Please forward genetic engineering research paper error screen to sharedip-16015312831. Gene therapy using an adenovirus vector.
In some cases, the adenovirus will insert the new gene into a cell. If the treatment is successful, the new gene will make a functional protein to treat a disease. Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I. 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. Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.
Martin Cline on 10 July 1980. The first somatic treatment that produced a permanent genetic change was performed in 1993. Gene therapy is a way to fix a genetic problem at its source. The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations. The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a «vector», which carries the molecule inside cells.
Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers’ attention, although as of 2014, it was still largely an experimental technique. The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine.
DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein. Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome. Generally, efforts focused on administering a gene that causes a needed protein to be expressed.
More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. Gene editing is a potential approach to alter the human genome to treat genetic diseases, viral diseases, and cancer. As of 2016 these approaches were still years from being medicine. A duplex of crRNA and tracrRNA acts as guide RNA to introduce a specifically located gene modification based on the RNA 5’ upstream of the crRNA. Over 600 clinical trials utilizing SCGT are underway in the US. Modifying a germ cell causes all the organism’s cells to contain the modified gene.
The delivery of DNA into cells can be accomplished by multiple methods. In order to replicate, viruses introduce their genetic material into the host cell, tricking the host’s cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell. Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Before gene therapy can become a permanent cure for a condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be stable.