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Wyszukujesz frazę ""MENDELL, JERRY R."" wg kryterium: Autor


Wyświetlanie 1-9 z 9
Tytuł:
Assessing the value of delandistrogene moxeparvovec (SRP-9001) gene therapy in patients with Duchenne muscular dystrophy in the United States.
Autorzy:
Klimchak AC; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
Sedita LE; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
Rodino-Klapac LR; Global HEOR, RWE & Analytics, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
Mendell JR; Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.; Department of Pediatrics and Neurology, The Ohio State University, Columbus, OH, USA.
McDonald CM; Department of Pediatrics, University of California Davis School of Medicine, Davis, CA, USA.
Gooch KL; Research & Development, Sarepta Therapeutics, Inc, Cambridge, MA, USA.
Malone DC; College of Pharmacy, University of Utah, Salt Lake City, UT, USA.
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Źródło:
Journal of market access & health policy [J Mark Access Health Policy] 2023 May 26; Vol. 11 (1), pp. 2216518. Date of Electronic Publication: 2023 May 26 (Print Publication: 2023).
Typ publikacji:
Journal Article
Czasopismo naukowe
Tytuł:
AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models.
Autorzy:
Sondergaard PC; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Griffin DA; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Pozsgai ER; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio ; Biomedical Sciences Graduate Program, The Ohio State University Columbus, Ohio.
Johnson RW; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Grose WE; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Heller KN; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Shontz KM; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Montgomery CL; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Liu J; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio.
Clark KR; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio ; Biomedical Sciences Graduate Program, The Ohio State University Columbus, Ohio.
Sahenk Z; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics, The Ohio State University Columbus, Ohio ; Department of Neurology, The Ohio State University Columbus, Ohio.
Mendell JR; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics, The Ohio State University Columbus, Ohio ; Department of Neurology, The Ohio State University Columbus, Ohio.
Rodino-Klapac LR; Center for Gene Therapy, Nationwide Children's Hospital Columbus, Ohio ; Biomedical Sciences Graduate Program, The Ohio State University Columbus, Ohio ; Department of Pediatrics, The Ohio State University Columbus, Ohio.
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Źródło:
Annals of clinical and translational neurology [Ann Clin Transl Neurol] 2015 Mar; Vol. 2 (3), pp. 256-70. Date of Electronic Publication: 2015 Jan 20.
Typ publikacji:
Journal Article
Czasopismo naukowe
Tytuł:
A slowly progressive form of limb-girdle muscular dystrophy type 2C associated with founder mutation in the SGCG gene in Puerto Rican Hispanics.
Autorzy:
Al-Zaidy SA; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics and Neurology, The Ohio State University Columbus, Ohio.
Malik V; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio.
Kneile K; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital Columbus, Ohio.
Rosales XQ; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics and Neurology, The Ohio State University Columbus, Ohio.
Gomez AM; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio.
Lewis S; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio ; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital Columbus, Ohio.
Hashimoto S; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital Columbus, Ohio.
Gastier-Foster J; Department of Pathology, Ohio State University and Nationwide Children's Hospital Columbus, Ohio ; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital Columbus, Ohio.
Kang P; Department of Neurology, Boston Children's Hospital and Harvard Medical School Boston, Massachusetts.
Darras B; Department of Neurology, Boston Children's Hospital and Harvard Medical School Boston, Massachusetts.
Kunkel L; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital Boston, Massachusetts.
Carlo J; Department of Neurology, School of Medicine, University of Puerto Rico San Juan, Puerto Rico.
Sahenk Z; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics and Neurology, The Ohio State University Columbus, Ohio.
Moore SA; Department of Pathology, The University of Iowa Carver College of Medicine Iowa City, Iowa.
Pyatt R; Department of Pathology, Ohio State University and Nationwide Children's Hospital Columbus, Ohio ; Department of Pathology and Laboratory Medicine, Nationwide Children's Hospital Columbus, Ohio.
Mendell JR; Center for Gene Therapy and Paul D. Wellstone Muscular Dystrophy Research Center, Nationwide Children's Hospital Columbus, Ohio ; Department of Pediatrics and Neurology, The Ohio State University Columbus, Ohio ; Department of Pathology, Ohio State University and Nationwide Children's Hospital Columbus, Ohio.
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Źródło:
Molecular genetics & genomic medicine [Mol Genet Genomic Med] 2015 Mar; Vol. 3 (2), pp. 92-8. Date of Electronic Publication: 2015 Jan 08.
Typ publikacji:
Journal Article
Czasopismo naukowe
Tytuł:
Dexmedetomidine and ketamine sedation for muscle biopsies in patients with Duchenne muscular dystrophy.
Autorzy:
Kako H; Department of Anesthesiology & Pain Medicine, Nationwide Children's Hospital, Columbus, OH, USA; Department of Anesthesiology, The Ohio State University, Columbus, OH, USA.
Corridore M
Kean J
Mendell JR
Flanigan KM
Tobias JD
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Źródło:
Paediatric anaesthesia [Paediatr Anaesth] 2014 Aug; Vol. 24 (8), pp. 851-6. Date of Electronic Publication: 2014 Mar 20.
Typ publikacji:
Journal Article; Observational Study; Research Support, Non-U.S. Gov't
MeSH Terms:
Anesthetics, Dissociative*
Dexmedetomidine*
Hypnotics and Sedatives*
Ketamine*
Muscle, Skeletal/*surgery
Muscular Dystrophy, Duchenne/*surgery
Biopsy ; Child ; Drug Therapy, Combination ; Humans ; Male ; Muscle, Skeletal/pathology ; Prospective Studies
Czasopismo naukowe
Tytuł:
Homologous recombination mediates functional recovery of dysferlin deficiency following AAV5 gene transfer.
Autorzy:
Grose WE; Department of Pediatrics, The Ohio State University, Columbus, Ohio, United States of America.
Clark KR
Griffin D
Malik V
Shontz KM
Montgomery CL
Lewis S
Brown RH Jr
Janssen PM
Mendell JR
Rodino-Klapac LR
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Źródło:
PloS one [PLoS One] 2012; Vol. 7 (6), pp. e39233. Date of Electronic Publication: 2012 Jun 15.
Typ publikacji:
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms:
Gene Transfer Techniques*
Genetic Vectors*
Recombination, Genetic*
Dependovirus/*genetics
Membrane Proteins/*genetics
Muscle Proteins/*genetics
Dysferlin ; Humans
Czasopismo naukowe
Tytuł:
Cardiovascular magnetic resonance of cardiomyopathy in limb girdle muscular dystrophy 2B and 2I.
Autorzy:
Rosales XQ; Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, Columbus, Ohio 43205, USA.
Moser SJ
Tran T
McCarthy B
Dunn N
Habib P
Simonetti OP
Mendell JR
Raman SV
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Źródło:
Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance [J Cardiovasc Magn Reson] 2011 Aug 04; Vol. 13, pp. 39. Date of Electronic Publication: 2011 Aug 04.
Typ publikacji:
Journal Article
MeSH Terms:
Magnetic Resonance Imaging, Cine*
Cardiomyopathies/*diagnosis
Muscular Dystrophies, Limb-Girdle/*complications
Myocardium/*pathology
Adult ; Cardiomyopathies/etiology ; Cardiomyopathies/pathology ; Cardiomyopathies/physiopathology ; Child ; Contrast Media ; DNA Mutational Analysis ; Dysferlin ; Female ; Fibrosis ; Genetic Predisposition to Disease ; Humans ; Male ; Membrane Proteins/genetics ; Middle Aged ; Muscle Proteins/genetics ; Muscular Dystrophies, Limb-Girdle/genetics ; Mutation ; Ohio ; Predictive Value of Tests ; Prognosis ; Severity of Illness Index ; Stroke Volume ; Ventricular Function, Left ; Young Adult
SCR Disease Name:
Limb-girdle muscular dystrophy, type 2B
Czasopismo naukowe
Tytuł:
Novel and recurrent EMD mutations in patients with Emery-Dreifuss muscular dystrophy, identify exon 2 as a mutation hot spot.
Autorzy:
Brown CA; Diagnostics Systems, BD Diagnostics, Durham, NC, USA.
Scharner J
Felice K
Meriggioli MN
Tarnopolsky M
Bower M
Zammit PS
Mendell JR
Ellis JA
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Źródło:
Journal of human genetics [J Hum Genet] 2011 Aug; Vol. 56 (8), pp. 589-94. Date of Electronic Publication: 2011 Jun 23.
Typ publikacji:
Journal Article; Research Support, Non-U.S. Gov't
MeSH Terms:
Mutation*
Exons/*genetics
Membrane Proteins/*genetics
Muscular Dystrophy, Emery-Dreifuss/*genetics
Nuclear Proteins/*genetics
Adolescent ; Adult ; Child ; Child, Preschool ; Codon, Nonsense ; Cohort Studies ; DNA Mutational Analysis ; Female ; Frameshift Mutation ; Humans ; Infant, Newborn ; Male ; Middle Aged ; Mutagenesis, Insertional ; Mutation, Missense ; Sequence Deletion ; Young Adult
Czasopismo naukowe
Tytuł:
A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy.
Autorzy:
Rodino-Klapac LR; Center for Gene Therapy, Columbus Children's Research Institute, Columbus Children's Hospital, 700 Children's Dr., Columbus, Ohio 43205, USA. />Janssen PM
Montgomery CL
Coley BD
Chicoine LG
Clark KR
Mendell JR
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Źródło:
Journal of translational medicine [J Transl Med] 2007 Sep 24; Vol. 5, pp. 45. Date of Electronic Publication: 2007 Sep 24.
Typ publikacji:
Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
MeSH Terms:
Dystrophin/*genetics
Endothelium, Vascular/*physiopathology
Genetic Therapy/*methods
Muscular Dystrophy, Duchenne/*therapy
Animals ; Disease Models, Animal ; Gene Transfer Techniques ; Humans ; Incidence ; Male ; Mice ; Muscle, Skeletal/pathology ; Muscle, Skeletal/physiopathology ; Muscular Dystrophy, Animal/genetics ; Muscular Dystrophy, Animal/therapy ; Muscular Dystrophy, Duchenne/epidemiology ; Muscular Dystrophy, Duchenne/genetics ; Muscular Dystrophy, Duchenne/physiopathology ; Pressure
Czasopismo naukowe
    Wyświetlanie 1-9 z 9

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