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

Mehta, Neel; Gilbert, Rénald; Chahal, Parminder S.; Moreno, Maria J.; Nassoury, Nasha; Coulombe, Nathalie; Lytvyn, Viktoria; Mercier, Mario; Fatehi, Dorothy; Lin, Wendy; Harvey, Emily M.; Zhang, Lin-Hua; Nazemi-Moghaddam, Nazila; Elahi, Seyyed Mehdy; Ross, Colin J.D.; Stanimirovic, Danica B.; Hayden, Michael R.

doi:10.1089/hum.2023.075

Human Gene Therapy

2023

DOI: 10.1089/hum.2023.075

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

Neel Mehta,

Rénald Gilbert,

Parminder S. Chahal

et al.

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Cited by 4 publications

References 63 publications

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The GPIHBP1-LPL complex and its role in plasma triglyceride metabolism: Insights into chylomicronemia

Jiang,

Ren,

Yang

et al. 2023

Biomedicine & Pharmacotherapy

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The GPIHBP1-LPL complex and its role in plasma triglyceride metabolism: Insights into chylomicronemia

Jiang,

Ren,

Yang

et al. 2023

Biomedicine & Pharmacotherapy

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Adeno-associated virus therapies: Pioneering solutions for human genetic diseases

Liu,

Li,

Liu

et al. 2024

Cytokine & Growth Factor Reviews

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Application of the Insect Cell-Baculovirus Expression Vector System in Adeno-Associated Viral Production

Wang,

Sun,

Guo

et al. 2024

Applied Sciences

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Insect Cell-Baculovirus Expression Vector System (IC-BEVS) is an efficient protein expression platform, which is famous for its high-level expression of complex protein in insect cells. The system is based on baculoviruses such as Autographa californica multiple nucleopolyhedrovirus (AcMNPV), and the expression efficiency of the target proteins has been significantly improved by optimizing the viral vectors and cell lines. In recent years, IC-BEVS have shown great potential for Adeno-Associated Virus (AAV) production, particularly excelling in AAV structural protein expression and recombinant AAV production. The system not only improves the yield and purity of AAV, but also shortens the production cycle, providing an efficient and reliable tool for gene therapy. However, the system also has some challenges, including protein modification differences, limitations in expression levels, and production costs. This paper reviews the development of the insect baculovirus expression system, its application in AAV production, and its prospects in gene therapy, aiming to provide a systematic reference and outlook for research in related fields.

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

Cited by 4 publications

References 63 publications

The GPIHBP1-LPL complex and its role in plasma triglyceride metabolism: Insights into chylomicronemia

The GPIHBP1-LPL complex and its role in plasma triglyceride metabolism: Insights into chylomicronemia

Adeno-associated virus therapies: Pioneering solutions for human genetic diseases

Application of the Insect Cell-Baculovirus Expression Vector System in Adeno-Associated Viral Production

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