Gene therapy is an insertion of functional gene in neighboring or instead of dysfunctional genes.
CRISPR /Cas9 has been implemented for editing human embryos
Ma H, Marti-Gutierrez N, Park SW, Wu J, Lee Y, Suzuki K, et al. Correction of a pathogenic gene mutation in human embryos. Nature. 2017;548:413–9. 7
See also,
Fogarty NM, McCarthy A, Snijders KE, Powell BE, Kubikova N, Blakeley P, et al. Genome editing reveals a role for OCT4 in human embryogenesis. Nature. 2017;550:67–73.
Hello
Also the gene therapy was used for human Lung Adenocarcinoma
These links will help you :
Article Gene Therapy for Human Lung Adenocarcinoma Using a Suicide G...
https://www.ndsu.edu/pubweb/~mcclean/plsc431/students99/anderson.htm
http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0157865
Good Luck
Gene therapy has experimented in human beings long time ago. One person even died. See this 2001 paper.
Title: Death but one unintended consequence of gene-therapy trial Article Death but one unintended consequence of gene-therapy trial
From the paper:
"Jesse Gelsinger wanted to help others overcome the same metabolic disorder he had, so he agreed to enter a gene-therapy trial. A short time later, the 18-year-old American became the first person to die because of participation in gene-therapy research."
This is an interesting Timeline for gene-therapy (from New Scientists--https://www.newscientist.com/article/mg22029413-200-bubble-kid-success-puts-gene-therapy-back-on-track/)
Trials and tribulations of gene therapy:
- 1990 First approved gene therapy trial. Immune cells from 4-year-old Ashanti DeSilva are given working versions of the ADA gene to treat severe combined immunodeficiency disorder (SCID). It works, but only temporarily.
- 1992 ADA-SCID is successfully treated through gene therapy on stem cells harvested from bone marrow.
- 1999 18-year-old Jesse Gelsinger dies following an immune reaction to the virus vector used to insert the corrected gene. US Food and Drug Administration suspends several trials pending re-evaluation of ethical and procedural practices.
- 2000 Announcement that two boys in France with X-linked SCID or “bubble boy” disease have been cured using gene therapy.
- 2002 French SCID trial suspended after four children develop leukaemia as a result of the retrovirus vector.
- 2003 Chinese company Shenzhen SiBiono GeneTech gains approval for treating head and neck cancer with Gendicine, a modified adenovirus carrying a tumour-suppressor gene.
- 2003 Researchers in the US begin the first human trial using a modified lentivirus. It is a disabled HIV virus carrying a gene to inhibit replication. Trial is a success.
- 2009 Eight-year-old Corey Haas, who has a rare inherited eye disease and is almost blind, gains normal vision following gene therapy to replace a retinal pigment protein.
- 2009 Progression of the degenerative disease adrenoleukodystrophy is halted in two boys using gene therapy.
- 2010 An adult with blood disorder beta-thalassaemia no longer needs monthly blood transfusions following gene therapy to insert a corrected beta-globin gene into stem cells that make blood.
- 2011 Six people with clotting disorder haemophilia B see a reduction in symptoms after gene therapy on liver cells.
- 2012 Glybera becomes the first gene therapy drug to be approved in the West, with European approval to treat lipoprotein lipase deficiency.
- 2013 Two papers describe the treatment of children with a degenerative disorder called metachromatic leukodystrophy and immune disorder Wiskott-Aldrich syndrome using gene therapy (Science, doi.org/pnv; doi.org/ppk).
Also see this story (2013 Nov.):
Title: ‘Bubble kid’ success puts gene therapy back on track--Five children with a genetic disease that wipes out their immune system have successfully been treated with gene therapy.
Those children were born with no immune system. After gene therapy (replace a faulty gene with a normal copy), now their white blood cells almost doubled (meaning they have an immune system now).
https://www.newscientist.com/article/mg22029413-200-bubble-kid-success-puts-gene-therapy-back-on-track/ (cope and paste to veiw)
I would like to express my opinion against the notorious gene therapy.
I have studied about the biotinidase deficiency (or abnormal biotinidase gene) in vain in Japanese, although such patients are reported in USA.
We have surely found three patients of biotin deficiency (please see file; JMBT Alopecia 1). These three patients have changed glycochains of biotinidase, which give thermo-labile biotinidase causing biotin deficiency by drinking some allergy milks, which contain low level of biotin.
I am now considering that many diseases are caused by changed glycochain-structures, and are not caused by abnormal genes (HCC case: please see file; Dr. Aoyagi Fucose). Further, it seems that changed glycochain structures are induced by virus. Thus, Fucoidan or brown algae of "Mozuku" is a unique and strong drug and/or foodstuff effectively reducing the infected or hindered virus. Virus can hinder against vaccine and immunity, and I have found that vaccinia (VACV) and variola (VARV) virus are frequently found, although WHO has declared that variora virus (VARV) has been controlled (our unpublished observation and please see file; HepG2 Fucoidan).
ClinicalTrials.gov is a very useful database proveding information about gene therapy clinical trials in different countries around the world.
A piece of news from GEN (2018/1/30):
Title: Gene Therapy May Not Be a Viable Option for Many Patients --A Large Portion of Patients Have Pre-Existing Antibodies Against AAV
AAV is a common vector used to deliver gene-therapy components (in this case, CRISPR/Cas9).
Source: https://www.genengnews.com/gen-exclusives/gene-therapy-may-not-be-a-viable-option-for-many-patients/77901045?utm_medium=newsletter&utm_source=GEN+Tech+Focus&utm_content=01&utm_campaign=GEN+Tech+Focus_20180321&ajs_trait_oebid=9675B9130356B7S
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