Mediaspace scheduled maintenance: Aug 25, 2026 07:00 - 12:00 AM. During this time, videos will be temporarily unavailable. Check status updates.

Lecture

Approaches in Gene Therapy

Description

This lecture explores different approaches in gene therapy for various diseases, such as spinal muscular atrophy, Huntington disease, and Parkinson's disease. It discusses the advantages of gene transfer over conventional drugs, including accessibility, long-term treatment, and more physiological delivery. The lecture also covers delivery vectors, focusing on viral vectors and their characteristics in gene delivery.

In MOOC

Neuroscience Reconstructed: Genetics and Brain Development

Official source

Quiz

Ontological neighbourhood

Biology

Molecular biology: Topics in molecular biology

Related concepts (24)

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.

Gene therapy

Gene therapy is a medical technology which aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.

Transduction (genetics)

Transduction is the process by which foreign DNA is introduced into a cell by a virus or viral vector. An example is the viral transfer of DNA from one bacterium to another and hence an example of horizontal gene transfer. Transduction does not require physical contact between the cell donating the DNA and the cell receiving the DNA (which occurs in conjugation), and it is DNase resistant (transformation is susceptible to DNase).

Gene delivery

Gene delivery is the process of introducing foreign genetic material, such as DNA or RNA, into host cells. Gene delivery must reach the genome of the host cell to induce gene expression. Successful gene delivery requires the foreign gene delivery to remain stable within the host cell and can either integrate into the genome or replicate independently of it. This requires foreign DNA to be synthesized as part of a vector, which is designed to enter the desired host cell and deliver the transgene to that cell's genome.

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.

Related lectures (32)

Gene Therapy for CNS and Sensory Organs

Explores gene therapy for CNS and sensory organs, focusing on rare genetic diseases and advancements in precision medicine.

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

Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.

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.

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

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

Gene Therapy: Targets and Therapeutics