Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results
- Location
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Deutsche Nationalbibliothek Frankfurt am Main
- Extent
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1 Online-Ressource.
- Language
-
Englisch
- Bibliographic citation
-
Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1/2 trial interim results ; volume:30 ; number:5 ; day:14 ; month:5 ; year:2024 ; pages:1406-1415 ; date:5.2024
Nature medicine ; 30, Heft 5 (14.5.2024), 1406-1415, 5.2024
- Creator
-
Sevigny, Jeffrey
Uspenskaya, Olga
Heckman, Laura Dean
Wong, Li Chin
Hatch, Daniel A.
Tewari, Ambika
Vandenberghe, Rik
Irwin, David J.
Saracino, Dario
Ber, Isabelle Le
Ahmed, Rebekah
Rohrer, Jonathan D.
Boxer, Adam L.
Boland, Sebastian
Sheehan, Patricia
Brandes, Alissa
Burstein, Suzanne R.
Shykind, Benjamin M.
Kamalakaran, Sitharthan
Daniels, Carter W.
David Litwack, E.
Mahoney, Erin
Velaga, Jenny
McNamara, Ilan
Sondergaard, Patricia
Sajjad, Syed A.
Kobayashi, Yvonne M.
Abeliovich, Asa
Hefti, Franz
- Contributor
-
SpringerLink (Online service)
- DOI
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10.1038/s41591-024-02973-0
- URN
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urn:nbn:de:101:1-2407311022066.926132386881
- Rights
-
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
-
14.08.2025, 11:00 AM CEST
Data provider
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Associated
- Sevigny, Jeffrey
- Uspenskaya, Olga
- Heckman, Laura Dean
- Wong, Li Chin
- Hatch, Daniel A.
- Tewari, Ambika
- Vandenberghe, Rik
- Irwin, David J.
- Saracino, Dario
- Ber, Isabelle Le
- Ahmed, Rebekah
- Rohrer, Jonathan D.
- Boxer, Adam L.
- Boland, Sebastian
- Sheehan, Patricia
- Brandes, Alissa
- Burstein, Suzanne R.
- Shykind, Benjamin M.
- Kamalakaran, Sitharthan
- Daniels, Carter W.
- David Litwack, E.
- Mahoney, Erin
- Velaga, Jenny
- McNamara, Ilan
- Sondergaard, Patricia
- Sajjad, Syed A.
- Kobayashi, Yvonne M.
- Abeliovich, Asa
- Hefti, Franz
- SpringerLink (Online service)
Other Objects (12)
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Reduced Tau protein expression is associated with frontotemporal degeneration with progranulin mutation
Analytical and clinical validation of a blood progranulin ELISA in frontotemporal dementias
Impaired β-glucocerebrosidase activity and processing in frontotemporal dementia due to progranulin mutations
Diagnostic value of cerebrospinal fluid tau, neurofilament, and progranulin in definite frontotemporal lobar degeneration
Neuropathological and behavioral characterization of aged Grn R493X progranulin-deficient frontotemporal dementia knockin mice
Single-cell multimodal analysis in a case with reduced penetrance of Progranulin-Frontotemporal Dementia
Altered microRNA expression in frontotemporal lobar degeneration with TDP-43 pathology caused by progranulin mutations
Epigenetic modulation through BET bromodomain inhibitors as a novel therapeutic strategy for progranulin-deficient frontotemporal dementia
Processing of progranulin into granulins involves multiple lysosomal proteases and is affected in frontotemporal lobar degeneration
Progranulin in frontotemporal lobar degeneration and neuroinflammation
Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia
Preclinical Interventions in Mouse Models of Frontotemporal Dementia Due to Progranulin Mutations
Reduced Tau protein expression is associated with frontotemporal degeneration with progranulin mutation
Analytical and clinical validation of a blood progranulin ELISA in frontotemporal dementias
Impaired β-glucocerebrosidase activity and processing in frontotemporal dementia due to progranulin mutations
Diagnostic value of cerebrospinal fluid tau, neurofilament, and progranulin in definite frontotemporal lobar degeneration
Neuropathological and behavioral characterization of aged Grn R493X progranulin-deficient frontotemporal dementia knockin mice
Single-cell multimodal analysis in a case with reduced penetrance of Progranulin-Frontotemporal Dementia
Altered microRNA expression in frontotemporal lobar degeneration with TDP-43 pathology caused by progranulin mutations
Epigenetic modulation through BET bromodomain inhibitors as a novel therapeutic strategy for progranulin-deficient frontotemporal dementia
Processing of progranulin into granulins involves multiple lysosomal proteases and is affected in frontotemporal lobar degeneration
Progranulin in frontotemporal lobar degeneration and neuroinflammation
Latozinemab, a novel progranulin-elevating therapy for frontotemporal dementia
Preclinical Interventions in Mouse Models of Frontotemporal Dementia Due to Progranulin Mutations
Reduced Tau protein expression is associated with frontotemporal degeneration with progranulin mutation
Analytical and clinical validation of a blood progranulin ELISA in frontotemporal dementias
Impaired β-glucocerebrosidase activity and processing in frontotemporal dementia due to progranulin mutations
Diagnostic value of cerebrospinal fluid tau, neurofilament, and progranulin in definite frontotemporal lobar degeneration
Neuropathological and behavioral characterization of aged Grn R493X progranulin-deficient frontotemporal dementia knockin mice
Single-cell multimodal analysis in a case with reduced penetrance of Progranulin-Frontotemporal Dementia
Altered microRNA expression in frontotemporal lobar degeneration with TDP-43 pathology caused by progranulin mutations
Epigenetic modulation through BET bromodomain inhibitors as a novel therapeutic strategy for progranulin-deficient frontotemporal dementia