共 8 条
- [1] Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors[J]. NATURE BIOTECHNOLOGY, 2020, 38 (07) : 824 - 844Anzalone, Andrew V.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAKoblan, Luke W.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USALiu, David R.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst MIT & Harvard, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
- [2] Single-dose CRISPR-Cas9 therapy extends lifespan of mice with Hutchinson-Gilford progeria syndrome[J]. NATURE MEDICINE, 2019, 25 (03) : 419 - +Beyret, Ergin论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USALiao, Hsin-Kai论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USAYamamoto, Mako论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Univ Catolica San Antonio Murcia, Murcia, Spain Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA论文数: 引用数: h-index:机构:Fu, Yunpeng论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USAErikson, Galina论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USAReddy, Pradeep论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USABelmonte, Juan Carlos Izpisua论文数: 0 引用数: 0 h-index: 0机构: Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA Salk Inst Biol Studies, 10010 N Torrey Pines Rd, La Jolla, CA 92037 USA
- [3] A targeted antisense therapeutic approach for Hutchinson-Gilford progeria syndrome[J]. NATURE MEDICINE, 2021, 27 (03) : 536 - +Erdos, Michael R.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USACabral, Wayne A.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USATavarez, Urraca L.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USACao, Kan论文数: 0 引用数: 0 h-index: 0机构: Univ Maryland, Dept Cell Biol & Mol Genet, College Pk, MD 20742 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAGvozdenovic-Jeremic, Jelena论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USANarisu, Narisu论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAZerfas, Patricia M.论文数: 0 引用数: 0 h-index: 0机构: NIH, Diagnost & Res Serv Branch, Div Vet Resources, Off Res Serv, Bldg 10, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USACrumley, Stacy论文数: 0 引用数: 0 h-index: 0机构: Sarepta Therapeut Inc, Cambridge, MA USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USABoku, Yoseph论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAHanson, Gunnar论文数: 0 引用数: 0 h-index: 0机构: Sarepta Therapeut Inc, Cambridge, MA USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAMourich, Dan V.论文数: 0 引用数: 0 h-index: 0机构: Oregon State Univ, Dept Vet Hlth Sci, Corvallis, OR 97331 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAKole, Ryszard论文数: 0 引用数: 0 h-index: 0机构: Sarepta Therapeut Inc, Cambridge, MA USA Univ N Carolina, Dept Pharmacol, Chapel Hill, NC 27515 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAEckhaus, Michael A.论文数: 0 引用数: 0 h-index: 0机构: NIH, Diagnost & Res Serv Branch, Div Vet Resources, Off Res Serv, Bldg 10, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USAGordon, Leslie B.论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Dept Pediat, Hasbro Childrens Hosp, Providence, RI 02912 USA Brown Univ, Warren Alpert Med Sch, Providence, RI 02912 USA Boston Childrens Hosp, Dept Anesthesiol Preoperat & Pain Med, Boston, MA USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USACollins, Francis S.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA NHGRI, Mol Genet Sect, Med Genom & Metab Genet Branch, NIH, Bethesda, MD 20892 USA
- [4] Impact of Farnesylation Inhibitors on Survival in Hutchinson-Gilford Progeria Syndrome[J]. CIRCULATION, 2014, 130 (01) : 27 - +Gordon, Leslie B.论文数: 0 引用数: 0 h-index: 0机构: Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USA Brown Univ, Warren Alpert Med Sch, Providence, RI 02912 USA Boston Childrens Hosp, Div Crit Care Med, Dept Anesthesia, Boston, MA USA Harvard Univ, Sch Med, Boston, MA USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USAMassaro, Joe论文数: 0 引用数: 0 h-index: 0机构: Boston Univ, Dept Math & Stat, Harvard Clin Res Inst, Boston, MA 02215 USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USAD'Agostino, Ralph B.论文数: 0 引用数: 0 h-index: 0机构: Boston Univ, Dept Math & Stat, Harvard Clin Res Inst, Boston, MA 02215 USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USACampbell, Susan E.论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Ctr Gerontol & Hlth Care Res, Providence, RI 02912 USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USABrazier, Joan论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Ctr Gerontol & Hlth Care Res, Providence, RI 02912 USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USABrown, W. Ted论文数: 0 引用数: 0 h-index: 0机构: New York State Inst Basic Res, Dept Genet, Staten Isl, NY USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USAKleinman, Monica E.论文数: 0 引用数: 0 h-index: 0机构: Boston Childrens Hosp, Div Crit Care Med, Dept Anesthesia, Boston, MA USA Harvard Univ, Sch Med, Boston, MA USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USAKieran, Mark W.论文数: 0 引用数: 0 h-index: 0机构: Harvard Univ, Sch Med, Boston, MA USA Boston Childrens Hosp, Div Pediat Oncol, Dana Farber Canc Inst, Boston, MA USA Hasbro Childrens Hosp, Dept Pediat, Providence, RI 02903 USA
- [5] In vivo base editing rescues Hutchinson-Gilford progeria syndrome in mice[J]. NATURE, 2021, 589 (7843) : 608 - +Koblan, Luke W.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAErdos, Michael R.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAWilson, Christopher论文数: 0 引用数: 0 h-index: 0机构: Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USACabral, Wayne A.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USALevy, Jonathan M.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAXiong, Zheng-Mei论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USATavarez, Urraca L.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USADavison, Lindsay M.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Div Cardiovasc Med, Nashville, TN 37235 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAGete, Yantenew G.论文数: 0 引用数: 0 h-index: 0机构: Univ Maryland, Dept Cell Biol & Mol Genet, College Pk, MD 20742 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAMao, Xiaojing论文数: 0 引用数: 0 h-index: 0机构: Univ Maryland, Dept Cell Biol & Mol Genet, College Pk, MD 20742 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USANewby, Gregory A.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USADoherty, Sean P.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Div Cardiovasc Med, Nashville, TN 37235 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USANarisu, Narisu论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USASheng, Quanhu论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Dept Biostat, Nashville, TN USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAKrilow, Chad论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USALin, Charles Y.论文数: 0 引用数: 0 h-index: 0机构: Baylor Coll Med, Dept Mol & Human Genet, Houston, TX 77030 USA Baylor Coll Med, Dept Biochem & Mol Biol, Therapeut Innovat Ctr, Houston, TX 77030 USA Kronos Bio Inc, Cambridge, MA USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USAGordon, Leslie B.论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Alpert Med Sch, Hasbro Childrens Hosp, Providence, RI 02912 USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USACao, Kan论文数: 0 引用数: 0 h-index: 0机构: Univ Maryland, Dept Cell Biol & Mol Genet, College Pk, MD 20742 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USACollins, Francis S.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, NIH, Bethesda, MD 20892 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USABrown, Jonathan D.论文数: 0 引用数: 0 h-index: 0机构: Vanderbilt Univ, Med Ctr, Div Cardiovasc Med, Nashville, TN 37235 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USALiu, David R.论文数: 0 引用数: 0 h-index: 0机构: Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA Harvard Univ, Dept Chem & Chem Biol, Cambridge, MA 02138 USA Harvard Univ, Howard Hughes Med Inst, Cambridge, MA 02138 USA Broad Inst Harvard & MIT, Merkin Inst Transformat Technol Healthcare, Cambridge, MA 02142 USA
- [6] In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates[J]. NATURE, 2021, 593 (7859) : 429 - +Musunuru, Kiran论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USA Univ Penn, Dept Med, Perelman Sch Med, Div Cardiovasc Med, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Dept Genet, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAChadwick, Alexandra C.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAMizoguchi, Taiji论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAGarcia, Sara P.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USADeNizio, Jamie E.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAReiss, Caroline W.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAWang, Kui论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAIyer, Sowmya论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USADutta, Chaitali论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAClendaniel, Victoria论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAAmaonye, Michael论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USABeach, Aaron论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USABerth, Kathleen论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USABiswas, Souvik论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USABraun, Maurine C.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAChen, Huei-Mei论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAColace, Thomas, V论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAGaney, John D.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAGangopadhyay, Soumyashree A.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAGarrity, Ryan论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAKasiewicz, Lisa N.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USALavoie, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAMadsen, James A.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAMatsumoto, Yuri论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAMazzola, Anne Marie论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USANasrullah, Yusuf S.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USANneji, Joseph论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USARen, Huilan论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USASanjeev, Athul论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAShay, Madeleine论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAStahley, Mary R.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAFan, Steven H. Y.论文数: 0 引用数: 0 h-index: 0机构: Acuitas Therapeut, Vancouver, BC, Canada Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USATam, Ying K.论文数: 0 引用数: 0 h-index: 0机构: Acuitas Therapeut, Vancouver, BC, Canada Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAGaudelli, Nicole M.论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut, Cambridge, MA USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USACiaramella, Giuseppe论文数: 0 引用数: 0 h-index: 0机构: Beam Therapeut, Cambridge, MA USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAStolz, Leslie E.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAMalyala, Padma论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USACheng, Christopher J.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USARajeev, Kallanthottathil G.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USARohde, Ellen论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USABellinger, Andrew M.论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USAKathiresan, Sekar论文数: 0 引用数: 0 h-index: 0机构: Verve Therapeut, Cambridge, MA 02139 USA Univ Penn, Perelman Sch Med, Cardiovasc Inst, Philadelphia, PA 19104 USA
- [7] Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson-Gilford progeria syndrome[J]. NATURE MEDICINE, 2021, 27 (03) : 526 - +Puttaraju, Madaiah论文数: 0 引用数: 0 h-index: 0机构: NCI, NIH, Bethesda, MD 20892 USA NCI, NIH, Bethesda, MD 20892 USAJackson, Michaela论文数: 0 引用数: 0 h-index: 0机构: Ionis Pharmaceut, Carlsbad, CA USA NCI, NIH, Bethesda, MD 20892 USAKlein, Stephanie论文数: 0 引用数: 0 h-index: 0机构: Ionis Pharmaceut, Carlsbad, CA USA NCI, NIH, Bethesda, MD 20892 USAShilo, Asaf论文数: 0 引用数: 0 h-index: 0机构: NCI, NIH, Bethesda, MD 20892 USA NCI, NIH, Bethesda, MD 20892 USABennett, C. Frank论文数: 0 引用数: 0 h-index: 0机构: Ionis Pharmaceut, Carlsbad, CA USA NCI, NIH, Bethesda, MD 20892 USAGordon, Leslie论文数: 0 引用数: 0 h-index: 0机构: Brown Univ, Dept Pediat, Hasbro Childrens Hosp, Div Genet, Providence, RI 02912 USA Brown Univ, Warren Alpert Med Sch, Providence, RI 02912 USA Boston Childrens Hosp, Dept Anesthesiol Perioperat & Pain Med, Boston, MA USA Harvard Med Sch, Boston, MA 02115 USA NCI, NIH, Bethesda, MD 20892 USARigo, Frank论文数: 0 引用数: 0 h-index: 0机构: Ionis Pharmaceut, Carlsbad, CA USA NCI, NIH, Bethesda, MD 20892 USAMisteli, Tom论文数: 0 引用数: 0 h-index: 0机构: NCI, NIH, Bethesda, MD 20892 USA NCI, NIH, Bethesda, MD 20892 USA
- [8] Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome[J]. NATURE MEDICINE, 2019, 25 (03) : 423 - +Santiago-Fernandez, Olaya论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainOsorio, Fernando G.论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainQuesada, Victor论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Ctr Invest Biomed Red Canc, Madrid, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainRodriguez, Francisco论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainBasso, Sammy论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainMaeso, Daniel论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainRolas, Loic论文数: 0 引用数: 0 h-index: 0机构: Queen Mary Univ London, Barts & London Sch Med & Dent, William Harvey Res Inst, London, England Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain论文数: 引用数: h-index:机构:Nourshargh, Sussan论文数: 0 引用数: 0 h-index: 0机构: Queen Mary Univ London, Barts & London Sch Med & Dent, William Harvey Res Inst, London, England Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainFolgueras, Alicia R.论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainFreije, Jose M. P.论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Ctr Invest Biomed Red Canc, Madrid, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, SpainLopez-Otin, Carlos论文数: 0 引用数: 0 h-index: 0机构: Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain Ctr Invest Biomed Red Canc, Madrid, Spain Univ Oviedo, Fac Med, Dept Bioquim & Biol Mol, Inst Univ Oncol Principado Asturias, Oviedo, Spain