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Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. [ 1 ] [ 2 ] [ 3 ]
This is an accepted version of this page This is the latest accepted revision, reviewed on 13 February 2025. Manipulation of an organism's genome For a non-technical introduction to the topic of genetics, see Introduction to genetics. For the song by Orchestral Manoeuvres in the Dark, see Genetic Engineering (song). For the Montreal hardcore band, see Genetic Control. Part of a series on ...
The gene must then be isolated and incorporated, along with other genetic elements, into a suitable vector. This vector is then used to insert the gene into the host genome, creating a transgenic or edited organism. The ability to genetically engineer organisms is built on years of research and discovery on gene function and manipulation.
Gain-of-function research (GoF research or GoFR) is medical research that genetically alters an organism in a way that may enhance the biological functions of gene products. This may include an altered pathogenesis , transmissibility , or host range , i.e., the types of hosts that a microorganism can infect.
The safety of gene therapy treatment is of utmost concern, especially during clinical trials when off-target modifications can block the further development of a candidate product. [57] Perhaps the most well-known example of modern gene therapy is CAR-T therapy, which is used for the treatment of B-cell lymphoma .
In gene therapy a gene that is intended for delivery is packaged into a replication-deficient viral particle to form a viral vector. [29] Viruses used for gene therapy to date include retrovirus, adenovirus, adeno-associated virus and herpes simplex virus. However, there are drawbacks to using viruses to deliver genes into cells.
The first retinal gene therapy to be approved by the FDA was Voretigene neparvovec in 2017, which treats Leber's congenital amaurosis, a genetic disorder that can lead to blindness. These treatments also use subretinal injections of AAV vector and are therefore foundational to research in gene therapy for color blindness. [2] [3]
Gene targeting has also been investigated for gene therapy to correct disease-causing mutations. However the low efficiency of delivery of the gene-targeting machinery into cells has hindered this, with research conducted into viral vectors for gene targeting to try and address these challenges.