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المؤلفون: Johnson AK; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., McCurdy VJ; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Gray-Edwards HL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Maguire AS; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Cochran JN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Gross AL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Skinner HE; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Randle AN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Shirley JL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Brunson BL; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Bradbury AM; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Leroy SG; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA., Hwang M; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Rockwell HE; Biology Department, Boston College, Chestnut Hill, Massachusetts, USA., Cox NR; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Baker HJ; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA., Seyfried TN; Biology Department, Boston College, Chestnut Hill, Massachusetts, USA., Sena-Esteves M; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, Massachusetts, USA., Martin DR; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.; Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, Alabama, USA.

المصدر: Annals of neurology [Ann Neurol] 2023 Nov; Vol. 94 (5), pp. 969-986. Date of Electronic Publication: 2023 Aug 16.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Wiley-Liss Country of Publication: United States NLM ID: 7707449 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1531-8249 (Electronic) Linking ISSN: 03645134 NLM ISO Abbreviation: Ann Neurol Subsets: MEDLINE

مواضيع طبية MeSH: Sandhoff Disease*/genetics , Sandhoff Disease*/therapy , Sandhoff Disease*/veterinary , Gangliosidoses, GM2*, Child ; Animals ; Cats ; Humans ; Multiple Organ Failure/therapy ; Genetic Vectors ; Central Nervous System/pathology ; Genetic Therapy

المؤلفون: Dadali T; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Diers AR; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Kazerounian S; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Muthuswamy SK; Department of Medicine, Cancer Research Institute, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA., Awate P; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Ng R; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Mogre S; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Spencer C; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Krumova K; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Rockwell HE; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., McDaniel J; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Chen EY; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Gao F; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Diedrich KT; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Vemulapalli V; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Rodrigues LO; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Akmaev VR; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Thapa K; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Hidalgo M; Department of Medicine, Cancer Research Institute, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA., Bose A; Department of Medicine, Cancer Research Institute, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA., Vishnudas VK; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Moser AJ; Department of Medicine, Cancer Research Institute, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, 02215, USA., Granger E; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Kiebish MA; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Gesta S; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Narain NR; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA., Sarangarajan R; BERG LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA, 01710, USA. Rangaprasad.Sarangarajan@BergHealth.com.

المصدر: Scientific reports [Sci Rep] 2021 Mar 11; Vol. 11 (1), pp. 5749. Date of Electronic Publication: 2021 Mar 11.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 101563288 Publication Model: Electronic Cited Medium: Internet ISSN: 2045-2322 (Electronic) Linking ISSN: 20452322 NLM ISO Abbreviation: Sci Rep Subsets: MEDLINE

مواضيع طبية MeSH: Apoptosis*, Mitochondria/*metabolism , Pancreatic Neoplasms/*pathology , Reactive Oxygen Species/*metabolism , Ubiquinone/*analogs & derivatives, Animals ; Cell Line, Tumor ; Cell Proliferation ; Cell Respiration ; Cell Survival ; Electron Transport Complex II/metabolism ; Glycerol-3-Phosphate Dehydrogenase (NAD+) ; Humans ; Membrane Potential, Mitochondrial ; Mice, Nude ; Organoids/pathology ; Oxidative Stress ; Oxygen Consumption ; Pancreatic Neoplasms/metabolism ; Substrate Specificity ; Ubiquinone/metabolism ; Mice

المؤلفون: Haraszti RA; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: rharaszti@gmail.com., Miller R; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA; Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Dubuke ML; Mass Spectrometry Facility, Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School, Shrewsbury, MA, USA., Rockwell HE; BERG LLC, Framingham, MA, USA., Coles AH; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Sapp E; Mass General Institute for Neurodegenerative Disease, Boston, MA, USA., Didiot MC; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Echeverria D; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Stoppato M; MassBiologics, Boston, MA, USA., Sere YY; MassBiologics, Boston, MA, USA., Leszyk J; Mass Spectrometry Facility, Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School, Shrewsbury, MA, USA., Alterman JF; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Godinho BMDC; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., Hassler MR; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA., McDaniel J; BERG LLC, Framingham, MA, USA., Narain NR; BERG LLC, Framingham, MA, USA., Wollacott R; MassBiologics, Boston, MA, USA., Wang Y; MassBiologics, Boston, MA, USA., Shaffer SA; Mass Spectrometry Facility, Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School, Shrewsbury, MA, USA., Kiebish MA; BERG LLC, Framingham, MA, USA., DiFiglia M; Mass General Institute for Neurodegenerative Disease, Boston, MA, USA., Aronin N; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA; Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: neil.aronin@umassmed.edu., Khvorova A; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, USA. Electronic address: anastasia.khvorova@umassmed.edu.

المصدر: IScience [iScience] 2019 Jun 28; Vol. 16, pp. 230-241. Date of Electronic Publication: 2019 May 27.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Cell Press Country of Publication: United States NLM ID: 101724038 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2589-0042 (Electronic) Linking ISSN: 25890042 NLM ISO Abbreviation: iScience Subsets: PubMed not MEDLINE

المؤلفون: Gao F; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., McDaniel J; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Chen EY; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Rockwell HE; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Nguyen C; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Lynes MD; Section on Integrative Physiology and Metabolism, Joslin Diabetes Center, Harvard Medical School, Boston, MA 02215, USA., Tseng YH; Section on Integrative Physiology and Metabolism, Joslin Diabetes Center, Harvard Medical School, Boston, MA 02215, USA.; Harvard Stem Cell Institute, Harvard University, Cambridge, MA 02138, USA., Sarangarajan R; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Narain NR; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA., Kiebish MA; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701, USA.

المصدر: Lipids [Lipids] 2018 Jan; Vol. 53 (1), pp. 133-142.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Wiley Subscription Services, Inc Country of Publication: United States NLM ID: 0060450 Publication Model: Print Cited Medium: Internet ISSN: 1558-9307 (Electronic) Linking ISSN: 00244201 NLM ISO Abbreviation: Lipids Subsets: MEDLINE

مواضيع طبية MeSH: Metabolomics* , Spectrometry, Mass, Electrospray Ionization*, Cardiolipins/*genetics , Lipids/*genetics, Cardiolipins/chemistry ; Cardiolipins/classification ; Humans ; Lipids/chemistry ; Mitochondrial Membranes/chemistry ; Mitochondrial Membranes/metabolism

المؤلفون: Gao F; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., McDaniel J; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Chen EY; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Rockwell HE; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Drolet J; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Vishnudas VK; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Tolstikov V; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Sarangarajan R; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Narain NR; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA., Kiebish MA; BERG, LLC, 500 Old Connecticut Path, Bldg B, 3rd Floor, Framingham, MA 01701 USA.

المصدر: Nutrition & metabolism [Nutr Metab (Lond)] 2017 Mar 20; Vol. 14, pp. 28. Date of Electronic Publication: 2017 Mar 20 (Print Publication: 2017).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: BioMed Central Country of Publication: England NLM ID: 101231644 Publication Model: eCollection Cited Medium: Print ISSN: 1743-7075 (Print) Linking ISSN: 17437075 NLM ISO Abbreviation: Nutr Metab (Lond) Subsets: PubMed not MEDLINE

المؤلفون: Haraszti RA; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Didiot MC; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, USA., Sapp E; MassGeneral Institute for Neurodegenerative Disease, Charlestown, MA, USA., Leszyk J; Proteomics and Mass Spectrometry Facility, University of Massachusetts Medical School, Worcester, MA, USA., Shaffer SA; Biochemistry and Molecular Pharmacology, University of Massachusetts Medical School, Worcester, MA, USA., Rockwell HE; Berg LLC, Framingham, MA, USA., Gao F; Berg LLC, Framingham, MA, USA., Narain NR; Berg LLC, Framingham, MA, USA., DiFiglia M; MassGeneral Institute for Neurodegenerative Disease, Charlestown, MA, USA., Kiebish MA; Berg LLC, Framingham, MA, USA., Aronin N; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.; Department of Medicine, University of Massachusetts Medical School, Worcester, MA, USA; Neil.aronin@umassmed.edu., Khvorova A; RNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, MA, USA.; Program in Molecular Medicine, University of Massachusetts Medical School, Worcester, MA, USA; Anastasia.khvorova@umassmed.edu.

المصدر: Journal of extracellular vesicles [J Extracell Vesicles] 2016 Nov 17; Vol. 5, pp. 32570. Date of Electronic Publication: 2016 Nov 17 (Print Publication: 2016).

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Wiley Country of Publication: United States NLM ID: 101610479 Publication Model: eCollection Cited Medium: Print ISSN: 2001-3078 (Print) Linking ISSN: 20013078 NLM ISO Abbreviation: J Extracell Vesicles Subsets: PubMed not MEDLINE

المؤلفون: Rockwell HE; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., Gao F; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., Chen EY; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., McDaniel J; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., Sarangarajan R; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., Narain NR; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA., Kiebish MA; BERG, LLC, 500 Old Connecticut Path, Bldg B (3rd Floor), Framingham, MA, 01701, USA. Michael.Kiebish@BergHealth.com.

المصدر: Lipids [Lipids] 2016 Jul; Vol. 51 (7), pp. 875-86. Date of Electronic Publication: 2016 Apr 01.

نوع المنشور: Journal Article

بيانات الدورية: Publisher: Wiley Subscription Services, Inc Country of Publication: United States NLM ID: 0060450 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1558-9307 (Electronic) Linking ISSN: 00244201 NLM ISO Abbreviation: Lipids Subsets: MEDLINE

مواضيع طبية MeSH: Chromatography, Liquid/*methods , Lipids/*urine , Tandem Mass Spectrometry/*methods, Female ; High-Throughput Screening Assays ; Humans ; Male ; Reproducibility of Results

المؤلفون: Rockwell HE; Boston College Biology Department, Chestnut Hill, MA, USA., McCurdy VJ; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, AL, USA., Eaton SC; Boston College Biology Department, Chestnut Hill, MA, USA., Wilson DU; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, AL, USA., Johnson AK; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, AL, USA., Randle AN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA., Bradbury AM; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, AL, USA., Gray-Edwards HL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA., Baker HJ; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Pathobiology, College of Veterinary Medicine, Auburn University, AL, USA., Hudson JA; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, AL, USA., Cox NR; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Pathobiology, College of Veterinary Medicine, Auburn University, AL, USA., Sena-Esteves M; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA., Seyfried TN; Boston College Biology Department, Chestnut Hill, MA, USA., Martin DR; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, AL, USA Department of Anatomy, Physiology & Pharmacology, College of Veterinary Medicine, Auburn University, AL, USA martidr@auburn.edu.

المصدر: ASN neuro [ASN Neuro] 2015 Apr 13; Vol. 7 (2). Date of Electronic Publication: 2015 Apr 13 (Print Publication: 2015).

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Sage Country of Publication: United States NLM ID: 101507115 Publication Model: Electronic-Print Cited Medium: Internet ISSN: 1759-0914 (Electronic) Linking ISSN: 17590914 NLM ISO Abbreviation: ASN Neuro Subsets: MEDLINE

مواضيع طبية MeSH: Dependovirus/*genetics , Genetic Therapy/*methods , Homeodomain Proteins/*genetics , Lysosomes/*metabolism , Sandhoff Disease/*therapy, Animals ; Brain/pathology ; Brain/physiopathology ; Cats ; Central Nervous System/metabolism ; Cerebrosides/metabolism ; Disease Models, Animal ; Disease Progression ; G(M2) Ganglioside/metabolism ; Gangliosides/metabolism ; Genetic Vectors ; Homeodomain Proteins/metabolism ; Quality of Life ; Sandhoff Disease/pathology ; Sandhoff Disease/physiopathology ; Sandhoff Disease/psychology ; Severity of Illness Index ; Spinal Cord/pathology ; Spinal Cord/physiopathology ; Sulfoglycosphingolipids/metabolism ; Treatment Outcome

المؤلفون: McCurdy VJ; 1] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA [2] Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Rockwell HE; Biology Department, Boston College, Chestnut Hill, MA, USA., Arthur JR; Biology Department, Boston College, Chestnut Hill, MA, USA., Bradbury AM; 1] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA [2] Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Johnson AK; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Randle AN; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Brunson BL; Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Hwang M; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Gray-Edwards HL; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Morrison NE; Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Johnson JA; Department of Clinical Sciences, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Baker HJ; 1] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA [2] Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Cox NR; 1] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA [2] Department of Pathobiology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA., Seyfried TN; Biology Department, Boston College, Chestnut Hill, MA, USA., Sena-Esteves M; Department of Neurology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA, USA., Martin DR; 1] Scott-Ritchey Research Center, College of Veterinary Medicine, Auburn University, Auburn, AL, USA [2] Department of Anatomy, Physiology and Pharmacology, College of Veterinary Medicine, Auburn University, Auburn, AL, USA.

المصدر: Gene therapy [Gene Ther] 2015 Feb; Vol. 22 (2), pp. 181-9. Date of Electronic Publication: 2014 Dec 04.

نوع المنشور: Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't

بيانات الدورية: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE

مواضيع طبية MeSH: Genetic Therapy*, Sandhoff Disease/*therapy, Animals ; Cats ; Dependovirus/genetics ; Dependovirus/immunology ; Disease Progression ; Gangliosides/metabolism ; Genetic Vectors ; Humans ; Immunity, Humoral ; Injections, Intraventricular ; Sandhoff Disease/pathology ; Transduction, Genetic ; Treatment Outcome ; beta-N-Acetylhexosaminidases/biosynthesis ; beta-N-Acetylhexosaminidases/genetics