Your browser doesn't support javascript.
loading
In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease.
Park, Hanseul; Oh, Jungju; Shim, Gayong; Cho, Byounggook; Chang, Yujung; Kim, Siyoung; Baek, Soonbong; Kim, Hongwon; Shin, Jeain; Choi, Hwan; Yoo, Junsang; Kim, Junyeop; Jun, Won; Lee, Minhyung; Lengner, Christopher J; Oh, Yu-Kyoung; Kim, Jongpil.
Afiliação
  • Park H; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Oh J; Department of Bioengineering, College of Engineering, Hanyang University, Seoul, Republic of Korea.
  • Shim G; College of Pharmacy, Seoul National University, Seoul, Republic of Korea.
  • Cho B; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Chang Y; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Kim S; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Baek S; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Kim H; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Shin J; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Choi H; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Yoo J; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Kim J; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea.
  • Jun W; Department of Nanobiomedical Science, Dankook University, Cheonan, Republic of Korea.
  • Lee M; Department of Bioengineering, College of Engineering, Hanyang University, Seoul, Republic of Korea.
  • Lengner CJ; Department of Biomedical Sciences, School of Veterinary Medicine and Institute for Regenerative Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Oh YK; College of Pharmacy, Seoul National University, Seoul, Republic of Korea.
  • Kim J; Department of Biomedical Engineering (BK21 plus), Dongguk University, Seoul, Republic of Korea. jpkim153@dongguk.edu.
Nat Neurosci ; 22(4): 524-528, 2019 04.
Article em En | MEDLINE | ID: mdl-30858603
ABSTRACT
In vivo gene editing in post-mitotic neurons of the adult brain may be a useful strategy for treating neurological diseases. Here, we develop CRISPR-Cas9 nanocomplexes and show they were effective in the adult mouse brain, with minimal off-target effects. Using this system to target Bace1 suppressed amyloid beta (Aß)-associated pathologies and cognitive deficits in two mouse models of Alzheimer's disease. These results broaden the potential application of CRISPR-Cas9 systems to neurodegenerative diseases.
Assuntos
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Peptídeos beta-Amiloides / Ácido Aspártico Endopeptidases / Secretases da Proteína Precursora do Amiloide / Doença de Alzheimer / Sistemas CRISPR-Cas / Edição de Genes / Neurônios Limite: Animals Idioma: En Revista: Nat Neurosci Assunto da revista: NEUROLOGIA Ano de publicação: 2019 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Peptídeos beta-Amiloides / Ácido Aspártico Endopeptidases / Secretases da Proteína Precursora do Amiloide / Doença de Alzheimer / Sistemas CRISPR-Cas / Edição de Genes / Neurônios Limite: Animals Idioma: En Revista: Nat Neurosci Assunto da revista: NEUROLOGIA Ano de publicação: 2019 Tipo de documento: Article