Gene therapy is a medical field that focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989. The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990. It is thought to be able to cure many genetic disorders or treat them over time.
The concept of gene therapy is to fix a genetic problem at its source. If, for instance, a mutation in a certain gene causes the production of a dysfunctional protein resulting (usually recessively) in an inherited disease, gene therapy could be used to deliver a copy of this gene that does not contain the deleterious mutation and thereby produces a functional protein. This strategy is referred to as gene replacement therapy and is employed to treat inherited retinal diseases.
Sample Chapter(s)
Preface (37 KB)
Components of the Book:
- Chapter 1.
Adenoviral vectors for cardiovascular gene therapy applications: a clinical and industry perspective
- Chapter 2.
Combination gene therapy for HIV using a conditional suicidal gene with CCR5 knockout
- Chapter 3.
Gene therapy for cystic fibrosis: new tools for precision medicine
- Chapter 4.
Homology‑directed gene‑editing approaches for hematopoietic stem and progenitor cell gene therapy
- Chapter 5.
Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study
- Chapter 6.
Potential of helper‑dependent Adenoviral vectors in CRISPR‑cas9‑mediated lung gene therapy
- Chapter 7.
The sodium iodide symporter (NIS) as theranostic gene: its emerging role in new imaging modalities and non‑viral gene therapy
- Chapter 8.
Autism spectrum disorder: prospects for treatment using gene therapy
- Chapter 9.
Gene therapy-emulating small molecule treatments in cystic fibrosis airway epithelial cells and patients
- Chapter 10.
Impact of gene therapy for canine monogenic diseases on the progress of preclinical studies
- Chapter 11.
Variation in market access decisions for cell and gene therapies across the United States, Canada, and Europe
- Chapter 12.
Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues
- Chapter 13.
Surviving a radiogenetic promoter for glioblastoma viral gene therapy independently from CArG motifs
Readership:
Students, academics, teachers and other people attending or interested in Gene Therapy
Himanshu Garg
Department of Molecular and Translational Medicine, Center of Emphasis in Infectious Diseases, Texas Tech University Health Sciences Center, 5001 El Paso Dr, El Paso, TX, 79905, USA
Anjali Joshi
Department of Molecular and Translational Medicine, Center of Emphasis in Infectious Diseases, Texas Tech University Health Sciences Center, 5001 El Paso Dr, El Paso, TX, 79905, USA
Jin-A Lee
Program in Developmental and Stem Cell Biology, Hospital for Sick Children, 686 Bay Street, PGCRL 16-9420, Toronto, ON, M5G0A4, Canada
Amy P. Wong
Program in Developmental and Stem Cell Biology, Hospital for Sick Children, 686 Bay Street, PGCRL 16-9420, Toronto, ON, M5G0A4, Canada
Daniel P. Hart
Royal London Haemophilia Centre, Barts and The London School of Medicine and Dentistry, Queen Mary University London, London, E1 1BB, UK
and more...