Chimeric RNA: DNA TracrRNA Improves Homology-Directed Repair In Vitro and In Vivo .

Autor: Simone BW; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Lee HB; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Daby CL; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Ata H; Department of Clinical and Translational Sciences, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Restrepo-Castillo S; Mayo Clinic Graduate School of Biomedical Sciences, Virology and Gene Therapy Track, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Martínez-Gálvez G; Mayo Clinic Graduate School of Biomedical Sciences, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Kar B; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Gendron WAC; Mayo Clinic Graduate School of Biomedical Sciences, Virology and Gene Therapy Track, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Clark KJ; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA., Ekker SC; Department of Biochemistry and Molecular Biology, Biomedical Engineering and Physiology Track, Mayo Clinic, Rochester, Minnesota, USA.

Publikováno v: The CRISPR journal [CRISPR J] 2022 Feb; Vol. 5 (1), pp. 40-52. Date of Electronic Publication: 2021 Dec 17.

Suppression-Replacement KCNQ1 Gene Therapy for Type 1 Long QT Syndrome.

Autor: Dotzler SM; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Kim CSJ; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Gendron WAC; Department of Virology & Gene Therapy, Vector and Vaccine Engineering Laboratory (W.A.C.G., M.A.B.), Mayo Clinic, Rochester, MN., Zhou W; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Ye D; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Bos JM; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN.; Department of Cardiovascular Medicine/Division of Heart Rhythm Services, Windland Smith Rice Genetic Heart Rhythm Clinic (J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Tester DJ; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN.; Department of Cardiovascular Medicine/Division of Heart Rhythm Services, Windland Smith Rice Genetic Heart Rhythm Clinic (J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN., Barry MA; Department of Virology & Gene Therapy, Vector and Vaccine Engineering Laboratory (W.A.C.G., M.A.B.), Mayo Clinic, Rochester, MN., Ackerman MJ; Department of Molecular Pharmacology & Experimental Therapeutics, Windland Smith Rice Sudden Death Genomics Laboratory (S.M.D., C.S.J.K., W.Z., D.Y., J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN.; Department of Cardiovascular Medicine/Division of Heart Rhythm Services, Windland Smith Rice Genetic Heart Rhythm Clinic (J.M.B., D.J.T., M.J.A.), Mayo Clinic, Rochester, MN.; Department of Pediatric and Adolescent Medicine/Division of Pediatric Cardiology (M.J.A.), Mayo Clinic, Rochester, MN.

Publikováno v: Circulation [Circulation] 2021 Apr 06; Vol. 143 (14), pp. 1411-1425. Date of Electronic Publication: 2021 Jan 28.

Expanding the CRISPR Toolbox with ErCas12a in Zebrafish and Human Cells.

Autor: Wierson WA; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa., Simone BW; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., WareJoncas Z; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Mann C; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa., Welker JM; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa., Kar B; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Emch MJ; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Friedberg I; Department of Veterinary Microbiology and Preventive Medicine, Iowa State University, Ames, Iowa., Gendron WAC; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Barry MA; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Clark KJ; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Dobbs DL; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa., McGrail MA; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa., Ekker SC; Department of Biochemistry and Molecular Biology, Mayo Clinic, Rochester, Minnesota., Essner JJ; Department of Genetics, Development and Cell Biology, Iowa State University, Ames, Iowa.

Publikováno v: The CRISPR journal [CRISPR J] 2019 Dec; Vol. 2 (6), pp. 417-433. Date of Electronic Publication: 2019 Nov 19.

The double whammy of ER-retention and dominant-negative effects in numerous autosomal dominant diseases: significance in disease mechanisms and therapy.

Autor: Gariballa, Nesrin¹ (AUTHOR), Mohamed, Feda^1,2 (AUTHOR), Badawi, Sally¹ (AUTHOR), Ali, Bassam R.^1,2 (AUTHOR) bassam.ali@uaeu.ac.ae

Publikováno v: Journal of Biomedical Science. 6/27/2024, Vol. 31 Issue 1, p1-24. 24p.

Cardiomyopathy: pathogenesis and therapeutic interventions.

Autor: Huang, Shitong, Li, Jiaxin, Li, Qiuying, Wang, Qiuyu, Zhou, Xianwu, Chen, Jimei, Chen, Xuanhui, Bellou, Abdelouahab, Zhuang, Jian, Lei, Liming

Publikováno v: MedComm; Nov2024, Vol. 5 Issue 11, p1-49, 49p