Preclinical Development and Characterization of Novel Adeno-Associated Viral Vectors for the Treatment of Lipoprotein Lipase Deficiency.

Humans Animals Mice Hyperlipoproteinemia Type I / genetics Tissue Distribution Transgenes Administration, Intravenous
Glybera adeno-associated virus (AAV) gene therapy lipoprotein lipase lipoprotein lipase deficiency

Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
09 2023
Historique:
medline: 21 9 2023
pubmed: 19 8 2023
entrez: 19 8 2023
Statut: ppublish

Résumé

Lipoprotein lipase deficiency (LPLD) results from mutations within the

Identifiants

DOI: 10.1089/hum.2023.075 PMID: 37597209
pubmed: 37597209
doi: 10.1089/hum.2023.075
doi:

Substances chimiques

Alipogene tiparvovec 20OK4AFR4Y

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

927-946

Subventions

Organisme : CIHR
ID : 201910MFE-430323-FPP-CAAA-267763
Pays : Canada

Auteurs

Neel Mehta (N)

Department of Medical Genetics, Center for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.

Rénald Gilbert (R)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.
Department of Bioengineering, McGill University, Montréal, Canada.

Parminder S Chahal (PS)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Maria J Moreno (MJ)

Department of Translational Biosciences, Human Health Therapeutics Research Center, National Research Council Canada, Ottawa, Canada.

Nasha Nassoury (N)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Nathalie Coulombe (N)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Viktoria Lytvyn (V)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Mario Mercier (M)

Department of Translational Biosciences, Human Health Therapeutics Research Center, National Research Council Canada, Ottawa, Canada.

Dorothy Fatehi (D)

Department of Translational Biosciences, Human Health Therapeutics Research Center, National Research Council Canada, Ottawa, Canada.

Wendy Lin (W)

Department of Medical Genetics, Center for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.

Emily M Harvey (EM)

Department of Medical Genetics, Center for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.
ORCID: 0000-0002-8848-5529

Lin-Hua Zhang (LH)

Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, Canada.

Nazila Nazemi-Moghaddam (N)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Seyyed Mehdy Elahi (SM)

Department of Production Platforms and Analytics, Human Health Therapeutics Research Center, National Research Council Canada, Montréal, Canada.

Colin J D Ross (CJD)

Department of Medical Genetics, Center for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.
Faculty of Pharmaceutical Sciences, University of British Columbia, Vancouver, Canada.

Danica B Stanimirovic (DB)

Department of Translational Biosciences, Human Health Therapeutics Research Center, National Research Council Canada, Ottawa, Canada.

Michael R Hayden (MR)

Department of Medical Genetics, Center for Molecular Medicine and Therapeutics, BC Children's Hospital Research Institute, University of British Columbia, Vancouver, Canada.
ORCID: 0000-0001-5159-1419

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Preclinical Development and Characterization...
questionsmedicales.fr › Eucaryotes › Animaux › Preclinical Development and Characterization...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Preclinical Development and Characterization...
questionsmedicales.fr › Maladies métaboliques et nutritionnelles › Maladies métaboliques › Erreurs innées du métabolisme › Erreurs innées du métabolisme lipidique › Hyperlipoprotéinémie de type I › Preclinical Development and Characterization...
questionsmedicales.fr › Phénomènes physiologiques › Phénomènes pharmacologiques et toxicologiques › Pharmacocinétique › Distribution tissulaire › Preclinical Development and Characterization...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Génome › Composants de génome › Gènes › Transgènes › Preclinical Development and Characterization...
questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Voies d'administration de substances chimiques et des médicaments › Administration par voie intraveineuse › Preclinical Development and Characterization...