrocytes, which have been favorite targets transfer experiments. This review questions why OLs left out most therapy attempts. The first explanation is that pathogenic role still discussed in CNS diseases. Another reason so-called ubiquitous CAG, CBA, CBh, CMV promoters—widely used studies—are unable poorly able to activate transcription episomal transgene copies brought by adeno-associated virus (AAV) vectors OLs. Accordingly, expression has either not found evaluated studies rodents non-human primates. aims current are give rightful place among cells future target and encourage researchers test effect OL transduction various

Язык: Английский

Процитировано

0