scholarly journals The Influence of Murine Genetic Background in Adeno-Associated Virus Transduction of the Mouse Brain

2019 ◽  
Vol 30 (4) ◽  
pp. 169-181 ◽  
Author(s):  
Ting He ◽  
Michelle S. Itano ◽  
Lauriel F. Earley ◽  
Nikita E. Hall ◽  
Natallia Riddick ◽  
...  
Keyword(s):  
Mouse Brain ◽  
Genetic Background ◽  
Adeno Associated Virus

scholarly journals Complete Correction of Enzymatic Deficiency and Neurochemistry in the GM1-gangliosidosis Mouse Brain by Neonatal Adeno-associated Virus–mediated Gene Delivery

2007 ◽  
Vol 15 (1) ◽  
pp. 30-37 ◽  
Author(s):  
M L D Broekman ◽  
R C Baek ◽  
L A Comer ◽  
J L Fernandez ◽  
T N Seyfried ◽  
...  
Keyword(s):  
Gene Delivery ◽  
Mouse Brain ◽  
Gm1 Gangliosidosis ◽  
Adeno Associated Virus

scholarly journals Local Knockdown of ERK2 in the Adult Mouse Brain Via Adeno-Associated Virus-Mediated RNA Interference

2008 ◽  
Vol 41 (3) ◽  
pp. 308-308
Author(s):  
Barbara Di Benedetto ◽  
Benedikt Wefers ◽  
Wolfgang Wurst ◽  
Ralf Kühn
Keyword(s):  
Rna Interference ◽  
Mouse Brain ◽  
Adult Mouse ◽  
Adult Mouse Brain ◽  
Adeno Associated Virus

scholarly journals Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain

2011 ◽  
Vol 20 (22) ◽  
pp. 4371-4380 ◽  
Author(s):  
Timothy J. Sargeant ◽  
Susan Wang ◽  
Josephine Bradley ◽  
Nicolas J.C. Smith ◽  
Animesh A. Raha ◽  
...  
Keyword(s):  
Mouse Brain ◽  
Neuronal Loss ◽  
Adeno Associated Virus

scholarly journals High levels of AAV vector integration into CRISPR-induced DNA breaks

2019 ◽  
Vol 10 (1) ◽  
Author(s):  
Killian S. Hanlon ◽  
Benjamin P. Kleinstiver ◽  
Sara P. Garcia ◽  
Mikołaj P. Zaborowski ◽  
Adrienn Volak ◽  
...  
Keyword(s):  
Mouse Brain ◽  
Cultured Cells ◽  
Double Strand Breaks ◽  
Dna Breaks ◽  
Adeno Associated Virus ◽  
Strand Breaks ◽  
Vector Integration ◽  
Genome Wide ◽  
Vector Genome

Abstract Adeno-associated virus (AAV) vectors have shown promising results in preclinical models, but the genomic consequences of transduction with AAV vectors encoding CRISPR-Cas nucleases is still being examined. In this study, we observe high levels of AAV integration (up to 47%) into Cas9-induced double-strand breaks (DSBs) in therapeutically relevant genes in cultured murine neurons, mouse brain, muscle and cochlea. Genome-wide AAV mapping in mouse brain shows no overall increase of AAV integration except at the CRISPR/Cas9 target site. To allow detailed characterization of integration events we engineer a miniature AAV encoding a 465 bp lambda bacteriophage DNA (AAV-λ465), enabling sequencing of the entire integrated vector genome. The integration profile of AAV-465λ in cultured cells display both full-length and fragmented AAV genomes at Cas9 on-target sites. Our data indicate that AAV integration should be recognized as a common outcome for applications that utilize AAV for genome editing.

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