腺相关病毒载体在灵长类动物大脑中的引导传递。
Guided delivery of adeno-associated viral vectors into the primate brain.
机构信息
Department of Neurological Surgery, University of California San Francisco, San Francisco, CA 94103–0555, USA.
出版信息
Adv Drug Deliv Rev. 2012 May 15;64(7):598-604. doi: 10.1016/j.addr.2011.10.005. Epub 2011 Oct 20.
Abstract
In this review, we discuss recent developments in the delivery of adeno-associated virus-based vectors (AAV), particularly with respect to the role of axonal transport in vector distribution in the brain. The use of MRI-guidance and new stereotactic aiming devices have now established a strong foundation for neurological gene therapy to become an accepted procedure in interventional neurology.
摘要
在这篇综述中，我们讨论了腺相关病毒载体（AAV）传递的最新进展，特别是轴突运输在脑内载体分布中的作用。磁共振成像（MRI）引导和新型立体定向瞄准装置的使用，为神经基因治疗成为介入神经学中一种公认的方法奠定了坚实的基础。
相似文献
1
Guided delivery of adeno-associated viral vectors into the primate brain.
Adv Drug Deliv Rev. 2012 May 15;64(7):598-604. doi: 10.1016/j.addr.2011.10.005. Epub 2011 Oct 20.
2
AAV viral vector delivery to the brain by shape-conforming MR-guided infusions.
J Control Release. 2016 Oct 28;240:434-442. doi: 10.1016/j.jconrel.2016.02.034. Epub 2016 Feb 27.
3
MRI-Guided Delivery of Viral Vectors.
Methods Mol Biol. 2016;1382:217-30. doi: 10.1007/978-1-4939-3271-9_15.
4
[Characterization and application of adeno-associated virus serotype 8 as a gene transfer vector].
Bing Du Xue Bao. 2011 Jan;27(1):91-5.
5
Neuroimaging Applications for the Delivery and Monitoring of Gene Therapy for Central Nervous System Diseases.
Hum Gene Ther. 2024 Nov;35(21-22):886-895. doi: 10.1089/hum.2024.057. Epub 2024 Nov 8.
6
Adeno-associated viral vectors for gene therapy of inherited retinal degenerations.
Methods Mol Biol. 2013;935:351-69. doi: 10.1007/978-1-62703-080-9_25.
7
Adeno-associated virus (AAV) vectors in cancer gene therapy.
J Control Release. 2016 Oct 28;240:287-301. doi: 10.1016/j.jconrel.2016.01.001. Epub 2016 Jan 12.
8
Adeno-associated Virus as a Mammalian DNA Vector.
Microbiol Spectr. 2015 Aug;3(4). doi: 10.1128/microbiolspec.MDNA3-0052-2014.
9
Adeno-associated virus vector as a platform for gene therapy delivery.
Nat Rev Drug Discov. 2019 May;18(5):358-378. doi: 10.1038/s41573-019-0012-9.
10
Unlocking the potential of adeno-associated virus in neuroscience: a brief review.
Mol Biol Rep. 2024 Apr 22;51(1):563. doi: 10.1007/s11033-024-09521-6.
引用本文的文献
1
Artificial Bacteriophages for Treating Oral Infectious Disease via Localized Bacterial Capture and Enhanced Catalytic Sterilization.
Adv Sci (Weinh). 2024 Nov;11(41):e2400394. doi: 10.1002/advs.202400394. Epub 2024 Aug 19.
2
Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.
Adv Drug Deliv Rev. 2024 Aug;211:115363. doi: 10.1016/j.addr.2024.115363. Epub 2024 Jun 19.
3
Gene replacement therapies for inherited disorders of neurotransmission: Current progress in succinic semialdehyde dehydrogenase deficiency.
J Inherit Metab Dis. 2024 May;47(3):476-493. doi: 10.1002/jimd.12735. Epub 2024 Apr 6.
4
Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders.
Int J Mol Sci. 2024 Jan 15;25(2):1050. doi: 10.3390/ijms25021050.
5
CXCL10 and Nrf2-upregulated mesenchymal stem cells reinvigorate T lymphocytes for combating glioblastoma.
J Immunother Cancer. 2023 Dec 6;11(12):e007481. doi: 10.1136/jitc-2023-007481.
6
Nanoparticles exhibiting virus-mimic surface topology for enhanced oral delivery.
Nat Commun. 2023 Nov 24;14(1):7694. doi: 10.1038/s41467-023-43465-y.
7
rAAV2-Mediated Restoration of GALC in Neural Stem Cells from Krabbe Patient-Derived iPSCs.
Pharmaceuticals (Basel). 2023 Apr 20;16(4):624. doi: 10.3390/ph16040624.
8
Intravenous functional gene transfer throughout the brain of non-human primates using AAV.
Res Sq. 2023 Jan 13:rs.3.rs-1370972. doi: 10.21203/rs.3.rs-1370972/v1.
9
RNAi and chemogenetic reporter co-regulation in primate striatal interneurons.
Gene Ther. 2022 Feb;29(1-2):69-80. doi: 10.1038/s41434-021-00260-y. Epub 2021 May 19.
10
Gene Therapy for Neurodegenerative Diseases: Slowing Down the Ticking Clock.
Front Neurosci. 2020 Sep 18;14:580179. doi: 10.3389/fnins.2020.580179. eCollection 2020.
本文引用的文献
1
Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain.
Stereotact Funct Neurosurg. 2011;89(3):141-51. doi: 10.1159/000323544. Epub 2011 Apr 14.
2
T2 imaging in monitoring of intraparenchymal real-time convection-enhanced delivery.
Neurosurgery. 2011 Jul;69(1):154-63; discussion 163. doi: 10.1227/NEU.0b013e318217217e.
3
Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease.
Mol Ther. 2011 Jun;19(6):1048-57. doi: 10.1038/mt.2011.11. Epub 2011 Feb 22.
4
Human/nonhuman primate AC-PC ratio--considerations for translational brain measurements.
J Neurosci Methods. 2011 Mar 15;196(1):124-30. doi: 10.1016/j.jneumeth.2010.12.023. Epub 2010 Dec 24.
5
Qualitative imaging of adeno-associated virus serotype 2-human aromatic L-amino acid decarboxylase gene therapy in a phase I study for the treatment of Parkinson disease.
Neurosurgery. 2010 Nov;67(5):1377-85. doi: 10.1227/NEU.0b013e3181f53a5c.
6
Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF.
J Neurosci. 2010 Jul 14;30(28):9567-77. doi: 10.1523/JNEUROSCI.0942-10.2010.
7
Real-time MR imaging with Gadoteridol predicts distribution of transgenes after convection-enhanced delivery of AAV2 vectors.
Mol Ther. 2010 Aug;18(8):1490-5. doi: 10.1038/mt.2010.114. Epub 2010 Jun 15.
8
Magnetic resonance imaging-guided delivery of adeno-associated virus type 2 to the primate brain for the treatment of lysosomal storage disorders.
Hum Gene Ther. 2010 Sep;21(9):1093-103. doi: 10.1089/hum.2010.040.
9
Cannula placement for effective convection-enhanced delivery in the nonhuman primate thalamus and brainstem: implications for clinical delivery of therapeutics.
J Neurosurg. 2010 Aug;113(2):240-8. doi: 10.3171/2010.2.JNS091744.
10
Image-guided convection-enhanced delivery of GDNF protein into monkey putamen.
Neuroimage. 2011 Jan;54 Suppl 1:S189-95. doi: 10.1016/j.neuroimage.2010.01.023. Epub 2010 Jan 18.
Zotero 插件