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Wyświetlanie 1-4 z 4
Tytuł :
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
Autorzy :
Pickar-Oliver A; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Gough V; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Bohning JD; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Liu S; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Graduate Program in Computational Biology and Bioinformatics, Duke University, Durham, NC 27708, USA.
Robinson-Hamm JN; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Daniels H; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA.
Majoros WH; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Center for Statistical Genetics and Genomics, Duke University, Durham, NC 27708, USA; Division of Integrative Genomics, Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, NC 27710, USA.
Devlin G; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA.
Asokan A; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA; Regeneration Next Initiative, Duke University, Durham, NC 27710, USA.
Gersbach CA; Department of Biomedical Engineering, Room 1427 FCIEMAS, 101 Science Drive, Box 90281, Duke University, Durham, NC 27708, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Center for Genomic and Computational Biology, Duke University, Durham, NC 27708, USA; Graduate Program in Computational Biology and Bioinformatics, Duke University, Durham, NC 27708, USA; Department of Surgery, Duke University School of Medicine, Durham, NC 27710, USA; Regeneration Next Initiative, Duke University, Durham, NC 27710, USA; Department of Cell Biology, Duke University Medical Center, Durham, NC 27710, USA. Electronic address: .
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Źródło :
Molecular therapy : the journal of the American Society of Gene Therapy [Mol Ther] 2021 Sep 10. Date of Electronic Publication: 2021 Sep 10.
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy.
Autorzy :
Kwon JB; University Program in Genetics and Genomics, Duke University Medical Center, Durham, NC 27710, USA.; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.
Ettyreddy AR; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Vankara A; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Bohning JD; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Devlin G; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.
Hauschka SD; Department of Biochemistry, University of Washington, Seattle, WA, USA.
Asokan A; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.; Regeneration Next Initiative, Duke University Medical Center, Durham, NC 27710, USA.
Gersbach CA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.; Regeneration Next Initiative, Duke University Medical Center, Durham, NC 27710, USA.
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Źródło :
Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Sep 28; Vol. 19, pp. 320-329. Date of Electronic Publication: 2020 Sep 28 (Print Publication: 2020).
Typ publikacji :
Journal Article
Czasopismo naukowe
Tytuł :
Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators.
Autorzy :
Kwon JB; University Program in Genetics and Genomics, Duke University Medical Center, Durham, NC 27710, USA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA.
Vankara A; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Ettyreddy AR; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Bohning JD; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA.
Gersbach CA; Center for Advanced Genomic Technologies, Duke University, Durham, NC 27708, USA; Department of Biomedical Engineering, Duke University, Durham, NC 27708, USA; Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA. Electronic address: .
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Źródło :
Stem cell reports [Stem Cell Reports] 2020 May 12; Vol. 14 (5), pp. 755-769. Date of Electronic Publication: 2020 Apr 23.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
Cell Lineage*
Cellular Reprogramming Techniques/*methods
Induced Pluripotent Stem Cells/*metabolism
Myoblasts/*metabolism
PAX7 Transcription Factor/*metabolism
Animals ; CRISPR-Cas Systems ; Cell Differentiation ; Cells, Cultured ; Dystrophin/genetics ; Dystrophin/metabolism ; Epigenesis, Genetic ; Female ; Gene Editing/methods ; HEK293 Cells ; Humans ; Induced Pluripotent Stem Cells/cytology ; Infant, Newborn ; Male ; Mice ; Mice, Inbred NOD ; Mice, SCID ; Muscle Development ; Myoblasts/cytology ; PAX7 Transcription Factor/genetics
Czasopismo naukowe
Tytuł :
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Autorzy :
Nelson CE; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Wu Y; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
Gemberling MP; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Oliver ML; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
Waller MA; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Bohning JD; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Robinson-Hamm JN; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Bulaklak K; Department of Biomedical Engineering, Duke University, Durham, NC, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA.
Castellanos Rivera RM; Gene Therapy Center, University of North Carolina Chapel Hill, Chapel Hill, NC, USA.
Collier JH; Department of Biomedical Engineering, Duke University, Durham, NC, USA.
Asokan A; Department of Surgery, Duke University Medical Center, Durham, NC, USA.; Department of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, USA.
Gersbach CA; Department of Biomedical Engineering, Duke University, Durham, NC, USA. .; Center for Genomic and Computational Biology, Duke University, Durham, NC, USA. .; Department of Orthopaedic Surgery, Duke University Medical Center, Durham, NC, USA. .
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Źródło :
Nature medicine [Nat Med] 2019 Mar; Vol. 25 (3), pp. 427-432. Date of Electronic Publication: 2019 Feb 18.
Typ publikacji :
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
CRISPR-Cas Systems/*genetics
Gene Editing/*methods
Muscular Dystrophy, Duchenne/*therapy
Animals ; Animals, Newborn ; CRISPR-Cas Systems/immunology ; Dependovirus ; Disease Models, Animal ; Dystrophin/genetics ; Genetic Therapy/methods ; Genetic Vectors ; Immunity, Cellular/immunology ; Immunity, Humoral/immunology ; Mice ; Mice, Inbred mdx ; Muscular Dystrophy, Duchenne/genetics
Czasopismo naukowe
    Wyświetlanie 1-4 z 4

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