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Wyświetlanie 1-6 z 6
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 :
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
Tytuł :
Genome engineering: a new approach to gene therapy for neuromuscular disorders.
Autorzy :
Nelson CE; Department of Biomedical Engineering, 101 Science Drive, FCIEMAS Building, Box 90281, Duke University, Durham, North Carolina 27708-0281, USA.; Center for Genomic & Computational Biology, 101 Science Drive, DUMC Box 3382, Duke University, Durham, North Carolina 27708, USA.
Robinson-Hamm JN; Department of Biomedical Engineering, 101 Science Drive, FCIEMAS Building, Box 90281, Duke University, Durham, North Carolina 27708-0281, USA.; Center for Genomic & Computational Biology, 101 Science Drive, DUMC Box 3382, Duke University, Durham, North Carolina 27708, USA.
Gersbach CA; Department of Biomedical Engineering, 101 Science Drive, FCIEMAS Building, Box 90281, Duke University, Durham, North Carolina 27708-0281, USA.; Center for Genomic & Computational Biology, 101 Science Drive, DUMC Box 3382, Duke University, Durham, North Carolina 27708, USA.; Department of Orthopaedic Surgery, Duke University Medical Center, 200 Trent Drive, Durham, North Carolina 27710, USA.
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Źródło :
Nature reviews. Neurology [Nat Rev Neurol] 2017 Nov; Vol. 13 (11), pp. 647-661. Date of Electronic Publication: 2017 Sep 29.
Typ publikacji :
Journal Article; Review
MeSH Terms :
Genetic Engineering/*methods
Neuromuscular Diseases/*therapy
Animals ; CRISPR-Cas Systems ; Cell- and Tissue-Based Therapy ; Deoxyribonucleases/physiology ; Disease Models, Animal ; Forecasting ; Gene Editing/methods ; Gene Transfer Techniques ; Genetic Therapy/methods ; Genome, Human ; Humans ; Minisatellite Repeats ; Muscular Atrophy, Spinal/genetics ; Muscular Atrophy, Spinal/therapy ; Muscular Dystrophies/genetics ; Muscular Dystrophies/therapy ; Neuromuscular Diseases/genetics ; Transcription Activator-Like Effector Nucleases/physiology ; Transcription Activator-Like Effectors/physiology ; Zinc Fingers/physiology
Czasopismo naukowe
Tytuł :
Generation and comparison of CRISPR-Cas9 and Cre-mediated genetically engineered mouse models of sarcoma.
Autorzy :
Huang J; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Chen M; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.; Medical Scientist Training Program, Duke University Medical Center, Durham, North Carolina 27710, USA.
Whitley MJ; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.; Medical Scientist Training Program, Duke University Medical Center, Durham, North Carolina 27710, USA.
Kuo HC; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Xu ES; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Walens A; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Mowery YM; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Van Mater D; Division of Hematology-Oncology, Department of Pediatrics, Duke University Medical Center, Durham, North Carolina 27710, USA.
Eward WC; Department of Orthopedic Surgery, Duke University, Durham, North Carolina 27710, USA.
Cardona DM; Department of Pathology, Duke University, Durham, North Carolina 27710, USA.
Luo L; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Ma Y; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Lopez OM; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Nelson CE; Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708, USA.; Duke Center for Genomic and Computational Biology, Duke University, Durham, North Carolina 27708, USA.
Robinson-Hamm JN; Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708, USA.; Duke Center for Genomic and Computational Biology, Duke University, Durham, North Carolina 27708, USA.
Reddy A; Duke Center for Genomic and Computational Biology, Duke University, Durham, North Carolina 27708, USA.
Dave SS; Duke Center for Genomic and Computational Biology, Duke University, Durham, North Carolina 27708, USA.; Department of Medicine, Duke University Medical Center, Durham, North Carolina 27710, USA.
Gersbach CA; Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708, USA.; Duke Center for Genomic and Computational Biology, Duke University, Durham, North Carolina 27708, USA.
Dodd RD; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.
Kirsch DG; Department of Radiation Oncology, Duke University Medical Center, Durham, North Carolina 27710, USA.; Department of Pharmacology and Cancer Biology, Duke University Medical Center, Durham, North Carolina 27710, USA.
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Źródło :
Nature communications [Nat Commun] 2017 Jul 10; Vol. 8, pp. 15999. Date of Electronic Publication: 2017 Jul 10.
Typ publikacji :
Comparative Study; Evaluation Study; Journal Article; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, Non-P.H.S.
MeSH Terms :
CRISPR-Cas Systems*
Integrases*
Sarcoma, Experimental/*genetics
Animals ; Electroporation ; Gene Editing/methods ; Male ; Mice ; Mice, Nude ; Mutation ; NIH 3T3 Cells ; Neurilemmoma/genetics ; Neurilemmoma/pathology ; Sarcoma, Experimental/pathology
Czasopismo naukowe
Tytuł :
Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy.
Autorzy :
Robinson-Hamm JN; Department of Biomedical Engineering, Duke University, Room 1427, Fitzpatrick CIEMAS, 101 Science Drive, Box 90281, Durham, NC, 27708-0281, USA.; Center for Genomic and Computational Biology, Duke University, Durham, NC, 27708, USA.
Gersbach CA; Department of Biomedical Engineering, Duke University, Room 1427, Fitzpatrick CIEMAS, 101 Science Drive, Box 90281, Durham, NC, 27708-0281, USA. .; Center for Genomic and Computational Biology, Duke University, Durham, NC, 27708, USA. .; Department of Orthopaedic Surgery, Duke University Medical Center, Durham, NC, 27710, USA. .
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Źródło :
Human genetics [Hum Genet] 2016 Sep; Vol. 135 (9), pp. 1029-40. Date of Electronic Publication: 2016 Aug 20.
Typ publikacji :
Journal Article; Review; Research Support, U.S. Gov't, Non-P.H.S.; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms :
Genetic Therapy*
Dystrophin/*genetics
Muscular Dystrophy, Duchenne/*therapy
Animals ; Exons ; Humans ; Mice ; Muscular Dystrophy, Duchenne/genetics
Czasopismo naukowe
Tytuł :
Transgenic overexpression of ribonucleotide reductase improves cardiac performance.
Autorzy :
Nowakowski SG; Department of Bioengineering, University of Washington, Seattle, WA 98195, USA.
Kolwicz SC
Korte FS
Luo Z
Robinson-Hamm JN
Page JL
Brozovich F
Weiss RS
Tian R
Murry CE
Regnier M
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Źródło :
Proceedings of the National Academy of Sciences of the United States of America [Proc Natl Acad Sci U S A] 2013 Apr 09; Vol. 110 (15), pp. 6187-92. Date of Electronic Publication: 2013 Mar 25.
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 :
Gene Expression Regulation*
Myocardium/*metabolism
Ribonucleotide Reductases/*metabolism
Adenosine Triphosphate/metabolism ; Animals ; Echocardiography ; Magnetic Resonance Spectroscopy ; Mice ; Mice, Transgenic ; Myocytes, Cardiac/cytology ; Phenotype ; Ribonucleotide Reductases/genetics ; Sarcomeres/metabolism ; Systole ; Transgenes
Czasopismo naukowe
    Wyświetlanie 1-6 z 6

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