Lentiviral Vectors; a Research Tool Used To Introduce a Gene Product Into In Vitro Systems

 

Lentiviral Vectors

Lentiviral Vectors in gene treatment is a technique by which genetically engineered viruses can be introduced, modified, or even deleted from living organisms with the help of lentivirus. These viruses are then injected into various target organs where they multiply and reproduce. Usually, these viruses are produced by e-fertilization of healthy tissues with DNA that has been taken from other sources. However, for this technology to work, the natural genetic material, i.e., the DNA of the recipient organism, must be in perfect order.

Lentivirus are a family of viruses that are responsible for chronic and deadly diseases in the human and other mammalian species. The best known lentivirus is human immunodeficiency virus (HIV), which causes AIDS. Lentiviral vectors are derived from HIV and hence, these vectors are highly efficient for in vivo gene delivery for gene therapies. Moreover, lentiviral vectors are used in both gene therapy and research applications. These vectors can also be used to treat variety of rare genetic disorders such as Metachromatic Leukodystrophy, X-linked severe combined immunodeficiency, X-linked Adrenoleukodystrophy, and β-thalassemia.

Thus, with increase in research and development for gene therapies, the demand for lentiviral vectors is also increasing. For example, in 2019, Avrobio Inc. (the U.S. based biotechnology company) started a phase I and phase II trial for gene therapy, including lentiviral vector, for patients with type 1 Gaucher disease; a rare, inherited metabolic disorder. Moreover, in May 2020, Avrobio Inc. revealed how lentiviral gene therapy may potentially correct Pompe disease manifestations in the muscle and central nervous system.

The reason why lentiviral vector therapy is more popular than any other form of retrovirus therapy; the process is faster, more effective, and less costly. Lentivirus is primarily a research tool used to introduce a gene product into in vitro systems or animal models. Furthermore, third-gen lentiviral vectors have recently been used in multiple clinical trials to introduce genes into hematopoietic stem cells to correct primary hemoglobinopathies and immunodeficiencies.


Location: India

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