Year: 2020
Type: article
Abstract: (Mol Ther. 26, 1818–1827; July 5, 2018) In this article, the Animal Experiments section, subheading “AAV Injections,” originally read “All animal procedures were performed in accordance with protocols approved by the Institutional Animal Care and Use Committee (IACUC) of the University of California, San Diego and adhered to the ARVO Statement for the Use of Animals in Ophthalmic and Vision Research.” The online text has been updated to read “All animal work was pursuant to protocols ultimately approved by the IACUC of the University of California San Diego. However, there were deviations from the approved protocol of Dr. Zhang related to retinitis pigmentosa on mouse neonates (P7) before those deviations were incorporated in an amended protocol, which was promptly approved by the University of California San Diego IACUC.” In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene RegulationMoreno et al.Molecular TherapyApril 24, 2018In BriefWe developed a modular adeno-associated viral vector platform for CRISPR-Cas9 delivery to enable targeted in situ gene regulation, demonstrating robust transcriptional repression and activation of genes in cell culture and mice. This approach could potentially enable disease targeting in a genomically scarless and reversible manner. Full-Text PDF Open Archive
Source: Molecular Therapy
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: hybrid
Estimated APC paid: $3,500
OpenAlex ID: W4229922551