Abstract
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.
Highlights
The idea that a gene can be delivered into specific cell types and its expression can lead to therapeutic efficacy, dramatically improving the patients’ quality of life, was originally introduced by Theodore Friedmann 45 years ago and was later strongly encouraged and realized by George Stamatoyannopoulos, one of the founding members of the American Society of Gene and Cell Therapy (ASGCT)
One important difference between the two studies was the multiplicity of infection (MOI); in the first study, an MOI = 10 was employed, while in the second study, the authors experimented with MOI = 100. These results immediately suggest that the MOI plays a crucial role during the manufacturing process since both studies employed a VSV-G pseudotyped GFP encoding lentiviral vector and used cord blood CD34+ cells
The results of this study proved the efficacy of nanoparticles in terms of phenotype correction in the context of monogenic diseases
Summary
Specialty section: This article was submitted to Genome Editing in Blood Disorders, a section of the journal Frontiers in Genome Editing 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 improve their quality of life. 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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