AAV gene therapy for Tay-Sachs disease.

Autor: Flotte TR; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA. Terry.Flotte@umassmed.edu.; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA. Terry.Flotte@umassmed.edu., Cataltepe O; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Department of Neurosurgery, UMass Chan Medical School, Worcester, MA, USA., Puri A; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Batista AR; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA., Moser R; Department of Neurosurgery, UMass Chan Medical School, Worcester, MA, USA., McKenna-Yasek D; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA., Douthwright C; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA., Gernoux G; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA., Blackwood M; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA., Mueller C; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA., Tai PWL; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA., Jiang X; Department of Medicine and Cardiovascular Division, Washington University School of Medicine, St. Louis, MO, USA., Bateman S; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA., Spanakis SG; Departments of Anesthesiology and Perioperative Medicine, UMass Chan Medical School, Worcester, MA, USA., Parzych J; Departments of Anesthesiology and Perioperative Medicine, UMass Chan Medical School, Worcester, MA, USA., Keeler AM; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA., Abayazeed A; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Rohatgi S; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Gibson L; Department of Pediatrics, UMass Chan Medical School, Worcester, MA, USA.; Department of Internal Medicine, UMass Chan Medical School, Worcester, MA, USA., Finberg R; Department of Internal Medicine, UMass Chan Medical School, Worcester, MA, USA., Barton BA; Population and Quantitative Health Sciences, UMass Chan Medical School, Worcester, MA, USA., Vardar Z; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Shazeeb MS; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Gounis M; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Tifft CJ; National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA., Eichler FS; Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Boston, MA, USA., Brown RH Jr; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA., Martin DR; Scott-Ritchey Research Center, Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Gray-Edwards HL; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA.; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA., Sena-Esteves M; Horae Gene Therapy Center and The Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA. Miguel.esteves@umassmed.edu.; Department of Neurology, UMass Chan Medical School, Worcester, MA, USA. Miguel.esteves@umassmed.edu.

Publikováno v: Nature medicine [Nat Med] 2022 Feb; Vol. 28 (2), pp. 251-259. Date of Electronic Publication: 2022 Feb 10.

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Fatty acid binding protein 4 is expressed in distinct endothelial and non-endothelial cell populations in glioblastoma.

Autor: Cataltepe, O.¹, Arikan, M. C.¹, Ghelfi, E.², Karaaslan, C.², Ozsurekci, Y.², Dresser, K.³, Li, Y.⁴, Smith, T. W.³, Cataltepe, S.²

Publikováno v: Neuropathology & Applied Neurobiology. Aug2012, Vol. 38 Issue 5, p400-410. 11p. 3 Color Photographs, 2 Charts.

Clinical and Translational Landscape of Viral Gene Therapies.

Autor: Yudaeva, Alexandra¹ (AUTHOR) aleksa.yudaeva@gmail.com, Kostyusheva, Anastasiya¹ (AUTHOR) kostyusheva_ap@mail.ru, Kachanov, Artyom¹ (AUTHOR) kachanov.av99@gmail.com, Brezgin, Sergey^1,2 (AUTHOR) aparodi.sechenovuniversity@gmail.com, Ponomareva, Natalia^1,2,3 (AUTHOR) ponomareva.n.i13@yandex.ru, Parodi, Alessandro² (AUTHOR), Pokrovsky, Vadim S.^2,4,5 (AUTHOR), Lukashev, Alexander^1,6 (AUTHOR) alexander_lukashev@hotmail.com, Chulanov, Vladimir⁷ (AUTHOR) vladimir@chulanov.ru, Kostyushev, Dmitry^1,2,8 (AUTHOR) dkostushev@gmail.com

Publikováno v: Cells (2073-4409). Nov2024, Vol. 13 Issue 22, p1916. 47p.

Case report: cranial angiosarcoma with multiple hemorrhagic brain metastasis in a child.

Autor: Lee V; University of Massachusetts Medical School, Worcester, MA, USA., Gessler D; University of Massachusetts Medical School, Worcester, MA, USA.; Horae Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA.; Department of Neurosurgery, University of Minnesota, Minneapolis, MN, USA., Cataltepe O; Department of Neurosurgery, University of Massachusetts Memorial Medical Center, 55 N Lake Ave., Worcester, MA, 01655, USA. Oguz.Cataltepe@umassmemorial.org.

Publikováno v: Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery [Childs Nerv Syst] 2020 Sep; Vol. 36 (9), pp. 2103-2107. Date of Electronic Publication: 2020 Mar 10.