Impaired LEF1 Activation Accelerates iPSC-Derived Keratinocytes Differentiation in Hutchinson-Gilford Progeria Syndrome.

Autor: Mao X; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA., Xiong ZM; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA.; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA., Xue H; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA., Brown MA; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA., Gete YG; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA., Yu R; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA., Sun L; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA.; Key Laboratory of Diagnosis and Treatment of Aging and Physical-Chemical Injury Diseases of Zhejiang Province, The First Affiliated Hospital, School of Medicine, Zhejiang University, Hangzhou 310027, China., Cao K; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD 20817, USA.

Publikováno v: International journal of molecular sciences [Int J Mol Sci] 2022 May 14; Vol. 23 (10). Date of Electronic Publication: 2022 May 14.

In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice.

Autor: Koblan LW; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Erdos MR; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Wilson C; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Cabral WA; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Levy JM; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Xiong ZM; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Tavarez UL; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Davison LM; Division of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA., Gete YG; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA., Mao X; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA., Newby GA; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA., Doherty SP; Division of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA., Narisu N; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Sheng Q; Department of Biostatistics, Vanderbilt University Medical Center, Nashville, TN, USA., Krilow C; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Lin CY; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA.; Therapeutic Innovation Center, Department of Biochemistry and Molecular Biology, Baylor College of Medicine, Houston, TX, USA.; Kronos, Bio Inc., Cambridge, MA, USA., Gordon LB; Hasbro Children's Hospital, Alpert Medical School of Brown University, Providence, RI, USA.; Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Cao K; Department of Cell Biology and Molecular Genetics, University of Maryland, College Park, MD, USA., Collins FS; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA. francis.collins3@nih.gov., Brown JD; Division of Cardiovascular Medicine, Vanderbilt University Medical Center, Nashville, TN, USA. jonathan.d.brown@vumc.org., Liu DR; Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.; Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.; Howard Hughes Medical Institute, Harvard University, Cambridge, MA, USA. drliu@fas.harvard.edu.

Publikováno v: Nature [Nature] 2021 Jan; Vol. 589 (7843), pp. 608-614. Date of Electronic Publication: 2021 Jan 06.