Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa

Targeted transcriptional modulation in the central nervous system (CNS) can be achieved by adeno-associated virus (AAV) delivery of CRISPR activation (CRISPRa) and interference (CRISPRi) transgenes. To enable AAV packaging, we constructed minimal CRISPRa and CRISPRi transgenes by fusing catalyticall...

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Detalles Bibliográficos
Autores principales: Lau, Cia-Hin, Ho, Jonathan Weng-Thim, Lo, Pik Kwan, Tin, Chung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6526230/
https://www.ncbi.nlm.nih.gov/pubmed/31108320
http://dx.doi.org/10.1016/j.omtn.2019.04.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6526230/
https://www.ncbi.nlm.nih.gov/pubmed/31108320
http://dx.doi.org/10.1016/j.omtn.2019.04.015

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