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8 Οκτ 2008 · The basic process of ex-vivo gene therapy. There are also non-viral methods for gene therapy ( 1 – 4 ). Although low levels of transfection and gene expression mean that non-viral vectors are still less efficient, they present certain advantages over viral methods with simple large scale production and low host immunogenicity being just two.
- PMC Free Article
MATERIALS AND METHODS. Animals. Normal C3H/HeOuJ mice were...
- PubMed
In summary, this is the first report of ex vivo...
- PMC Free Article
27 Ιουλ 2016 · This study demonstrates correction of disease in a pig model of metabolic liver disease by ex vivo gene therapy. To date, ex vivo gene therapy has only been successful in small animal models. We conclude that further exploration of ex vivo hepatocyte genetic correction is warranted for clinical use.
23 Αυγ 2023 · To fully harness the potential of AAV-gene therapies to cure a multitude of currently unmet clinical needs, appropriate non-clinical animal models must be selected for each novel AAV-gene...
29 Ιαν 2018 · Here the authors transduce mouse embryos with CRISPR-Cas9 components using rAAVs in explant culture or in vivo to produce gene-edited animals.
Gene editing approaches to restore the DMD open reading frame and rescue functional dystrophin expression have recently been demonstrated in large animal models of DMD. Using CRISPR-Cas9 to target the DMD exon 51 splice acceptor site, Amoasii et al. 63 co-delivered AAV9-Cas9 and AAV9-gRNA to deltaE50-MD dogs.
3 Νοε 2021 · ex vivo gene therapy. Introduction. Animal models are indispensable elements of translational research that have contributed enormously to our enhanced understanding of disease pathophysiology, transplantation biology, and gene therapy.
In summary, this is the first report of ex vivo hepatocyte-directed gene repair using CRISPR/Cas9 to demonstrate curative therapy in an animal model of liver disease. Keywords: CRISPR/Cas9; gene therapy; hepatocytes; hereditary tyrosinemia type 1; metabolic liver disease.
