Cns Gene Delivery
Cns Gene Delivery - Related Videos
13:12
Efficient Gene Delivery into Multiple CNS Territories Using In Utero Electroporation
0 Views •
Cited by 31 •
2011
In utero electroporation allows for rapid gene delivery in a spatially- and temporally-controlled manner in the developing central nervous system (CNS). Here we describe a highly adaptable in utero electroporation protocol that can be used to deliver expression constructs into multiple embryonic CNS domains, including the telencephalon, diencephalon and retina.
10:33
Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes
0 Views •
Cited by 18 •
2016
The goal of this protocol is to use cationic/anionic liposomes with a neuro-targeting peptide as a CNS delivery system to enable siRNA to cross the BBB. The optimization of a delivery system for treatments, like siRNA, would allow for more treatment options for prion and other neurodegenerative diseases.
05:00
Ex Vivo Culture of Patient Tissue & Examination of Gene Delivery
0 Views •
Cited by 4 •
2010
This article describes the culture of patient tissue slices for gene delivery studies and subsequent analysis of gene expression using IVIS bioluminescence imaging.
05:12
Subpial Gene Delivery in the Mouse Spinal Cord to Evaluate Gene Expression
0 Views •
2025
Source: Tadokoro, T., et al. Subpial Adeno-associated Virus 9 (AAV9) Vector Delivery in Adult Mice. J. Vis. Exp. (2017)This video demonstrates a subpial injection technique for gene delivery to the spinal cord in mice. The procedure involves exposing the dura mater, puncturing the pia mater, and injecting a recombinant adeno-associated virus (rAAV) carrying a fluorescent protein-expressing gene into the subpial space. Following injection and recovery, the virus facilitates gene expression in...
03:31
Laser-Assisted Lentiviral Gene Delivery: A Technique to Permeabilize Mouse Fertilized Eggs to Facilitate Lentiviral Gene Delivery
0 Views •
2025
In this video, we demonstrate the laser-assisted permeation of the protective layer of zona pellucida in mouse fertilized eggs for facilitating lentiviral gene delivery. Lentivirus enables the generation of transgenic animals with a gene of interest stably integrated into their genome.
FAQs
The delivery system must transport genetic material across or around the blood-brain barrier before neural cells can take it up. Viral and nonviral vectors provide alternative carriers for DNA, RNA, or gene-editing components. Once uptake occurs, the delivered material can alter gene expression in targeted neural cells, making cellular entry a central determinant of the resulting biological effect.
The payload determines the type of genetic change the treatment is intended to produce. DNA or RNA may support altered gene expression, while gene-editing components are used when modification of genetic information is the goal. Depending on the approach, expression may be transient or sustained, and the strategy may involve gene replacement, gene silencing, or editing.
Administration routes influence how genetic material reaches the central nervous system and where it becomes available. Intrathecal, intracerebral, and systemic delivery can produce different patterns of tissue distribution, cellular targeting, and safety. Consequently, route selection affects not only access to brain or spinal-cord tissue but also the likely extent and character of the treatment response.
A basic workflow links four decisions: choose the genetic material, select a viral or nonviral vector, determine how it will be administered, and evaluate cellular uptake and gene-expression results. The delivered DNA, RNA, or editing components must reach relevant neural cells, where expression or genetic modification can then be assessed for the intended biological outcome.
Researchers compare these routes by considering their effects on tissue distribution, cellular targeting, and safety. Intrathecal, intracerebral, and systemic administration each provide a different way to deliver material to the brain and spinal cord or to approach those tissues indirectly. The comparison helps align the administration strategy with the desired anatomical reach and experimental or therapeutic objective.
In neuroscience, these approaches support investigations of neural circuitry and disease mechanisms while also contributing to treatment development. They are relevant to neurodegenerative and genetic disorders, as well as other central nervous system conditions in which conventional drugs may not reach affected tissue effectively. Outcomes can include altered gene expression, gene replacement, silencing, or editing.