Approved gene therapies in Australia: coming to a store near you

Approved gene therapies in Australia: coming to a store near you.

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Item Type: Review
Status: Published
Official URL:
Journal or Publication Title: Internal Medicine Journal
Volume: 52
Number: 8
Page Range: pp. 1313-1321
Date: August 2022
Divisions: Cancer and Gene Regulation
Gene and Stem Cell Therapy
Depositing User: General Admin
Identification Number: 10.1111/imj.15880
ISSN: 1444-0903
Date Deposited: 26 Sep 2022 00:53

Gene therapy has been promising paradigm-shifting advances in medical science for over two decades. Broadly, it is defined as a human therapy in which an existing defective gene function is added to, replaced, edited or disrupted to achieve a clinical benefit, up to and including a potential lifelong cure. Although originally set out to treat monogenic disorders, gene therapy has since been utilised to treat neoplasia, cardiovascular and neurodegenerative diseases, as well as infections. The realisation of this therapy has been dependent on the achievement of fundamental milestones in medicine, from determining the human genome sequence to identifying effective vehicles for the gene of interest, ultimately facilitating gene delivery in humans. In this review, six approved gene and cell therapies available in Australia are described. Their efficacy, adverse effects, limitations and eligibility are discussed, as well as an overview of cost and future directions.

Mallik, Shreyashee
Bailey, Charles G.
Rasko, John E. J.
Last Modified: 26 Sep 2022 00:53

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