Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.[1][2][3]
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.[4] 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. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in phase I.[5] In 2003, Gendicine became the first gene therapy to receive regulatory approval. Since that time, further gene therapy drugs were approved, such as Glybera (2012), Strimvelis (2016), Kymriah (2017), Luxturna (2017), Onpattro (2018), Zolgensma (2019), Abecma (2021), Adstiladrin, Roctavian and Hemgenix (all 2022). Most of these approaches utilize adeno-associated viruses (AAVs) and lentiviruses for performing gene insertions, in vivo and ex vivo, respectively. AAVs are characterized by stabilizing the viral capsid, lower immunogenicity, ability to transduce both dividing and nondividing cells, the potential to integrate site specifically and to achieve long-term expression in the in-vivo treatment.[6] ASO / siRNA approaches such as those conducted by Alnylam and Ionis Pharmaceuticals require non-viral delivery systems, and utilize alternative mechanisms for trafficking to liver cells by way of GalNAc transporters.
Not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[7]
^Kaji EH, Leiden JM (February 2001). "Gene and stem cell therapies". JAMA. 285 (5): 545–550. doi:10.1001/jama.285.5.545. PMID 11176856.
^Ermak G (2015). Emerging Medical Technologies. World Scientific. ISBN 978-981-4675-81-9.
^Research, Center for Biologics Evaluation and (9 December 2020). "What is Gene Therapy?". FDA.
^Rosenberg SA, Aebersold P, Cornetta K, Kasid A, Morgan RA, Moen R, et al. (August 1990). "Gene transfer into humans--immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction". The New England Journal of Medicine. 323 (9): 570–578. doi:10.1056/NEJM199008303230904. PMID 2381442.
^"Gene Therapy Clinical Trials Worldwide Database". The Journal of Gene Medicine. Wiley. June 2016. Archived from the original on 31 July 2020.
^Gorell E, Nguyen N, Lane A, Siprashvili Z (April 2014). "Gene therapy for skin diseases". Cold Spring Harbor Perspectives in Medicine. 4 (4): a015149. doi:10.1101/cshperspect.a015149. PMC 3968787. PMID 24692191.
^Zimmer C (16 September 2013). "DNA Double Take". The New York Times. Archived from the original on 2 January 2022.
Genetherapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological...
Genetherapy utilizes the delivery of DNA into cells, which can be accomplished by several methods, summarized below. The two major classes of methods...
This article contains a list of commercially available genetherapies. Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency...
Genetherapy for color blindness is an experimental genetherapy of the human retina aiming to grant typical trichromatic color vision to individuals with...
designed to address the root cause are currently available including genetherapy (Elevidys), and antisense drugs (Ataluren, Eteplirsen etc.). Other medications...
genes: genetherapygenetherapy for epilepsy genetherapy for osteoarthritis genetherapy for color blindness genetherapy of the human retina gene therapy...
using gene therapy as routine therapy is not suggested. A small study published in 2015 found a small benefit. The focus of much CF genetherapy research...
and thus its host organism. Gene delivery is a necessary step in genetherapy for the introduction or silencing of a gene to promote a therapeutic outcome...
Novartis GeneTherapies, until 2020 known as AveXis, is a biotechnology company that develops treatments for rare neurological genetic disorders. It was...
people with severe disease. Currently there is only one FDA-approved genetherapy that is commercially available to RP patients with Leber congenital amaurosis...
Lentiviral vectors in genetherapy is a method by which genes can be inserted, modified, or deleted in organisms using lentiviruses. Lentiviruses are...
Emmanuel (February 2016). "GeneTherapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β – Gene". Human GeneTherapy. 27 (2): 148–165. doi:10...
introduction (through genetherapy) of suicide genes is a potential way of treating cancer or other proliferative diseases. Suicide genes form the basis of...
desmopressin may be used in those with mild haemophilia A. Studies of genetherapy are in early human trials. Haemophilia A affects about 1 in 5,000–10...
stem-cell-based therapy. Other genetherapy approaches has also been explored in the context of glioblastoma, including suicide genetherapy. Suicide genetherapy is...
purposes, by targeting mutations to specific genes, and in genetherapy. By inserting a functional gene into an organism and targeting it to replace the...
Genetherapy for osteoarthritis is the application of genetherapy to treat osteoarthritis (OA). Unlike pharmacological treatments which are administered...
genetic engineering has the potential to cure genetic diseases through genetherapy. Chinese hamster ovary (CHO) cells are used in industrial genetic engineering...
transfusion and the medication hydroxycarbamide (hydroxyurea). In 2023, new genetherapies were approved. A small percentage of people can be cured by a transplant...
regulation of disease-related genes may enable novel therapies for many diseases, especially in cases where adequate genetherapies are not yet developed or...
"Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine". Genes & Diseases. 4 (2):...
choroideremia; however, retinal genetherapy clinical trials have demonstrated a possible treatment. Since the CHM gene is located on the X chromosome...
Gene doping is the hypothetical non-therapeutic use of genetherapy by athletes in order to improve their performance in those sporting events which prohibit...
are limited. For the childhood cerebral form, stem cell transplant and genetherapy are options if the disease is detected early in the clinical course....
occur. More recently genetherapy has been attempted as an alternative to the bone marrow transplant. Transduction of the missing gene to hematopoietic stem...
genetic modification. This could be done in order to cure diseases (genetherapy), prevent the possibility of getting a particular disease (similarly...
definitive therapy available for ADA deficiency is genetherapy. These therapies use a viral vector to integrate a working copy of the gene into the patient's...