共 50 条
- [31] Characterization and AAV-Mediated Correction of Xeroderma Pigmentosum-Cockayne Syndrome (XP/CS)MOLECULAR THERAPY, 2015, 23 : S73 - S73Rizzo, Skylar A.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USADonate, Amy L.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USAGosiker, Bennett J.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USASantostefano, Katherine E.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USATerada, Naohiro论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USAAlkuraya, Fowzan S.论文数: 0 引用数: 0 h-index: 0机构: Alfaisal Univ, Riyadh, Saudi Arabia UF, Gainesville, FL USAKang, Peter B.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USAPacak, Christina A.论文数: 0 引用数: 0 h-index: 0机构: UF, Gainesville, FL USA UF, Gainesville, FL USA
- [32] Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVIIBLOOD, 2016, 127 (05) : 565 - 571Marcos-Contreras, Oscar A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USASmith, Shannon M.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USABellinger, Dwight A.论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, Dept Pathol & Lab Med, Chapel Hill, NC USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USARaymer, Robin A.论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, Dept Pathol & Lab Med, Chapel Hill, NC USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAMerricks, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, Dept Pathol & Lab Med, Chapel Hill, NC USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAFaella, Armida论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAPavani, Giulia论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAZhou, Shangzhen论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USANichols, Timothy C.论文数: 0 引用数: 0 h-index: 0机构: Univ N Carolina, Dept Pathol & Lab Med, Chapel Hill, NC USA Univ N Carolina, Dept Med, Chapel Hill, NC USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAHigh, Katherine A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Howard Hughes Med Inst, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USAMargaritis, Paris论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Raymond G Perelman Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Perelman Sch Med, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Pediat, Philadelphia, PA 19104 USA
- [33] AAV-Mediated Phenotypic Correction of the CNS Manifestations of a Mouse Model of Mucopolysaccharidosis Type IMOLECULAR THERAPY, 2014, 22 : S146 - S147Van de Graaf, Benjamin G.论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USAWang, Marlene论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USAKwaa, Abena论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USAHackett, Neil R.论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USACrystal, Ronald G.论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USASondhi, Dolan论文数: 0 引用数: 0 h-index: 0机构: Weill Cornell Med Coll, New York, NY USA Weill Cornell Med Coll, New York, NY USA
- [34] Correction of PTEN mutations in glioblastoma cell lines via AAV-mediated gene editingPLOS ONE, 2017, 12 (05):Hill, Victoria K.论文数: 0 引用数: 0 h-index: 0机构: Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USA Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USAKim, Jung-Sik论文数: 0 引用数: 0 h-index: 0机构: Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USA Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USAJames, C. David论文数: 0 引用数: 0 h-index: 0机构: Northwestern Univ, Feinberg Sch Med, Dept Neurol Surg, Chicago, IL 60611 USA Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USAWaldman, Todd论文数: 0 引用数: 0 h-index: 0机构: Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USA Georgetown Univ, Sch Med, Lombardi Comprehens Canc Ctr, Washington, DC 20057 USA
- [35] Author Correction: Targeted exon skipping with AAV-mediated split adenine base editorsCell Discovery, 5Jackson Winter论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringAlan Luu论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringMichael Gapinske论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringSony Manandhar论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringShraddha Shirguppe论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringWendy S. Woods论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringJun S. Song论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of BioengineeringPablo Perez-Pinera论文数: 0 引用数: 0 h-index: 0机构: University of Illinois at Urbana-Champaign,Department of Bioengineering
- [36] CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosanpj Regenerative Medicine, 1Beau R Webber论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationMark J Osborn论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationAmber N McElroy论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationKirk Twaroski论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationCara-lin Lonetree论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationAnthony P DeFeo论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationLily Xia论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationCindy Eide论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationChristopher J Lees论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationRon T McElmurry论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationMegan J Riddle论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationChong Jai Kim论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationDharmeshkumar D Patel论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationBruce R Blazar论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow TransplantationJakub Tolar论文数: 0 引用数: 0 h-index: 0机构: University of Minnesota,Department of Pediatrics, Division of Blood and Marrow Transplantation
- [37] CRISPR/Cas9-based genetic correction for recessive dystrophic epidermolysis bullosaNPJ REGENERATIVE MEDICINE, 2016, 1Webber, Beau R.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAOsborn, Mark J.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Stem Cell Inst, Minneapolis, MN 55455 USA Univ Minnesota, Ctr Genome Engn, Minneapolis, MN USA Asan Minnesota Inst Innovating Transplantat, Seoul, South Korea Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAMcElroy, Amber N.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USATwaroski, Kirk论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USALonetree, Cara-lin论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USADefeo, Anthony P.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAXia, Lily论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAEide, Cindy论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USALees, Christopher J.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAMcElmurry, Ron T.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USARiddle, Megan J.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAKim, Chong Jai论文数: 0 引用数: 0 h-index: 0机构: Asan Minnesota Inst Innovating Transplantat, Seoul, South Korea Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USAPatel, Dharmeshkumar D.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USABlazar, Bruce R.论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Stem Cell Inst, Minneapolis, MN 55455 USA Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USATolar, Jakub论文数: 0 引用数: 0 h-index: 0机构: Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA Univ Minnesota, Stem Cell Inst, Minneapolis, MN 55455 USA Asan Minnesota Inst Innovating Transplantat, Seoul, South Korea Univ Minnesota, Dept Pediat, Div Blood & Marrow Transplantat, Minneapolis, MN 55455 USA
- [38] AAV-mediated gene transfer for treatment of hemophiliaCURRENT GENE THERAPY, 2005, 5 (03) : 349 - 360Wang, LX论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Abramson Res Ctr 302B, Philadelphia, PA 19104 USAHerzog, RW论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Abramson Res Ctr 302B, Philadelphia, PA 19104 USA
- [39] AAV-Mediated Antibody Delivery for Hereditary AngioedemaMOLECULAR THERAPY, 2020, 28 (04) : 94 - 94Bruder, Joseph Thomas论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAWang, Xu论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAMcDougald, Devin S.论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAGlenn, Justin D.论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USALee, Wei-Hua论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAQiao, Chunping论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAYang, Lin论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAKarumuthil-Melethil, Subha论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAMercer, Andrew论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USAFiscella, Michele论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USALiu, Ye论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USADanos, Oliver论文数: 0 引用数: 0 h-index: 0机构: Regenxbio Inc, Res & Early Dev, Rockville, MD USA Regenxbio Inc, Res & Early Dev, Rockville, MD USA
- [40] Response: AAV-mediated gene transfer to liverBLOOD, 2003, 101 (08) : 3338 - 3339High, KA论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USAHerzog, R论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USAArruda, V论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Div Hematol, Philadelphia, PA 19104 USA