The Promise and the Hope of Gene Therapy

It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the aff...

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Bibliographic Details
Published inFrontiers in genome editing Vol. 3; p. 618346
Main Authors Papanikolaou, Eleni, Bosio, Andreas
Format Journal Article
LanguageEnglish
Published Frontiers Media S.A 24.03.2021
Subjects
CRISPR
designer nucleases
Genome Editing
hemopoietic stem cell
retroviral vectors
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ISSN2673-3439
2673-3439
DOI10.3389/fgeed.2021.618346

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Summary:It has been over 30 years since visionary scientists came up with the term “Gene Therapy,” suggesting that for certain indications, mostly monogenic diseases, substitution of the missing or mutated gene with the normal allele via gene addition could provide long-lasting therapeutic effect to the affected patients and consequently improve their quality of life. This notion has recently become a reality for certain diseases such as hemoglobinopathies and immunodeficiencies and other monogenic diseases. However, the therapeutic wave of gene therapies was not only applied in this context but was more broadly employed to treat cancer with the advent of CAR-T cell therapies. This review will summarize the gradual advent of gene therapies from bench to bedside with a main focus on hemopoietic stem cell gene therapy and genome editing and will provide some useful insights into the future of genetic therapies and their gradual integration in the everyday clinical practice.
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This article was submitted to Genome Editing in Blood Disorders, a section of the journal Frontiers in Genome Editing
Edited by: Carsten Werner Lederer, The Cyprus Institute of Neurology and Genetics, Cyprus
Reviewed by: Jose Segovia, Centro de Investigaciones y Estudios Avanzados, Instituto Politécnico Nacional de México (CINVESTAV), Mexico; Alessia Finotti, University of Ferrara, Italy
ISSN:2673-3439
2673-3439
DOI:10.3389/fgeed.2021.618346