High-Throughput
Animals
Capsid
/ metabolism
Cell Line
Cell Line, Tumor
Cells, Cultured
DNA, Viral
Dependovirus
/ genetics
Female
Fibroblasts
Gene Transfer Techniques
Genetic Therapy
Genetic Vectors
/ genetics
HeLa Cells
High-Throughput Nucleotide Sequencing
High-Throughput Screening Assays
/ methods
Humans
Induced Pluripotent Stem Cells
Male
Mice
Receptor, EphB2
T-Lymphocytes
Transduction, Genetic
Transgenes
AAV
adeno-associated virus
gene therapy
iPSC
next-generation sequencing
viral vectors
Journal
Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950
Informations de publication
Date de publication:
05 2020
05 2020
Historique:
pubmed:
1
2
2020
medline:
24
8
2021
entrez:
1
2
2020
Statut:
ppublish
Résumé
Adeno-associated virus (AAV) vectors are quickly becoming the vectors of choice for therapeutic gene delivery. To date, hundreds of natural isolates and bioengineered variants have been reported. While factors such as high production titer and low immunoreactivity are important to consider, the ability to deliver the genetic payload (physical transduction) and to drive high transgene expression (functional transduction) remains the most important feature when selecting AAV variants for clinical applications. Reporter expression assays are the most commonly used methods for determining vector fitness. However, such approaches are time consuming and become impractical when evaluating a large number of variants. Limited access to primary human tissues or challenging model systems further complicates vector testing. To address this problem, convenient high-throughput methods based on next-generation sequencing (NGS) are being developed. To this end, we built an AAV Testing Kit that allows inherent flexibility in regard to number and type of AAV variants included, and is compatible with
Identifiants
pubmed: 32000541
doi: 10.1089/hum.2019.264
pmc: PMC7232709
doi:
Substances chimiques
DNA, Viral
0
EPHB2 protein, human
EC 2.7.10.1
Receptor, EphB2
EC 2.7.10.1
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
575-589Subventions
Organisme : Wellcome Trust
ID : 104807/Z/14/Z
Pays : United Kingdom
Organisme : Department of Health
Pays : United Kingdom
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