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A universal gene correction approach for FKRP-associated dystroglycanopathies to enable autologous cell therapy.
Dhoke, Neha R; Kim, Hyunkee; Selvaraj, Sridhar; Azzag, Karim; Zhou, Haowen; Oliveira, Nelio A J; Tungtur, Sudheer; Ortiz-Cordero, Carolina; Kiley, James; Lu, Qi Long; Bang, Anne G; Perlingeiro, Rita C R.
Affiliation
  • Dhoke NR; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Kim H; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Selvaraj S; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Azzag K; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Zhou H; Conrad Prebys Center for Chemical Genomics, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, USA.
  • Oliveira NAJ; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Tungtur S; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Ortiz-Cordero C; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Kiley J; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
  • Lu QL; McColl-Lockwood Laboratory for Muscular Dystrophy Research, Cannon Research Center, Carolinas Medical Center, Atrium Health, Charlotte, NC, USA.
  • Bang AG; Conrad Prebys Center for Chemical Genomics, Sanford Burnham Prebys Medical Discovery Institute, La Jolla, CA, USA.
  • Perlingeiro RCR; Lillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA. Electronic address: perli032@umn.edu.
Cell Rep ; 36(2): 109360, 2021 07 13.
Article in En | MEDLINE | ID: mdl-34260922
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pentosyltransferases / Genetic Therapy / Dystroglycans / Cell- and Tissue-Based Therapy / Muscular Dystrophies Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Child, preschool / Humans / Male Language: En Journal: Cell Rep Year: 2021 Document type: Article
Fulltext
Print
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PubMed Links
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Pentosyltransferases / Genetic Therapy / Dystroglycans / Cell- and Tissue-Based Therapy / Muscular Dystrophies Type of study: Prognostic_studies / Risk_factors_studies Limits: Animals / Child, preschool / Humans / Male Language: En Journal: Cell Rep Year: 2021 Document type: Article