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- [21] Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGRNature Medicine, 2020, 26 : 354 - 359Jasmina Cehajic-Kapetanovic论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesKanmin Xue论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesCristina Martinez-Fernandez de la Camara论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAnika Nanda论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAlexandra Davies论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesLaura J. Wood论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAnna Paola Salvetti论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesM. Dominik Fischer论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesJames W. Aylward论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAlun R. Barnard论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesJasleen K. Jolly论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesEdmond Luo论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesBrandon J. Lujan论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesTuyen Ong论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAniz Girach论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesGraeme C. M. Black论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesNinel Z. Gregori论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesJanet L. Davis论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesPotyra R. Rosa论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesAndrew J. Lotery论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesByron L. Lam论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesPaulo E. Stanga论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical NeurosciencesRobert E. MacLaren论文数: 0 引用数: 0 h-index: 0机构: University of Oxford,Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences
- [22] Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGRNATURE MEDICINE, 2020, 26 (03) : 354 - +Cehajic-Kapetanovic, Jasmina论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandXue, Kanmin论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandMartinez-Fernandez de la Camara, Cristina论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandNanda, Anika论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandDavies, Alexandra论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandWood, Laura J.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandSalvetti, Anna Paola论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandFischer, M. Dominik论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandAylward, James W.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandBarnard, Alun R.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandJolly, Jasleen K.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandLuo, Edmond论文数: 0 引用数: 0 h-index: 0机构: Nightstar Therapeut Ltd, London, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandLujan, Brandon J.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Casey Eye Inst, Portland, OR 97201 USA Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandOng, Tuyen论文数: 0 引用数: 0 h-index: 0机构: Nightstar Therapeut Ltd, London, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandGirach, Aniz论文数: 0 引用数: 0 h-index: 0机构: Nightstar Therapeut Ltd, London, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandBlack, Graeme C. M.论文数: 0 引用数: 0 h-index: 0机构: Manchester Univ Hosp NHS Fdn Trust, St Marys Hosp, Manchester Ctr Genom Med, Manchester, Lancs, England Manchester Univ Hosp NHS Fdn Trust, Manchester Acad Hlth Sci Ctr, Manchester Royal Eye Hosp, Manchester Vis Regenerat Lab, Manchester, Lancs, England Univ Manchester, Fac Biol Med & Hlth, Sch Biol Sci, Manchester, Lancs, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandGregori, Ninel Z.论文数: 0 引用数: 0 h-index: 0机构: Bascom Palmer Eye Inst, Miami, FL 33136 USA Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandDavis, Janet L.论文数: 0 引用数: 0 h-index: 0机构: Bascom Palmer Eye Inst, Miami, FL 33136 USA Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandRosa, Potyra R.论文数: 0 引用数: 0 h-index: 0机构: Bascom Palmer Eye Inst, Miami, FL 33136 USA Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandLotery, Andrew J.论文数: 0 引用数: 0 h-index: 0机构: Univ Southampton, Fac Med, Clin Neurosci Res Grp, Clin & Expt Sci, Southampton, Hants, England Univ Hosp Southampton NHS Fdn Trust, Southampton, Hants, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandLam, Byron L.论文数: 0 引用数: 0 h-index: 0机构: Bascom Palmer Eye Inst, Miami, FL 33136 USA Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandStanga, Paulo E.论文数: 0 引用数: 0 h-index: 0机构: Manchester Univ Hosp NHS Fdn Trust, St Marys Hosp, Manchester Ctr Genom Med, Manchester, Lancs, England Manchester Univ Hosp NHS Fdn Trust, Manchester Acad Hlth Sci Ctr, Manchester Royal Eye Hosp, Manchester Vis Regenerat Lab, Manchester, Lancs, England Univ Manchester, Fac Biol Med & Hlth, Sch Biol Sci, Manchester, Lancs, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, EnglandMacLaren, Robert E.论文数: 0 引用数: 0 h-index: 0机构: Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England Oxford Univ Hosp NHS Fdn Trust, Oxford Eye Hosp, Oxford, England Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England
- [23] First-In-Human Gene Therapy for Tay-Sachs Disease: Report of Two Infants Treated on an Expanded Access Clinical Trial of rAAVrh8-HexA/HexB (AXO-AAV-GM2)MOLECULAR THERAPY, 2020, 28 (04) : 553 - 554Flotte, Terence R.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USACataltepe, Oguz论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Neurosurg, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAPuri, Ajit论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Radiol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USABatista, Rita论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAMoser, Richard论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Neurosurg, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAMckenna-Yasek, Diane论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Neurol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USADouthwright, Catherine论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Neurol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAGernoux, Gwladys论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USABlackwood, Meghan论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAMueller, Christian论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USABateman, Scot论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Pediat, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USASpanakis, Spiro论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Anesthesiol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAParzych, Julia论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Anesthesiol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAKeeler, Allison M.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAAbayazeed, Aly论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Radiol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAGibson, Laura论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Infect Dis, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAFinberg, Robert论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Med, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAGounis, Matthew论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Radiol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USABrown, Robert H.论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Neurol, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USAGray-Edwards, Heather论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USASena-Esteves, Miguel论文数: 0 引用数: 0 h-index: 0机构: Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA Univ Massachusetts, Sch Med, Gene Therapy Ctr, Worcester, MA USA
- [24] Preliminary safety data of a phase 1 first in-human clinical trial support the use of high dose intrathecal AAV9/CLN7 for the treatment of patients with CLN7 diseaseMOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S65 - S65Kayani, Saima论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAChen, Xin论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAMessahel, Souad论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAEdgar, Veronica论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAIannaccone, Susan论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAGray, Steven论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAMinassian, Berge论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USAGreenberg, Benjamin论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dallas, TX 75390 USA
- [25] Single-dose AAV9-CLN6 gene transfer stabilizes motor and language function in CLN6-type Batten disease: Interim results from the first clinical gene therapy trialMOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S46 - S47de los Reyes, Emily论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Nationwide Childrens Hosp, Columbus, OH USA论文数: 引用数: h-index:机构:Lehwald, Lenora论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Nationwide Childrens Hosp, Columbus, OH USAAlbright, Charles论文数: 0 引用数: 0 h-index: 0机构: Nationwide Childrens Hosp, Columbus, OH USA Nationwide Childrens Hosp, Columbus, OH USACastelli, Jeff论文数: 0 引用数: 0 h-index: 0机构: Amicus Therapeut Inc, Cranbury, NJ USA Nationwide Childrens Hosp, Columbus, OH USAJiang, Hai论文数: 0 引用数: 0 h-index: 0机构: Amicus Therapeut Inc, Cranbury, NJ USA Nationwide Childrens Hosp, Columbus, OH USAReha, Allen论文数: 0 引用数: 0 h-index: 0机构: Amicus Therapeut Inc, Cranbury, NJ USA Nationwide Childrens Hosp, Columbus, OH USABarth, Jay论文数: 0 引用数: 0 h-index: 0机构: Amicus Therapeut Inc, Cranbury, NJ USA Nationwide Childrens Hosp, Columbus, OH USA
- [26] First-in-human trial of PCSK9 gene editing therapy for lowering cholesterol: a new frontier in cardiovascular pharmacotherapy? News from AHAEUROPEAN HEART JOURNAL-CARDIOVASCULAR PHARMACOTHERAPY, 2024, 10 (02) : 87 - 88Lewis, Basil S.论文数: 0 引用数: 0 h-index: 0机构: Technion Israel Inst Technol, Lady Davis Carmel Med Ctr, IL-3436212 Haifa, Israel Technion Israel Inst Technol, Ruth & Bruce Rappaport Sch Med, IL-3436212 Haifa, Israel Technion Israel Inst Technol, Lady Davis Carmel Med Ctr, IL-3436212 Haifa, Israel
- [27] AAV8 Gene Therapy as a Potential Treatment in Adults with Late-Onset OTC Deficiency: Results from a Phase 1/2 Clinical TrialMOLECULAR THERAPY, 2020, 28 (04) : 222 - 222Tan, Wen-Hann论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USAGeberhiwot, Tarekegn论文数: 0 引用数: 0 h-index: 0机构: Univ Birmingham, Birmingham, W Midlands, England Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA论文数: 引用数: h-index:机构:Aldamiz-Echevarria, Luis论文数: 0 引用数: 0 h-index: 0机构: Cruces Univ Hosp, Biocruces Bizkaia Hlth Res Inst, Baracaldo, Spain Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USADiaz, George A.论文数: 0 引用数: 0 h-index: 0机构: Icahn Sch Med Mt Sinai, New York, NY 10029 USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USAHarding, Cary O.论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Portland, OR 97201 USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USAKhan, Aneal论文数: 0 引用数: 0 h-index: 0机构: Univ Calgary, Calgary, AB, Canada Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USALee, Connie论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Gene Therapy, Cambridge, MA USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USAPuga, Ana Cristina论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Gene Therapy, Cambridge, MA USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USACrombez, Eric论文数: 0 引用数: 0 h-index: 0机构: Ultragenyx Gene Therapy, Cambridge, MA USA Harvard Med Sch, Boston Childrens Hosp, Boston, MA 02115 USA
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- [29] Low Seroprevalence Of Neutralizing Antibodies To Adeno-associated Virus Serotype 9 (AAV9) In Preparation For Mypeak-1, The First-in-Human Study Of TN-201, An Investigational AAV9-mediated Gene Therapy For Individuals With MYBPC3-associated Hypertrophic Cardiomyopathy (HCM)JOURNAL OF CARDIAC FAILURE, 2025, 31 (01)Haroldson, Jeff论文数: 0 引用数: 0 h-index: 0机构: Cleveland Clin Fdn, South San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USAMassera, Daniele论文数: 0 引用数: 0 h-index: 0机构: NYU Langone, New York, NY USA Cleveland Clin Fdn, South San Francisco, CA USAWang, Heng论文数: 0 引用数: 0 h-index: 0机构: DDC Clin Special Needs Children, Middlefield, OH USA Cleveland Clin Fdn, South San Francisco, CA USAWong, Tmothy c.论文数: 0 引用数: 0 h-index: 0机构: Cleveland Clin Fdn, South San Francisco, CA USAWever-pinzon, Omar论文数: 0 引用数: 0 h-index: 0机构: Univ Utah, Hlth Sci Ctr, Salt Lake City, UT USA Cleveland Clin Fdn, South San Francisco, CA USALakdawala, Neil k论文数: 0 引用数: 0 h-index: 0机构: Cleveland Clin Fdn, South San Francisco, CA USAGiudicessi, John论文数: 0 引用数: 0 h-index: 0机构: Mayo Clin, Rochester, MN USA Cleveland Clin Fdn, South San Francisco, CA USAAbraham, Theodore论文数: 0 引用数: 0 h-index: 0机构: UCSF, San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USANagueh, Sherif论文数: 0 引用数: 0 h-index: 0机构: Houston Methodist, Houston, TX USA Cleveland Clin Fdn, South San Francisco, CA USARader, Florian论文数: 0 引用数: 0 h-index: 0机构: Cedars Sinai Med Ctr, West Hollywood, CA USA Cleveland Clin Fdn, South San Francisco, CA USAMasr, Ahmad论文数: 0 引用数: 0 h-index: 0机构: Oregon Hlth & Sci Univ, Sch Med, Portland, OR USA Cleveland Clin Fdn, South San Francisco, CA USATurer, Aslan论文数: 0 引用数: 0 h-index: 0机构: UT Southwestern, Dallas, TX USA Cleveland Clin Fdn, South San Francisco, CA USAMushonga, Pfumo论文数: 0 引用数: 0 h-index: 0机构: BioAgilytix, Durham, NC USA Cleveland Clin Fdn, South San Francisco, CA USAButala-flores, Elizabeth论文数: 0 引用数: 0 h-index: 0机构: BioAgilytix, Durham, NC USA Cleveland Clin Fdn, South San Francisco, CA USAHarrison, Will论文数: 0 引用数: 0 h-index: 0机构: Tenaya Therapeut, South San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USAWang, Whedy论文数: 0 引用数: 0 h-index: 0机构: Tenaya Therapeut, South San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USARobertson, Laura论文数: 0 引用数: 0 h-index: 0机构: Tenaya Therapeut, South San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USAArgast, Gretchen论文数: 0 引用数: 0 h-index: 0机构: Tenaya Therapeut, South San Francisco, CA USA Cleveland Clin Fdn, South San Francisco, CA USA
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