Lecture

Production of Viral Vectors

Graph Chat

Chat with Graph Search

Ask any question about EPFL courses, lectures, exercises, research, news, etc. or try the example questions below.

DISCLAIMER: The Graph Chatbot is not programmed to provide explicit or categorical answers to your questions. Rather, it transforms your questions into API requests that are distributed across the various IT services officially administered by EPFL. Its purpose is solely to collect and recommend relevant references to content that you can explore to help you answer your questions.

Description

This lecture covers the production of viral vectors for gene therapy applications, focusing on adeno-associated virus (AAV) vectors. Topics include vector plasmids, regulatory sequences, AAV helper plasmids, adenovirus helper plasmids, transgene expression, serotypes, and production efficiency. The process involves large-scale production, purification, and quality control under Good Manufacturing Practice conditions.

In MOOC

Neuroscience Reconstructed: Genetics and Brain Development

Official source

Ontological neighbourhood

Biology

Molecular biology: Topics in molecular biology

Related concepts (32)

Viral vector

Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism (in vivo) or in cell culture (in vitro). Viruses have evolved specialized molecular mechanisms to efficiently transport their genomes inside the cells they infect. Delivery of genes or other genetic material by a vector is termed transduction and the infected cells are described as transduced. Molecular biologists first harnessed this machinery in the 1970s.

Vector (molecular biology)

In molecular cloning, a vector is any particle (e.g., plasmids, cosmids, Lambda phages) used as a vehicle to artificially carry a foreign nucleic sequence – usually DNA – into another cell, where it can be replicated and/or expressed. A vector containing foreign DNA is termed recombinant DNA. The four major types of vectors are plasmids, viral vectors, cosmids, and artificial chromosomes. Of these, the most commonly used vectors are plasmids. Common to all engineered vectors are an origin of replication, a multicloning site, and a selectable marker.

Gene therapy of the human retina

Retinal gene therapy holds a promise in treating different forms of non-inherited and inherited blindness. In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's congenital amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV). In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA.

Adeno-associated virus

Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus Dependoparvovirus, which in turn belongs to the family Parvoviridae. They are small (approximately 26 nm in diameter) replication-defective, nonenveloped viruses and have linear single-stranded DNA (ssDNA) genome of approximately 4.8 kilobases (kb). Several features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models.

Transgene

A transgene is a gene that has been transferred naturally, or by any of a number of genetic engineering techniques, from one organism to another. The introduction of a transgene, in a process known as transgenesis, has the potential to change the phenotype of an organism. Transgene describes a segment of DNA containing a gene sequence that has been isolated from one organism and is introduced into a different organism.

Related lectures (39)

Preclinical and Clinical Trials in SMA Gene Therapy MOOC: Neuroscience Reconstructed: Genetics and Brain Development

Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.

Gene Therapy: Neurological Diseases MOOC: Neuroscience Reconstructed: Genetics and Brain Development

Explores gene therapy for neurological diseases, including monogenic and multifactorial disorders, and the use of Cas9 and repair mechanisms.

Types of Viral Vectors MOOC: Neuroscience Reconstructed: Genetics and Brain Development

Explores the properties of commonly used viral vectors for gene therapy.

Regulatory Variation & Precision Medicine

Explores regulatory variation's impact on precision medicine, focusing on non-coding GWAS SNPs and gene expression variation.

Choosing a Viral Vector and Delivery Method MOOC: Neuroscience Reconstructed: Genetics and Brain Development