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- [21] Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIAMOLECULAR GENETICS AND METABOLISM, 2020, 129 (02) : S90 - S90Kinsella, Jane Louise论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandChurch, Heather论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandOgden, Wendy论文数: 0 引用数: 0 h-index: 0机构: Manchester Fdn Trust, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandJones, Ceri论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandBuckland, Karen论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandRivat, Christine论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandThrasher, Adrian论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandSnell, Katie论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandLeon-Rico, Diego论文数: 0 引用数: 0 h-index: 0机构: UCL Great Ormond St Inst Child Hlth, London, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandVaz, Fred论文数: 0 引用数: 0 h-index: 0机构: Amsterdam Med Ctr, Amsterdam, Netherlands Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandWijburg, Frits论文数: 0 引用数: 0 h-index: 0机构: Amsterdam Med Ctr, Amsterdam, Netherlands Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandJones, Simon论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandBigger, Brian论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, EnglandWynn, Robert论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Royal Manchester Childrens Hosp, Manchester, Lancs, England
- [22] Pre-clinical safety and efficacy evaluation of haematopoietic stem cell gene therapy for MPSIIIAHUMAN GENE THERAPY, 2018, 29 (12) : A127 - A127Ellison, S. M.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandWood, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandArmant, M.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Boston, MA 02115 USA Univ Manchester, Manchester, Lancs, EnglandFarzeneh, F.论文数: 0 引用数: 0 h-index: 0机构: Kings Coll London, London, England Univ Manchester, Manchester, Lancs, EnglandJones, S.论文数: 0 引用数: 0 h-index: 0机构: Manchester Childrens Hopsital, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandWynn, R.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandGaspar, B.论文数: 0 引用数: 0 h-index: 0机构: UCL Inst Child Hlth, London, England Univ Manchester, Manchester, Lancs, EnglandBiffi, A.论文数: 0 引用数: 0 h-index: 0机构: Boston Childrens Hosp, Boston, MA USA Univ Manchester, Manchester, Lancs, EnglandBigger, B.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, England
- [23] Neurological Correction of Mucopolysaccharidosis IIIB Mice by Haematopoietic Stem Cell Gene TherapyMOLECULAR THERAPY, 2016, 24 : S146 - S146Ellison, Stuart M.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England论文数: 引用数: h-index:机构:Fil, Daniel论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandMc Dermott, John论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandSenthivel, Nisha论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England论文数: 引用数: h-index:机构:Wilkinson, Fiona论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandJones, Simon论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Ctr Genom Med, Manchester M27 1HA, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandWynn, Rob论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Paediat Haematol, Manchester M27 1HA, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandKan, Shih-hsin论文数: 0 引用数: 0 h-index: 0机构: Harbor UCLA Med Ctr, Los Angeles Biomed Res Inst, Torrance, CA 90509 USA Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandDickson, Patti论文数: 0 引用数: 0 h-index: 0机构: Harbor UCLA Med Ctr, Los Angeles Biomed Res Inst, Torrance, CA 90509 USA Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, EnglandBigger, Brian论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England Univ Manchester, Stem Cell & Neurotherapies, Manchester, Lancs, England
- [24] Clinical Trial Update: Ex-vivo autologous haematopoietic stem cell gene therapy in MPSIIIAHUMAN GENE THERAPY, 2021, 32 (19-20) : A139 - A140Jones, S. A.论文数: 0 引用数: 0 h-index: 0机构: St Marys Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandKinsella, J. L.论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandPotter, J.论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandThrasher, A. J.论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Inst Child Hlth, London, England Univ Manchester, Manchester, Lancs, EnglandBooth, C.论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Inst Child Hlth, London, England Univ Manchester, Manchester, Lancs, EnglandBuckland, K. F.论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Inst Child Hlth, London, England Univ Manchester, Manchester, Lancs, EnglandIzotova, N.论文数: 0 引用数: 0 h-index: 0机构: Great Ormond St Inst Child Hlth, London, England Univ Manchester, Manchester, Lancs, EnglandRust, S.论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandWeisberg, D.论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandChurch, H. J.论文数: 0 引用数: 0 h-index: 0机构: St Marys Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandTylee, K. L.论文数: 0 引用数: 0 h-index: 0机构: St Marys Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandBrammeier, K. L.论文数: 0 引用数: 0 h-index: 0机构: St Marys Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandJones, C.论文数: 0 引用数: 0 h-index: 0机构: St Marys Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandLee, H.论文数: 0 引用数: 0 h-index: 0机构: Manchester Royal Infirm, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandFord, L.论文数: 0 引用数: 0 h-index: 0机构: Manchester Royal Infirm, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandSadegholnejat, A.论文数: 0 引用数: 0 h-index: 0机构: Manchester Royal Infirm, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandHolley, R. J.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, England论文数: 引用数: h-index:机构:Bigger, B. W.论文数: 0 引用数: 0 h-index: 0机构: Univ Manchester, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, EnglandWynn, R. F.论文数: 0 引用数: 0 h-index: 0机构: Royal Manchester Childrens Hosp, Manchester, Lancs, England Univ Manchester, Manchester, Lancs, England
- [25] .-Globin Lentiviral Vectors for Hematopoietic Stem Cell Gene Therapy of α-ThalassemiaBLOOD, 2023, 142Segura, Eva论文数: 0 引用数: 0 h-index: 0机构: UCLA, Los Angeles, CA USA UCLA, Los Angeles, CA USATam, Kevin论文数: 0 引用数: 0 h-index: 0机构: UCLA, Los Angeles, CA USA UCLA, Los Angeles, CA USAFernandez, Beatriz Campo论文数: 0 引用数: 0 h-index: 0机构: UCLA, Los Angeles, CA USAMacKenzie, Tippi C.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Francisco, Eli & Edythe Broad Ctr Regenerat Med, San Francisco, CA USA Univ Calif San Francisco, San Francisco, CA USA UCLA, Los Angeles, CA USAHollis, Roger论文数: 0 引用数: 0 h-index: 0机构: UCLA, Los Angeles, CA USAKohn, Donald B.论文数: 0 引用数: 0 h-index: 0机构: UCLA, Los Angeles, CA USA UCLA, Los Angeles, CA USA
- [26] Lentiviral Haematopoietic Stem Cell Gene Therapy for Late Juvenile Metachromatic LeukodystrophyMOLECULAR THERAPY, 2024, 32 (04) : 887 - 887Calbi, Valeria论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyGallo, Vera论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyDe Mattia, Fabiola论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyZambon, Alberto论文数: 0 引用数: 0 h-index: 0机构: IRCCS San Raffaele Sci Inst, Neurol & Neurophysiol Unit, Milan, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyRecupero, Salvatore论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyFratini, Elena Sophia论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyClerici, Alessandra论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy论文数: 引用数: h-index:机构:论文数: 引用数: h-index:机构:Brooks, Jean论文数: 0 引用数: 0 h-index: 0机构: Orchard Therapeut Europe Ltd, London, England San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyRichardson, Alan论文数: 0 引用数: 0 h-index: 0机构: Orchard Therapeut Europe Ltd, London, England San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyCambpell, Laura论文数: 0 引用数: 0 h-index: 0机构: Orchard Therapeut Europe Ltd, London, England San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyBernardo, Maria Ester论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyAiuti, Alessandro论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy Univ Vita Salute San Raffaele, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, ItalyFumagalli, Francesca论文数: 0 引用数: 0 h-index: 0机构: San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy San Raffaele Telethon Inst Gene Therapy SR TIGET, Milan, Italy
- [27] Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIICMOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 13 - 13Badell-Grau, Rafael论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USAPakravesh, Kasra论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USASivakumar, Anusha论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USALosay, Pauline论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USADuong, Kalvin论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USATran, Christine论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USAPithia, Rushil论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USAKhare, Veenita论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USAThai, Kevin论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USACherqui, Stephanie论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, San Diego, CA USA
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- [29] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIICMOLECULAR THERAPY, 2023, 31 (04) : 325 - 326Badell-Grau, Rafael A.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USASivakumar, Anusha论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USAKhare, Veenita论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, San Diego, CA USA Univ Calif San Diego, La Jolla, CA USADuong, Kalvin论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USALosay, Pauline论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USAPakravesh, Kasra论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USATran, Christine论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USAPithia, Rushil论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USALengsfield, Mira论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USAThai, Kevin论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USASharma, Jay论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USAChen, Rola论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USARainaldi, Joseph N.论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USACherqui, Stephanie论文数: 0 引用数: 0 h-index: 0机构: Univ Calif San Diego, La Jolla, CA USA Univ Calif San Diego, La Jolla, CA USA
- [30] Hematopoietic Stem Cell Gene Therapy for Mucopolysaccharidosis Type IIICMOLECULAR THERAPY, 2024, 32 (04) : 420 - 420Badell-Grau, Rafael A.论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAPakravesh, Kasra论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAChen, Rola论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAThai, Eric论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USASon, Frankie论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USADuong, Kalvin论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USALosay, Pauline论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAPithia, Rushil论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USASivakumar, Anusha论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAKhare, Veenita论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USACorl, Alexis N.论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USATran, Christine论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USAEsko, Jefferey论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USACherqui, Stephanie论文数: 0 引用数: 0 h-index: 0机构: UCSD, La Jolla, CA USA UCSD, La Jolla, CA USA