Gene Therapy

Here you will find some usefull information about gene therapy

What is Gene Therapy?

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

-Replacing a mutated gene that causes disease with a healthy copy of the gene.

-Inactivating, or “knocking out,” a mutated gene that is functioning improperly.

-Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures. (Source: NIH).


History of Gene Therapy


-1972, Friedmann and Roblin authored a paper in Science titled "Gene therapy for human genetic disease?" Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects


-1984, a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.


-1990, The first approved gene therapy clinical research in the US took place
-1992, Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.
-Jesse Gelsinger's death in 1999 impeded gene therapy research in the US. As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices
-2002, first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID) was performed


-2002, The modified cancer gene therapy strategy of antisense IGF-I RNA has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary
-2003, The first insertion of genes into the brain by using liposomes coated in a polymer called polyethylene glycol, which, unlike viral vectors, are small enough to cross the blood–brain barrier
-2006, researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system
-2007, the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson,
-2008, The first gene therapy results published of Leber's congenital amaurosis, an inherited blinding disease caused by mutations in the RPE65 gene, delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In all three clinical trials, patients recovered functional vision without apparent side-effects
-2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.


-2010, 18-year-old male patient in France with beta-thalassemia major had been successfully treated with a lentiviral vector to transduce the human ß-globin gene into purified blood and marrow cells
-2011, In 2007 and 2008, a man (Timothy Ray Brown) was cured of HIV by repeated hematopoietic stem cell transplantation with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011
*Genetically modified T cells to attack cells that expressed the CD19 protein to fight the chronic lymphocytic leukemia (CLL)
*Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF. Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF
-2012, The FDA approved Phase 1 clinical trials on thalassemia major patients in the US
*The European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis. The recommendation was endorsed by the European Commission in November 2012 and commercial rollout began in late -2014, Clinical trials of gene therapy for sickle cell disease were started
-2015, LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease
-Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]).
- Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis

2017 and later

-Kite’s Yescarta™ (Axicabtagene Ciloleucel) became first CAR T Therapy approved by the FDA for the treatment of adult patients with Relapsed or Refractory Large B-Cell Lymphoma after two or more lines of systemic therapy
-The FDA approved KYMRIAH® (Tisagenlecleucel), an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia, for acute lymphoblastic leukemia
-The results of using an adeno-associated virus with blood clotting factor VIII to treat nine haemophilia A patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels
-The FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber's congenital amaurosis
-Researchers at Stanford University School of Medicine are also planning to begin sickle-cell CRISPR therapy at their Center for Curative and Definitive Medicine, and intend to seek permission from the FDA in 2018.


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