Preprint Review Version 1 Preserved in Portico This version is not peer-reviewed

Viral Vectors Engineered for Gene Therapy

Version 1 : Received: 20 April 2023 / Approved: 21 April 2023 / Online: 21 April 2023 (08:55:15 CEST)

How to cite: Lundstrom, K. Viral Vectors Engineered for Gene Therapy. Preprints 2023, 2023040697. https://doi.org/10.20944/preprints202304.0697.v1 Lundstrom, K. Viral Vectors Engineered for Gene Therapy. Preprints 2023, 2023040697. https://doi.org/10.20944/preprints202304.0697.v1

Abstract

Gene therapy has seen major progress in recent years. Viral vectors have made a significant contribution through efficient engineering for improved delivery and safety. A large variety of indications such as cancer, cardiovascular, metabolic, hematological, neurological, muscular, ophthalmological, infectious diseases, and immunodeficiency have been targeted. Viral vectors based on adenoviruses, adeno-associated viruses, herpes simplex viruses, retroviruses including lentiviruses, alphaviruses, flaviviruses, measles viruses, rhabdoviruses, Newcastle disease virus, poxviruses, picornaviruses, reoviruses, and polyomaviruses have been used. Proof-of-concept has been demonstrated for different indications in animal models. Therapeutic efficacy has also been achieved in clinical trials. Several viral vector-based drugs have been approved for the treatment of cancer, and hematological, metabolic, and neurological diseases. Moreover, viral vector-based vaccines have been approved against COVID-19 and Ebola virus disease.

Keywords

viral vectors; gene therapy; animal models; clinical trials; drug approval

Subject

Biology and Life Sciences, Virology

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