X-SCID Gene Therapy Essay

Frank Emmrich is the director of the Fraunhofer IZI and a professor at the University of Leipzig, where he has headed the Institute of Clinical Immunology and Transfusion Medicine since 1994. This dual position enables the efficient sharing of experience, not to mention the optimal supervision of undergraduate and doctoral dissertations, and provides an excellent basis for cooperation. Both a doctor and an immunologist, Prof. Emmrich spent 13 years as both a researcher and department head at Max Planck Institutes in Freiburg and Erlangen. Over seven of these years he was a professor at the Friedrich Alexander University in Erlangen-Nuremberg. From its founding eight years ago up to this year he also was director of the BMBF funded institute of Translational Regenerative Medicine, the TRM, at Leipzig. Since 2008 he is also a member of the German Ethics Council and additionally member or chairman in a multitude of scientific organisations such as the German Stem Cell Network. He first came into contact with Dr. Burkhardt during her dissertation thesis on genetics of autoimmune diseases and since 2011 advised and accompanied Dr. Burkhardt’s BMBF funded project as her mentor at the Translational Center for Regenerative Medicine (TRM) Leipzig. Currently, more than 200 peer-reviewed scientific publications are listed by Prof. Emmrich in addition to several patent applications. In addition to supervision provided by Prof. Emmrich, Christopher Oelkrug, Head of Immunotherapy/Oncology Unit at the Fraunhofer IZI, will accompany the proposed project. He published over 20 peer-reviewed research articles with a focus on the development and testing of novel therapeutic strategies with the aid of innovative tumour models for the treatment of cancer patients and the enhancement of therapeutic cancer vaccines through different administration strategies. Thus, his research and expertise complements the purposed of this

In 1995, Timothy Ray Brown, more commonly known as the Berlin patient, was diagnosed with the HI Virus and like many patients, he began using HAART and expected a more normal life. However, in 2006 he was diagnosed with acute myeloid leukemia, which is treated in Europe with a stem cell transplant. In 2008, Mr. Brown agreed to a risky stem cell transplant, knowing that only %50 of patients survives. With the odds against him, he also agreed to a special treatment that would inject stem cells from a donor that had a natural immunity to the HI Virus, in an attempt to cure both the cancer and the virus at one time. Against all odds Mr. Brown survived the treatment, and both ailments were cured. Regardless of how extraordinary this cure was to find, it is not a broad case cure. The ability to match donor’s chromosomes for stem cell transplants is currently a daunting task; trying to find donors that contain the mutated gene that has a natural ability to resist the HI Virus would match less than .01% of the infected population. Although we have found this small victory, we must continue the search for a

Severe combined immunodeficiency (SCID) is a term that describes a broad range of genetic disorders that cause profound defects in the humoral and cellular immune system in humans. Patients that have been diagnosed with SCID usually record a low T cell and natural killer (NK) count in their body. On top of this, patients usually have a high number of B cells, but are usually deficient in specific antibody responses (Allenspach, et al., 2003). SCID can be treated through a bone marrow transplantation (BMT) operation and gene therapy. Patients who do not get treatment usually die in infancy due to infections caused by opportunistic pathogens that are able to thrive in the body due to a weakened immune system. There are two forms of SCID,

Demolishing genetic diseases from the human race has been and always will be a key role in science. However, there comes a point in time when moral standards have interjected the betterment of cleansing genetic diseases. One of the many branches of science that has the complete capability to exterminate our destiny of any genetic diseases, gene therapy, is being silenced due to the curse of society’s moral standards. There is a very narrow line in what is not morally correct and ethical to standards of people; causing the advancement of procedures like gene therapy to be narrow. The only explanation to gene therapy awaits on what we, as a community, can bring about for the future generations, but this is

While the majority of stem cell transplants go as planned, some patients develop serious or life threatening complications such as organ damage, infections, and even death. The procedure itself is relatively painless since the stem cells are infused through an intravenous (IV) or a central line. After the procedure, the stem cells will start making healthy cells in the blood, which would hopefully force the psoriasis into remission. It has been stated that, “Allogeneic BMT has led to durable remission of autoimmune disease in most patients, but the number of reports is small and the potential risks of the procedure are high.”, therefore the potentially deadly risk associated with stem cell transplants needs to be weighed against the benefit of extended psoriasis remission (remission of two or more years) (Adkins,

According to Rosenberg (1988), the field of immunotherapy has become an area of significant and increasing interest within the field of medical science, prompted by the recent advances in knowledge of both biotechnology and cellular immunology. The field of immunology has produced revolutionary developments such as vaccines, which have prevented and decreased the occurrence of numerous infectious diseases over the past two centuries (Oviedo-Orta et al. 2013). Vaccine development has in turn combined immunology and drug design, resulting in a great deal of innovation over the last four decades endeavouring to expand the benefit of immunology past vaccines against foreign pathogens, and towards the treatment of chronic debilitating diseases.

Surprisingly,scientists confirmed that therapeutic cloning or SCNT has been efficiently used to treat infection in similar subjects from whom the unique cells were derived. While this contemporary work is in animals, it ought to have future implications as this approach may be a conceivable method to diminish transplant dismissal and improve recuperation in other ailments and in other organ systems. Like most things, there are pros and cons to cloning. Therapeutic cloning would decrease the conceivable outcomes of an organ being rejected amid transplantation. Since the organs made would be of the individual's own cells, there would be an abundantly lessened danger of organ rejection once a transplant method was effectively finished. Since the cells would be created in light of a man's own particular interesting hereditary qualities and DNA, there would never again be a requirement for a second surgery of a giver all together for a transplant to be fruitful. But on the other end, some cells change and cause tumors in patients. Keeping in mind the end goal to cure infection, a large number of eggs are

The vaccine given to the children contained live but sufficiently weakened or attenuated viruses to stimulate immune response. The capacity to stimulate immune response was from the ability of human immune system to distinguish non-self cells that carry epitopes. First, the immune B cells with antibody would bind and engulf to process the attenuated virus. Then, helper T cells would bind to the processed virus and form plasma cell. The plasma cells then stimulate the production of more antibodies to mark virus for destruction. After destruction, some of the B cells and helper T cells left form memory cells to produce immediate response to 2nd attack of the same viruses.

In 1990 the first gene therapy procedure gained approval and a four-year-old girl with SCIDs disease was finally able to fight off a simple cold. She is now able to live a normal life with the help of continued treatment of gene therapy. Although gene therapy is an innovative and ideally favorable procedure when it comes to treating diseases, cancer, or inherited disorders, it is still a delicate procedure and is continuously studied to insure it is not only effective but also and most importantly safe. In order to insure the safety of patients a gene therapy drug must get the approval of the FDA before becoming available, as every drug must do. Since gene therapy is still very experimental; there are only a few gene therapy drugs that have been approved by the FDA to treat patients. However, on August 30th, 2017 history was made when the FDA approved Kymriah, the first CAR-T cell therapy drug to be available in the United States. Kymriah is a one-time treatment for patients with B-cell acute lymphoblastic leukemia, it was developed through the research collaboration of Novartis and the University of Pennsylvania, and it is manufactured for each individual patient. Kymriah’s only drawback would be the price tag of $475,000 for a course of treatment, however Novartis plans to wave the fee for patients where Kymriah is not successful.

A particularly inspiring case is that of Layla Richards, a one-year old baby diagnosed with infant acute lymphoblastic leukaemia (ALL) at merely three months of age. After several rounds of chemotherapy and a bone marrow transplant failed to free Layla of her disease, she was given months to live. Her doctors suggested palliative care. Her parents, refusing to give up on their daughter begged for the doctors to try anything. The ‘anything’ turned out to be modified T Cells.

Their research had been developed on a basis of an observation of an HIV positive patient, who was undergoing treatment for leukaemia via the bone marrow transplantation method. This method is known to introduce new bone marrow into the immune system of the effected patient to revive the patient’s “new” immune system. The bone marrow that this patient had received was genetically mutated of its ∆32 CCR5 gene that resulted in the inactivation of the gene.

Immunotherapy is a form of medical treatment intended to stimulate or restore the ability of the immune system to fight infection and disease. This can be by inducing, enhancing, or suppressing an immune response. Immunotherapies designed to elicit or amplify an immune response are classified as activation immunotherapies, while those that reduce or suppress immune response are suppression immunotherapies. Active immunotherapy has been effective against agents that normally cause acute self-limiting infectious disease. However, a more effective immunotherapy for chronic infectious diseases or cancer requires the use of appropriate target antigens; the

Cancer occurrs by the production of multiple mutations in a single cell that causes it to proliferate out of control. Cancer cells often different from their normal neighbors by a host of specific phenotypic changes, such as rapid division rate, invasion of new cellular territories, high metabolic rate, and altered shape. Some of those mutations may be transmitted from the parents through the germ line. Others arise de novo in the somatic cell lineage of a particular cell. Cancer-promoting mutations can be identified in a variety of ways. They can be cloned and studied to learn how they can be controlled.

There are three techniques that researchers are working on. The first and most common is ex vivo ( or "outside the living body") therapy. The defective cells are removed from the patient and replaced with the normal DNA before returning to the body. This therapy targets the blood cells because many genetic defects alter the functioning of one type of these cells or another. But since blood cells have limited life spans follow-up treatments are required. Future efforts will most likely target stem cells of the bone marrow. Stem cells are ideal for gene therapy because they appear to be immortal. Researchers have obtained stem cells from human bone marrow, but they are having difficulties getting genes into the cell as well as inducing the cells to produce many new blood cells (Anderson, 1995).

The word science comes from the Latin word "to know" (Science Made Simple, 2014). In