共 37 条
- [31] Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I TrialHUMAN GENE THERAPY, 2008, 19 (10) : 979 - 990Hauswirth, William W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAAleman, Tomas S.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAKaushal, Shalesh论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USACideciyan, Artur V.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USASchwartz, Sharon B.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAWang, Lili论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Pathol & Lab Med, Gene Therapy Program, Philadelphia, PA 19104 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAConlon, Thomas J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USABoye, Sanford L.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAFlotte, Terence R.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Worcester, MA 01655 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAByrne, Barry J.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USAJacobson, Samuel G.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Scheie Eye Inst, Dept Ophthalmol, Philadelphia, PA 19104 USA
- [32] Phase I Gene Therapy Trial in Israeli Patients with Leber Congenital Amaurosis Caused by a Founder RPE65 Mutation: Safety and Efficacy Update with Up to Two Years of Follow-upINVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2013, 54 (15)Banin, Eyal论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelObolensky, Alexey论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelHemo, Yitzhak论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelMarks-Ohana, Devora论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelSela, Malka论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelYamin, Esther论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelHauswirth, William论文数: 0 引用数: 0 h-index: 0机构: Univ Florida Gainsville, Ophthalmol, Gainesville, FL USA Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelJacobson, Samuel论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Philadelphia, PA 19104 USA Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, IsraelSharon, Dror论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, Ophthalmol, Jerusalem, Israel
- [33] Phase I gene therapy trial in Israeli patients with Leber Congenital Amaurosis caused by a founder RPE65 mutation: safety and efficacy update with up to three years of follow-upHUMAN GENE THERAPY, 2013, 24 (03) : A6 - A7Banin, E.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelObolensky, A.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelHemo, Y.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelMarks-Ohana, D.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelSela, M.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelYamin, E.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelHauswirth, W. W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Gainesville, FL 32611 USA Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelJacobson, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Scheie Eye Inst, Philadelphia, PA 19104 USA Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, IsraelSharon, D.论文数: 0 引用数: 0 h-index: 0机构: Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel Hadassah Hebrew Univ, Med Ctr, IL-91120 Jerusalem, Israel
- [34] Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal DystrophyOPHTHALMOLOGY, 2016, 123 (07) : 1606 - 1620Weleber, Richard G.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAPennesi, Mark E.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAWilson, David J.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAKaushal, Shalesh论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Med Ctr, Dept Ophthalmol, Worcester, MA USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAErker, Laura R.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAJensen, Lauren论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAMcBride, Maureen T.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAFlotte, Terence R.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Med Ctr, Off Dean, Worcester, MA USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAHumphries, Margaret论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Med Ctr, Dept Ophthalmol, Worcester, MA USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USACalcedo, Roberto论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Gene Therapy Program, Philadelphia, PA 19104 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAHauswirth, William W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Sch Med, Dept Ophthalmol, Gainesville, FL USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAChulay, Jeffrey D.论文数: 0 引用数: 0 h-index: 0机构: Appl Genet Technol Corp, Alachua, FL USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USAStout, J. Timothy论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA Oregon Hlth & Sci Univ, Casey Eye Inst, Dept Ophthalmol, Portland, OR 97201 USA
- [35] Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosisHUMAN MOLECULAR GENETICS, 2011, 20 (23) : 4569 - 4581Ku, Cristy A.论文数: 0 引用数: 0 h-index: 0机构: W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USA W Virginia Univ, Dept Ophthalmol, Morgantown, WV 26505 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USAChiodo, Vince A.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USABoye, Sanford L.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USAGoldberg, Andrew F. X.论文数: 0 引用数: 0 h-index: 0机构: Oakland Univ, Eye Res Inst, Rochester, MI 48309 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USALi, Tiansen论文数: 0 引用数: 0 h-index: 0机构: NEI, N NRL, Bethesda, MD 20892 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USAHauswirth, William W.论文数: 0 引用数: 0 h-index: 0机构: Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USARamamurthy, Visvanathan论文数: 0 引用数: 0 h-index: 0机构: W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USA W Virginia Univ, Dept Ophthalmol, Morgantown, WV 26505 USA W Virginia Univ, Dept Biochem, Robert C Byrd Hlth Sci Ctr, Morgantown, WV 26505 USA W Virginia Univ, Inst Eye, Ctr Neurosci, Morgantown, WV 26505 USA
- [36] Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis (vol 29, pg 2456, 2021)MOLECULAR THERAPY, 2021, 29 (12) : 3528 - 3528Aguirre, Gustavo D.论文数: 0 引用数: 0 h-index: 0V. Cideciyan, Artur论文数: 0 引用数: 0 h-index: 0Dufour, Valerie L.论文数: 0 引用数: 0 h-index: 0Ripolles-Garcia, Ana论文数: 0 引用数: 0 h-index: 0Sudharsan, Raghavi论文数: 0 引用数: 0 h-index: 0Swider, Malgorzata论文数: 0 引用数: 0 h-index: 0Nikonov, Roman论文数: 0 引用数: 0 h-index: 0Iwabe, Simone论文数: 0 引用数: 0 h-index: 0Boye, Sanford L.论文数: 0 引用数: 0 h-index: 0Hauswirth, William W.论文数: 0 引用数: 0 h-index: 0Jacobson, Samuel G.论文数: 0 引用数: 0 h-index: 0Beltran, William A.论文数: 0 引用数: 0 h-index: 0
- [37] Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trialLANCET, 2016, 388 (10045): : 661 - 672Bennett, Jean论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAWellman, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAMarshall, Kathleen A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAMcCague, Sarah论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAAshtari, Manzar论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Radiol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USADiStefano-Pappas, Julie论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Westat Biostat & Data Management Core, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAElci, Okan U.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Westat Biostat & Data Management Core, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAChung, Daniel C.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USASun, Junwei论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAWright, J. Fraser论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USACross, Dominique R.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAAravand, Puya论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USACyckowski, Laura L.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Dept Radiol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USABennicelli, Jeannette L.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAMingozzi, Federico论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Genethon, Immunol & Liver Gene Therapy, Evry, France Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAAuricchio, Alberto论文数: 0 引用数: 0 h-index: 0机构: Telethon Inst Genet & Med, Naples, Italy Univ Naples Federico II, Med Genet, Dept Pediat, Naples, Italy Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAPierce, Eric A.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Ophthalmol, Philadelphia, PA 19104 USA Harvard Med Sch, Ocular Genom Inst, Massachusetts Eye & Ear Infirm, Boston, MA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USARuggiero, Jason论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA论文数: 引用数: h-index:机构:Simonelli, Francesca论文数: 0 引用数: 0 h-index: 0机构: Univ Naples 2, Eye Clin, Multidisciplinary Dept Med Surg & Dent Sci, Naples, Italy Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAHigh, Katherine A.论文数: 0 引用数: 0 h-index: 0机构: Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Spark Therapeut, Philadelphia, PA USA Howard Hughes Med Inst, Philadelphia, PA USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USAMaguire, Albert M.论文数: 0 引用数: 0 h-index: 0机构: Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA Univ Penn, FM Kirby Ctr Mol Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Dept Ophthalmol, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Ctr Cellular & Mol Therapeut, Philadelphia, PA 19104 USA Childrens Hosp Philadelphia, Dept Ophthalmol, Philadelphia, PA 19104 USA Univ Penn, Ctr Adv Retinal & Ocular Therapeut, Philadelphia, PA 19104 USA