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PubPharm (65)
1
Deciphering conundrums of adeno-associated virus liver-directed gene therapy : focus on hemophilia
enthalten in:
Journal of thrombosis and haemostasis : JTH
| 2024
von
Pierce, G.
|
Fong, S.
|
Long, B.
| +1
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2
COMPOSITIONS AND METHODS FOR TREATING LONG QT SYNDROME
enthalten in:
Europäisches Patentamt
| 2024
von
LI JIN
|
KAMANI MUSTAFA
|
FONG SYLVIA
| +3
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3
Vector integration and fate in the hemophilia dog liver multi-years following AAV-FVIII gene transfer
enthalten in:
Blood
| 2024
von
Batty, P.
|
Fong, S.
|
Franco, M.
| +13
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4
Adeno-associated viruses for gene therapy - clinical implications and liver-related complications, a guide for hepatologists
enthalten in:
Journal of hepatology
| 2024
von
Mücke, M.
|
Fong, S.
|
Foster, G.
| +3
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5
Prophylactic Prednisolone Promotes AAV5 Hepatocyte Transduction Through the Novel Mechanism of AAV5 Coreceptor Platelet-Derived Growth Factor Receptor Alpha Upregulation and Innate Immune Suppression
enthalten in:
Human gene therapy
| 2024
von
Handyside, B.
|
Zhang, L.
|
Yates, B.
| +16
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6
Valoctocogene roxaparvovec gene transfer in participants with HIV
enthalten in:
Blood advances
| 2023
von
Ragni, M.
|
Majerus, E.
|
Fong, S.
| +6
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7
Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2022
von
Handyside, B.
|
Ismail, A.
|
Zhang, L.
| +16
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8
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction
enthalten in:
The Journal of biological chemistry
| 2022
von
Magat, J.
|
Jones, S.
|
Baridon, B.
| +29
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9
Application of in-vitro-cultured primary hepatocytes to evaluate species translatability and AAV transduction mechanisms of action
enthalten in:
Molecular therapy. Methods & clinical development
| 2022
von
Liu, S.
|
Razon, L.
|
Ritchie, O.
| +15
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10
Young mice administered adult doses of AAV5-hFVIII-SQ achieve therapeutic factor VIII expression into adulthood
enthalten in:
Molecular therapy. Methods & clinical development
| 2022
von
Zhang, L.
|
Yates, B.
|
Murphy, R.
| +23
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