共 28 条
- [11] Delivery of Glucosylceramidase Beta Gene Using AAV9 Vector Therapy as a Treatment Strategy in Mouse Models of Gaucher DiseaseHUMAN GENE THERAPY, 2019, 30 (02) : 155 - 167Du, Sichen论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaOu, Huayuan论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaCui, Renjie论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaJiang, Nan论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaZhang, Meiqin论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaLi, Xiaorong论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaMa, Jing论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Childrens Hosp, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaZhang, Jin论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R ChinaMa, Duan论文数: 0 引用数: 0 h-index: 0机构: Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Inst Biomed Sci, 238,130 Dongan Rd, Shanghai 200032, Peoples R China Fudan Univ, Childrens Hosp, Shanghai, Peoples R China Fudan Univ, Shanghai Med Coll, Sch Basic Med Sci, Key Lab Metab & Mol Med,Minist Educ,Dept Biochem, Shanghai, Peoples R China
- [12] Effect of Nuclear Factor κB Inhibition on Serotype 9 Adeno-Associated Viral (AAV9) Minidystrophin Gene Transfer to the mdx MouseMOLECULAR MEDICINE, 2012, 18 (03) : 466 - 476Reay, Daniel P.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA Dept Vet Affairs Med Ctr, Neurol Serv, Pittsburgh, PA USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USANiizawa, Gabriela A.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA Dept Vet Affairs Med Ctr, Neurol Serv, Pittsburgh, PA USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USAWatchko, Jon F.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Magee Womens Res Inst, Dept Pediat, Pittsburgh, PA 15213 USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USADaood, Molly论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Magee Womens Res Inst, Dept Pediat, Pittsburgh, PA 15213 USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USAReay, Ja'Nean C.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA Seton Hill Univ, LECOM, Greensburg, PA USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USARaggi, Eugene论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USAClemens, Paula R.论文数: 0 引用数: 0 h-index: 0机构: Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA Dept Vet Affairs Med Ctr, Neurol Serv, Pittsburgh, PA USA Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15213 USA
- [13] Effect of Nuclear Factor κB Inhibition on Serotype 9 Adeno-Associated Viral (AAV9) Minidystrophin Gene Transfer to the mdx MouseMolecular Medicine, 2012, 18 : 466 - 476Daniel P Reay论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServiceGabriela A Niizawa论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServiceJon F Watchko论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServiceMolly Daood论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServiceJa’Nean C Reay论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServiceEugene Raggi论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology ServicePaula R Clemens论文数: 0 引用数: 0 h-index: 0机构: Department of Veterans Affairs Medical Center,Neurology Service
- [14] Gene therapy for tuberous sclerosis complex type 2 in a mouse model by delivery of AAV9 encoding a condensed form of tuberinSCIENCE ADVANCES, 2021, 7 (02)Cheah, Pike-See论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Univ Putra Malaysia, Fac Med & Hlth Sci, Dept Human Anat, Serdang, Malaysia Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAPrabhakar, Shilpa论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAYellen, David论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USABeauchamp, Roberta L.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAZhang, Xuan论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAKasamatsu, Shingo论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Dept Anesthesia Crit Care & Pain Med, Massachusetts Gen Hosp, Charlestown, MA USA Shriners Hosp Children, Boston, MA USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USABronson, Roderick T.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Rodent Histopathol Core Facil, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAThiele, Elizabeth A.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Herscot Ctr Tuberous Sclerosis Complex, Massachusetts Gen Hosp, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Pediat Epilepsy Program, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAKwiatkowski, David J.论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Brigham & Womens Hosp, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAStemmer-Rachamimov, Anat论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Pathol, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAGyorgy, Bence论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Dept Neurobiol, Boston, MA 02115 USA Harvard Med Sch, Howard Hughes Med Inst, Boston, MA 02115 USA Inst Mol & Clin Ophthalmol, Basel, Switzerland Univ Basel, Dept Ophthalmol, Basel, Switzerland Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USALin, King-Hwa论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Dept Genet, Boston, MA 02115 USA Univ Putra Malaysia, Fac Med & Hlth Sci, Dept Biomed Sci, Serdang, Malaysia Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAKaneki, Masao论文数: 0 引用数: 0 h-index: 0机构: Harvard Med Sch, Dept Anesthesia Crit Care & Pain Med, Massachusetts Gen Hosp, Charlestown, MA USA Shriners Hosp Children, Boston, MA USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USATannous, Bakhos A.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USARamesh, Vijaya论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Ctr Genom Med, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USAMaguire, Casey A.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USABreakefield, Xandra O.论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA Massachusetts Gen Hosp, Dept Radiol, Ctr Mol Imaging Res, Boston, MA 02114 USA Harvard Med Sch, Program Neurosci, Boston, MA 02115 USA Massachusetts Gen Hosp, Dept Neurol, Mol Neurogenet Unit, Boston, MA 02114 USA
- [15] Gene Therapy for Tuberous Sclerosis Complex Type 2 in a Mouse Model by Delivery of AAV9 Encoding a Condensed Form of TuberinMOLECULAR THERAPY, 2020, 28 (04) : 563 - 563Prabhakar, Shilpa论文数: 0 引用数: 0 h-index: 0机构: Massachusetts Gen Hosp, Neurol, Charlestown, MA USA Massachusetts Gen Hosp, Neurol, Charlestown, MA USA
- [16] AAV9/MFSD8 gene therapy is effective in preclinical models of neuronal ceroid lipofuscinosis type 7 diseaseJOURNAL OF CLINICAL INVESTIGATION, 2022, 132 (05):Chen, Xin论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USADong, Thomas论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USAHu, Yuhui论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USAShaffo, Frances C.论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USABelur, Nandkishore R.论文数: 0 引用数: 0 h-index: 0机构: Northwestern Univ, Feinberg Sch Med, Ken & Ruth Davee Dept Neurol, Chicago, IL 60611 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USAMazzulli, Joseph R.论文数: 0 引用数: 0 h-index: 0机构: Northwestern Univ, Feinberg Sch Med, Ken & Ruth Davee Dept Neurol, Chicago, IL 60611 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USAGray, Steven J.论文数: 0 引用数: 0 h-index: 0机构: Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA Univ Texas Southwestern Med Ctr Dallas, Dept Pediat, 5323 Harry Hines Blvd, Dallas, TX 75390 USA
- [17] AAV-Mediated Gene Therapy to Treat Mucopolysaccharidosis Type IVA in a New Morquio a Mouse ModelMOLECULAR THERAPY, 2020, 28 (04) : 492 - 492Sanchez, Victor论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainBertolin, Joan论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainRibera, Albert论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainSanchez, Xavier论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainMarco, Sara论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainMotas, Sandra论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainPerez, Jennifer论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainRoca, Carles论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainElias, Gemma论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainGarcia, Miquel论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainLeon, Xavier论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainHaurigot, Virginia论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainRuberte, Jesus论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Anim Hlth & Anat, Cerdanyola Del Valles, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, SpainBosch, Fatima论文数: 0 引用数: 0 h-index: 0机构: Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Cerdanyola Del valles, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Ctr Anim Biotechnol & Gene Therapy CBATEG, Cerdanyola Del valles, Spain
- [18] AAV-mediated gene therapy to treat mucopolysaccharidosis type IVA in a new mouse model of the diseaseHUMAN GENE THERAPY, 2019, 30 (11) : A172 - A172Sanchez, V.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainBertolin, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainRibera, A.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainSanchez, X.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainMarco, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainMotas, S.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainPerez, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainRoca, C.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainElias, G.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainGarcia, M.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainLeon, X.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainHaurigot, V.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainRuberte, J.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Anim Hlth & Anat, Sch Vet Med, Barcelona, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, SpainBosch, F.论文数: 0 引用数: 0 h-index: 0机构: Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain Univ Autonoma Barcelona, Biochem & Mol Biol, Barcelona, Spain Ctr Invest Biomed Red Diabet & Enfermedades Metab, Madrid, Spain Univ Autonoma Barcelona, Ctr Anim Biotechnol & Gene Therapy, Barcelona, Spain
- [19] Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.BMOLECULAR GENETICS AND METABOLISM, 2018, 123 (02) : S36 - S37Davidson, Cristin论文数: 0 引用数: 0 h-index: 0机构: Albert Einstein Coll Med, Bronx, NY 10467 USA Albert Einstein Coll Med, Bronx, NY 10467 USAGibson, Alana论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Bethesda, MD 20892 USA Albert Einstein Coll Med, Bronx, NY 10467 USAGu, Tansy论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Bethesda, MD 20892 USA Albert Einstein Coll Med, Bronx, NY 10467 USAChandler, Randy论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Bethesda, MD 20892 USA Albert Einstein Coll Med, Bronx, NY 10467 USADeverman, Benjamin论文数: 0 引用数: 0 h-index: 0机构: CALTECH, Pasadena, CA 91125 USA Albert Einstein Coll Med, Bronx, NY 10467 USAGradinaru, Viviana论文数: 0 引用数: 0 h-index: 0机构: CALTECH, Pasadena, CA 91125 USA Albert Einstein Coll Med, Bronx, NY 10467 USAVenditti, Charles论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Bethesda, MD 20892 USA Albert Einstein Coll Med, Bronx, NY 10467 USAPavan, William论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Bethesda, MD 20892 USA Albert Einstein Coll Med, Bronx, NY 10467 USA
- [20] The First Viable Mouse Model of Combined Methylmalonic Acidemia and Homocystinuria, cblC Type Displays a Sustained Response to a Single Dose of AAV Gene Therapy and is Comparable to Chronic Hydroxocobalamin AdministrationMOLECULAR THERAPY, 2020, 28 (04) : 578 - 578Koutsoukos, Stefanos A.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USASloan, Jennifer L.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAMurphy, Kelsey C.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAArnold, Madeline L.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAAchilly, Nathan P.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAElliot, Gene论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Embryon Stem Cell & Transgen Mouse Core, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAZerfas, Patricia M.论文数: 0 引用数: 0 h-index: 0机构: NIH, Off Res Serv, Bldg 10, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAHoffmann, Victoria论文数: 0 引用数: 0 h-index: 0机构: NIH, Off Res Serv, Bldg 10, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USAVenditti, Charles P.论文数: 0 引用数: 0 h-index: 0机构: NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA NHGRI, Organ Acid Res Sect, Bethesda, MD 20892 USA