Use of CRISPR/Cas9-mediated disruption of CNS cell type genes to profile transduction of AAV by neonatal intracerebroventricular delivery in mice

Adeno-associated virus (AAV) transduction efficiency and tropism are conventionally determined by high expression of a fluorescent reporter gene. Emerging data has suggested that such conventional methods may underestimate AAV transduction for cells in which reporter expression from AAV vectors is u...

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Veröffentlicht in:	Gene therapy 2021-08, Vol.28 (7-8), p.456-468
Hauptverfasser:	Torregrosa, Tess, Lehman, Sydney, Hana, Sam, Marsh, Galina, Xu, Shanqin, Koszka, Kathryn, Mastrangelo, Nicole, McCampbell, Alexander, Henderson, Christopher E., Lo, Shih-Ching
Format:	Artikel
Sprache:	eng
Schlagworte:	14/32 38/1 38/44 38/71 42/41 631/61/201 631/61/2300/1850 Animals Astrocytes Biomedical and Life Sciences Biomedicine Blood-brain barrier Care and treatment Cell Biology Cellular signal transduction Central Nervous System CRISPR CRISPR-Cas Systems Dependovirus - genetics Dependoviruses Expression vectors Gene disruption Gene Expression Gene Therapy Gene Transfer Techniques Genes Genetic aspects Genetic vectors Genetic Vectors - genetics Glial fibrillary acidic protein Human Genetics Infants (Newborn) Methods Mice Nanotechnology Neonates Neurons Oligodendrocyte-myelin glycoprotein Oligodendrocytes Reporter gene Spinal cord Transduction Transduction, Genetic Tropism
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