Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys.

Tukov, Francis Fonyuy; Mansfield, Keith; Milton, Mark; Meseck, Emily; Penraat, Kelley; Chand, Deepa; Hartmann, Andreas

Tukov, Francis Fonyuy; Mansfield, Keith; Milton, Mark; Meseck, Emily; Penraat, Kelley; Chand, Deepa; Hartmann, Andreas.

Afiliação

Tukov FF; Department of Preclinical Safety/Translational Medicine, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
Mansfield K; Department of Discovery and Investigative Pathology, Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
Milton M; Department of PK Sciences-Gene Therapy TA, Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
Meseck E; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA.
Penraat K; Novartis Institutes for BioMedical Research, Cambridge, Massachusetts, USA.
Chand D; Novartis Gene Therapies, Inc., Bannockburn, Illinois, USA.
Hartmann A; Department of Pediatrics, Washington University School of Medicine and St Louis Children's Hospital, St Louis, Missouri, USA.

Hum Gene Ther ; 33(13-14): 740-756, 2022 07.

Article em En | MEDLINE | ID: mdl-35331006

Assuntos

Everolimo; Gânglios Espinais; Animais; Everolimo/metabolismo; Gânglios Espinais/metabolismo; Humanos; Inflamação/metabolismo; Macaca fascicularis; Prednisolona/metabolismo; Prednisolona/uso terapêutico; Rituximab/metabolismo

Palavras-chave

adeno-associated viruses; dorsal root ganglion; intrathecal administration; nonhuman primate; onasemnogene abeparvovec; spinal muscular atrophy

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Everolimo / Gânglios Espinais Limite: Animals / Humans Idioma: En Ano de publicação: 2022 Tipo de documento: Article

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