دورية أكاديمية
Intraparenchymal delivery of adeno-associated virus vectors for the gene therapy of neurological diseases.
| العنوان: | Intraparenchymal delivery of adeno-associated virus vectors for the gene therapy of neurological diseases. |
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| المؤلفون: | Kumagai S; Department of Neurosurgery, Jichi Medical University, Tochigi, Japan., Nakajima T; Department of Neurosurgery, Jichi Medical University, Tochigi, Japan., Muramatsu SI; Division of Neurological Gene Therapy, Jichi Medical University, Tochigi, Japan. |
| المصدر: | Expert opinion on biological therapy [Expert Opin Biol Ther] 2024 Aug; Vol. 24 (8), pp. 773-785. Date of Electronic Publication: 2024 Jul 31. |
| نوع المنشور: | Journal Article; Review |
| اللغة: | English |
| بيانات الدورية: | Publisher: Taylor & Francis Country of Publication: England NLM ID: 101125414 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1744-7682 (Electronic) Linking ISSN: 14712598 NLM ISO Abbreviation: Expert Opin Biol Ther Subsets: MEDLINE |
| أسماء مطبوعة: | Publication: 2015- : Abingdon, Oxford : Taylor & Francis Original Publication: London : Ashley Publications Ltd., c2001- |
| مواضيع طبية MeSH: | Dependovirus*/genetics , Genetic Therapy*/methods , Genetic Vectors*/genetics, Humans ; Animals ; Parkinson Disease/therapy ; Parkinson Disease/genetics ; Nervous System Diseases/genetics ; Nervous System Diseases/therapy ; Gene Transfer Techniques ; Aromatic-L-Amino-Acid Decarboxylases/genetics ; Aromatic-L-Amino-Acid Decarboxylases/deficiency ; Amino Acid Metabolism, Inborn Errors |
| مستخلص: | Introduction: In gene therapy with adeno-associated virus (AAV) vectors for diseases of the central nervous system, the vectors can be administered into blood vessels, cerebrospinal fluid space, or the brain parenchyma. When gene transfer to a large area of the brain is required, the first two methods are used, but for diseases in which local gene transfer is expected to be effective, vectors are administered directly into the brain parenchyma. Areas Covered: Strategies for intraparenchymal vector delivery in gene therapy for Parkinson's disease, aromatic l-amino acid decarboxylase (AADC) deficiency, and epilepsy are reviewed. Expert Opinion: Stereotactic intraparenchymal injection of AAV vectors allows precise gene delivery to the target site. Although more surgically invasive than intravascular or intrathecal administration, intraparenchymal vector delivery has the advantage of a lower vector dose, and preexisting neutralizing antibodies have little effect on the transduction efficacy. This approach improves motor function in AADC deficiency and led to regulatory approval of an AAV vector for the disease in the EU. Although further validation through clinical studies is needed, direct infusion of viral vectors into the brain parenchyma is expected to be a novel treatment for Parkinson's disease and drug-resistant epilepsy. |
| فهرسة مساهمة: | Keywords: AAV vector; Parkinson’s disease; aromatic l-amino acid decarboxylase; epilepsy; stereotactic surgery |
| المشرفين على المادة: | EC 4.1.1.28 (Aromatic-L-Amino-Acid Decarboxylases) |
| SCR Disease Name: | Aromatic amino acid decarboxylase deficiency |
| تواريخ الأحداث: | Date Created: 20240727 Date Completed: 20240827 Latest Revision: 20240827 |
| رمز التحديث: | 20240827 |
| DOI: | 10.1080/14712598.2024.2386339 |
| PMID: | 39066718 |
| قاعدة البيانات: | MEDLINE |
PDF full text from Publisher | تدمد: | 1744-7682 |
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| DOI: | 10.1080/14712598.2024.2386339 |