Search Results - "Collaud F"

TH.O.18 - Adeno associated vector-based gene therapy strategy for type 3 glycogen storage disease.

Authors: Vidal, P. Ronzitti, G. Collaud, F. et al.

Source: Neuromuscular Disorders. Oct2017 Supplement 2, Vol. 27, pS246-S246. 1p.

TH.O.17 - Whole-body rescue of Pompe disease with AAV liver delivery of engineered secretable GAA transgenes.

Authors: Colella, P. Puzzo, F. Biferi, M. et al.

Source: Neuromuscular Disorders. Oct2017 Supplement 2, Vol. 27, pS246-S246. 1p.

Gene supplementation therapy restores motor deficits in Gnao1[C215Y] mice and proves broad efficacy in C. elegans model.

Authors: Cocozza G D'Andrea T Di Rocco M et al.

Source: Acta neuropathologica communications [Acta Neuropathol Commun] 2026 Jul 21. Date of Electronic Publication: 2026 Jul 21.

Publication Type: Journal Article

Journal Info: Publisher: BioMed Central Country of Publication: England NLM ID: 101610673 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2051-5960 (Electronic) Linking ISSN: 20515960 NLM ISO Abbreviation: Acta Neuropathol Commun Subsets: MEDLINE

Muscle-specific, liver-detargeted adeno-associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa^-/- mice.

Authors: Sellier P Vidal P Bertin B et al.

Source: Journal of inherited metabolic disease [J Inherit Metab Dis] 2024 Jan; Vol. 47 (1), pp. 119-134. Date of Electronic Publication: 2023 May 29.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Wiley Country of Publication: United States NLM ID: 7910918 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1573-2665 (Electronic) Linking ISSN: 01418955 NLM ISO Abbreviation: J Inherit Metab Dis Subsets: MEDLINE

A myotropic AAV vector combined with skeletal muscle cis-regulatory elements improve glycogen clearance in mouse models of Pompe disease.

Authors: Sellier P Collaud F Benchekroun YK et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2025 Apr 09; Vol. 33 (2), pp. 101464. Date of Electronic Publication: 2025 Apr 09 (Print Publication: 2025).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: In Process

Editorial: Gene therapy and genome editing for metabolic liver disorders.

Authors: Collaud F Bortolussi G

Source: Frontiers in molecular medicine [Front Mol Med] 2025 Jan 09; Vol. 4, pp. 1543685. Date of Electronic Publication: 2025 Jan 09 (Print Publication: 2024).

Publication Type: Editorial

Journal Info: Publisher: Frontiers Media S.A Country of Publication: Switzerland NLM ID: 9918402378306676 Publication Model: eCollection Cited Medium: Internet ISSN: 2674-0095 (Electronic) Linking ISSN: 26740095 NLM ISO Abbreviation: Front Mol Med Subsets: PubMed not MEDLINE

Gene therapy for Crigler Najjar syndrome: results of the dose finding part of the CareCN, phase 1-2 clinical trial.

Authors: D'Antiga, L. Beuers, U. Brunetti-Pierri, N. et al.

Source: Journal of pediatric gastroenterology and nutrition 2022 Supplement; Vol. 74.

Journal Info: Publisher: Wiley-Blackwell ISSN: 0277-2116

Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.

Authors: Shi X Bortolussi G Collaud F et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2024 Oct 28; Vol. 32 (4), pp. 101363. Date of Electronic Publication: 2024 Oct 28 (Print Publication: 2024).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates.

Authors: Costa-Verdera H Collaud F Riling CR et al.

Source: Nature communications [Nat Commun] 2021 Nov 04; Vol. 12 (1), pp. 6393. Date of Electronic Publication: 2021 Nov 04.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver.

Authors: Jauze L Vie M Miagoux Q et al.

Source: JCI insight [JCI Insight] 2024 May 16; Vol. 9 (11). Date of Electronic Publication: 2024 May 16.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: Electronic Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE

Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors.

Authors: Barbon E Ayme G Mohamadi A et al.

Source: EMBO molecular medicine [EMBO Mol Med] 2020 Apr 07; Vol. 12 (4), pp. e11298. Date of Electronic Publication: 2020 Mar 11.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: EMBO Press Country of Publication: Germany NLM ID: 101487380 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1757-4684 (Electronic) Linking ISSN: 17574676 NLM ISO Abbreviation: EMBO Mol Med Subsets: MEDLINE

Lethality rescue and long-term amelioration of a citrullinemia type I mouse model by neonatal gene-targeting combined to SaCRISPR-Cas9.

Authors: Lisjak M Iaconcig A Guarnaccia C et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Aug 28; Vol. 31, pp. 101103. Date of Electronic Publication: 2023 Aug 28 (Print Publication: 2023).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Gene Therapy in Patients with the Crigler-Najjar Syndrome.

Authors: D'Antiga L Beuers U Ronzitti G et al.

Source: The New England journal of medicine [N Engl J Med] 2023 Aug 17; Vol. 389 (7), pp. 620-631.

Publication Type: Clinical Trial, Phase I; Clinical Trial, Phase II; Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Massachusetts Medical Society Country of Publication: United States NLM ID: 0255562 Publication Model: Print Cited Medium: Internet ISSN: 1533-4406 (Electronic) Linking ISSN: 00284793 NLM ISO Abbreviation: N Engl J Med Subsets: MEDLINE

Development of a dual hybrid AAV vector for endothelial-targeted expression of von Willebrand factor.

Authors: Barbon E Kawecki C Marmier S et al.

Source: Gene therapy [Gene Ther] 2023 Apr; Vol. 30 (3-4), pp. 245-254. Date of Electronic Publication: 2021 Jan 17.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Nature Publishing Group Country of Publication: England NLM ID: 9421525 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1476-5462 (Electronic) Linking ISSN: 09697128 NLM ISO Abbreviation: Gene Ther Subsets: MEDLINE

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans.

Authors: Kuranda K Jean-Alphonse P Leborgne C et al.

Source: The Journal of clinical investigation [J Clin Invest] 2018 Dec 03; Vol. 128 (12), pp. 5267-5279. Date of Electronic Publication: 2018 Oct 22.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 7802877 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1558-8238 (Electronic) Linking ISSN: 00219738 NLM ISO Abbreviation: J Clin Invest Subsets: MEDLINE

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration.

Authors: Meliani A Boisgerault F Hardet R et al.

Source: Nature communications [Nat Commun] 2018 Oct 05; Vol. 9 (1), pp. 4098. Date of Electronic Publication: 2018 Oct 05.

Publication Type: Journal Article; Research Support, Non-U.S. Gov't

Journal Info: Publisher: Nature Pub. Group Country of Publication: England NLM ID: 101528555 Publication Model: Electronic Cited Medium: Internet ISSN: 2041-1723 (Electronic) Linking ISSN: 20411723 NLM ISO Abbreviation: Nat Commun Subsets: MEDLINE

Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase.

Authors: Puzzo F Colella P Biferi MG et al.

Source: Science translational medicine [Sci Transl Med] 2017 Nov 29; Vol. 9 (418).

Publication Type: Journal Article

Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients.

Authors: Shi X Aronson SJ Ten Bloemendaal L et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Dec 03; Vol. 20, pp. 287-297. Date of Electronic Publication: 2020 Dec 03 (Print Publication: 2021).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.

Authors: De Sabbata G Boisgerault F Guarnaccia C et al.

Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2020 Nov 17; Vol. 20, pp. 169-180. Date of Electronic Publication: 2020 Nov 17 (Print Publication: 2021).

Publication Type: Journal Article

Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE

Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects.

Authors: Colella P Sellier P Gomez MJ et al.

Source: EBioMedicine [EBioMedicine] 2020 Nov; Vol. 61, pp. 103052. Date of Electronic Publication: 2020 Oct 09.

Publication Type: Journal Article

Journal Info: Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101647039 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3964 (Electronic) Linking ISSN: 23523964 NLM ISO Abbreviation: EBioMedicine Subsets: MEDLINE