A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing
Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR) can efficiently modify disease-relevant genes in somatic tissues with high efficiency. AAV vectors are a preferred delivery vehicle for tissue-directed gene therapy because of their ability to achieve sustained expres...
Main Authors: | Ang Li, Ciaran M. Lee, Ayrea E. Hurley, Kelsey E. Jarrett, Marco De Giorgi, Weiqi Lu, Karol S. Balderrama, Alexandria M. Doerfler, Harshavardhan Deshmukh, Anirban Ray, Gang Bao, William R. Lagor |
---|---|
Format: | Article |
Language: | English |
Published: |
Elsevier
2019-03-01
|
Series: | Molecular Therapy: Methods & Clinical Development |
Online Access: | http://www.sciencedirect.com/science/article/pii/S2329050118301219 |
Similar Items
-
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study
by: Alexandria M. Doerfler, et al.
Published: (2022-12-01) -
Depletion of essential isoprenoids and ER stress induction following acute liver-specific deletion of HMG-CoA reductase
by: Marco De Giorgi, et al.
Published: (2020-12-01) -
Targeting the Apoa1 locus for liver-directed gene therapy
by: Marco De Giorgi, et al.
Published: (2021-06-01) -
CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
by: Giridhar Murlidharan, et al.
Published: (2016-01-01) -
In vivo genome editing at the albumin locus to treat methylmalonic acidemia
by: Jessica L. Schneller, et al.
Published: (2021-12-01)