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PubPharm (29)
1
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates
enthalten in:
Nature communications
| 2021
von
Costa-Verdera, H.
|
Collaud, F.
|
Riling, C.
| +26
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2
Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects
enthalten in:
EBioMedicine
| 2020
von
Colella, P.
|
Sellier, P.
|
Gomez, M.
| +14
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3
Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2020
von
Cagin, U.
|
Puzzo, F.
|
Gomez, M.
| +15
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4
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
enthalten in:
Molecular therapy. Methods & clinical development
| 2019
von
Charrier, S.
|
Lagresle-Peyrou, C.
|
Poletti, V.
| +10
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5
Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer
enthalten in:
Molecular therapy. Methods & clinical development
| 2019
von
Da Rocha, S.
|
Bigot, J.
|
Onodi, F.
| +7
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6
An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress
enthalten in:
Molecular therapy. Methods & clinical development
| 2019
von
Israeli, D.
|
Cosette, J.
|
Corre, G.
| +6
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7
AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice
enthalten in:
Molecular therapy. Methods & clinical development
| 2019
von
Colella, P.
|
Sellier, P.
|
Costa Verdera, H.
| +12
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8
Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency
enthalten in:
Molecular therapy. Methods & clinical development
| 2018
von
Poletti, V.
|
Charrier, S.
|
Corre, G.
| +8
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9
Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
enthalten in:
Molecular therapy : the journal of the American Society of Gene Therapy
| 2018
von
Vidal, P.
|
Pagliarani, S.
|
Colella, P.
| +20
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10
Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
enthalten in:
Science translational medicine
| 2017
von
Puzzo, F.
|
Colella, P.
|
Biferi, M.
| +22
CommentIn: Ann Transl Med. 2018 Aug;6(15):313. - PMID 30211201
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Molecular therapy. Methods & clinical development
4
Molecular therapy : the journal of the American...
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Blood
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Brain : a journal of neurology
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Research Support, Non-U.S. Gov't
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gene therapy
4
AAV
4
EC 3.2.1.20
4
alpha-Glucosidases
3
9005-79-2
3
Glycogen
2
Biomarkers
2
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2
Pompe disease
1
9EI49ZU76O
1
ATP Binding Cassette Transporter, Subfamily B
1
ATP-Binding Cassette Transporters
1
Antibodies, Heterophile
1
Artemis
1
B cell
1
Blood Glucose
1
Chemokines
1
Clinical Trial, Phase I
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2020-
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2010-2019
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