History of gene therapy thomas wirth a, nigel parker b, seppo ylaherttuala a,c,d. There are many techniques of gene therapy, all of them still in experimental stages. Gene therapy had its start in the 1970s, including the creation of a regulatory body for gene therapy, the recombinant dna advisory committee rac. The genetic diseases in which a single known gene does not function properly, such as sickle cell anaemia, thalassaemia and leschnyhan syndrome, are most suitable to be treated with the gene therapy. Examples for these are the positive recommendation for a gene therapy product glybera by the ema for approval in the european union and the positive trials for the treatment of ada deficiency, scidx1 and adrenoleukodystrophy. But should germline genetic research still be pursued, and if so, why. First successful attempt of gene therapy first attempt of gene therapy on a human jan 1, 1990. Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase.
Although still in the early stages, gene therapy is a technique that treats or prevents disease through the use of genes. Gene therapy tools and potential applications intechopen. Gene therapy can improve patients compliance and decrease cost of therapy on long term bases. 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. It is a technique for correcting defective genes that are responsible for disease development. Emphasis is placed on the molecular bases of drug action, both applied and experimental. The hgp is like translating an entire library of books written in a foreign language the genetic code. Lewis is a fantastic storyteller and teacher, making the very technical aspects of the biology involved easy to understand for laypeople. Scientist and clinicians working in the gene therapy field have encountered many problems in the past that are now starting to be solved. In its current available form, gene therapy gt is an experimental therapy that aims at replacing the defective gene with a copy of the normal gene. Pbss documentary the gene doctors arrives amid a gene. Gene therapy applications the pharmaceutical journal. This perspective article argues that it should, but only if it is conducted under a transparent and enforceable ethicalregulatory framework.
Gene therapy has since been used experimentally to treat a number of conditions, including advanced metastatic melanoma, a myeloid disorder, and a rare hereditary condition that leads to severely. The science behind this new drug is groundbreaking. First attempt of gene therapy on a human jan 1, 1990. This type of genetic disorder is caused by both mutations in multiple genes and environmental. The announcement of an experiment on human embryos using germline genetic engineering has been met with near universal condemnation on ethical and scientific grounds. The treatment by the product glybera uses a virus to infect muscle cells with a working copy of the gene. The concepts of gene therapy arose initially during the 1960s and early 1970s whilst the development of genetically marked cells lines and the clarification of mechanisms of cell. The development of safer and more efficient gene transfer vectors and the advances on the cell therapy field have open new opportunities to tackle different diseases.
Gene therapy is a strategy used to treat disease by correcting defective genes or modifying how genes they are expressed. Single gene disorders are generally inherited autosomal dominantly shown below in blue figure, autosomal recessively shown below in pink figure, or through xlinked inheritance defective gene on the xlinked sex chromosome. Gene therapy office of history, national institutes of. Gene therapy is the process of replacing a defective gene inside a patients dna with a working gene that will produce the correct gene products.
The first, called the ex vivo technique, involves surgically removing cells from the patients, injecting or splicing the new dna. The concepts of gene therapy arose initially during the 1960s and early 1970s whilst the development of genetically marked cells lines and the clarification of mechanisms of cell transformation by. Gene therapy or the concept of treating an inherited disease with no cure by correcting or replacing a defective gene within the affected cells has long captivated scientists, clinicians and the. Conducting a clinical trial poses a unique set of challenges that must be addressed to ensure the. Gene therapy simple english wikipedia, the free encyclopedia. Oct 19, 2017 pbss documentary the gene doctors arrives amid a gene therapy boom. Latest clinical gene therapy trails at clinicaltrails.
Developments and future perspectives by chunsheng kang. Gene therapy studies updated or received in the last 30 days at clinicaltrials. History of gene therapy timeline timetoast timelines. Gene editinga regrettable history of dubious ethical conduct. Cells from individual patients were removed and treated with the gene therapy before being infused back into the patient ie, ex vivogene therapy with autologous haematopoietic progenitor cells. French anderson and michael blaese in the national heart, lung, and blood institute and the national cancer institute worked together to show that cells from patients with ada deficiency can be corrected in tissue culture. Criteria for assessing applicability to human germline gene therapy an ideal gene transfer system in the context of human germline gene therapy would have the following features. Gene therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease.
Aug 10, 20 despite the setbacks gene therapy has faced, success stories have increasingly emerged. Discovered that viruses can be used to transfer genes jan 1, 1970. The european commission has given glybera marketing authorisation, which means it can be sold throughout the eu. Isbn 9789533076171, pdf isbn 9789535164418, published 20110622. Gene therapy is the insertion, alteration, or removal of genes within an individuals cells and biological tissues to treat diseases. Translating gene therapy to the clinic, edited by dr. Timelinemilestones in gene therapy 2 min read april 27 reuters gene therapy is making a comeback, with a flood deals, stock market debuts and increased investment by big pharmaceutical companies. Jeffrey laurence and michael franklin, follows the recent, muchlauded special issue of translational research in emphasizing clinical milestones and critical barriers to further progress in the clinic. Translating gene therapy to the clinic 1st edition. The gene therapy field is living exciting times after more than 20 years of poor results. Apr 27, 2015 timelinemilestones in gene therapy 2 min read april 27 reuters gene therapy is making a comeback, with a flood deals, stock market debuts and increased investment by big pharmaceutical companies.
Volume 40, genetherapy, features important new research on gene transfers and therapy in the herpes simplex virus, antitumor immunity, steroid receptors, cystic fibroses, and more. A brief history of gene therapy georgetown university. The future successes of gene therapy will be built on the improvements in other fields, such as medical devices, cell therapies, protein therapies and nanoparticle technologies. But with the librarys information, researchers can investigate just what each gene is responsible for in the human body, and how mutations to genes cause genetic diseases.
Two methods are available for inserting genetic material into human chromosomes. The aim of this book is to cover key aspects of existing problems in the field of development and future perspectives in gene therapy. The concept of gene therapy arose during the 1960s and 1970s and is still in its infancy, meaning there is a paucity of reliable, longterm data on the safety and efficacy of this therapy. Learn about approaches to and issues surrounding gene therapy. Gene therapy to some could be the most important medical breakthroughs in a long time and can revolutionise medical practices. Gene therapy can be focused to a specific cell type to avoid potentially toxic systemic effects. Aug 23, 2018 gene therapy is a strategy used to treat disease by correcting defective genes or modifying how genes they are expressed. Gene therapy has only recently begun to make serious progress, beginning with two approved gene therapy trials in the united states in late 1990. Types of gene therapy 2,3 gene therapy can be targeted to somatic body or germ egg and sperm cells. In the medicine field gene therapy also called human gene transfer is the therapeutic delivery of nucleic acid into a patients cells as a drug to treat disease.
Gene therapy means putting in a working gene to a person who has a damaged gene. The techniques used involve administrating a specific dna or rna sequence. This allows the cell to start producing normal protein, which is supposed to cure the disease. Its field has much potential and to reduce risks and possible loss of life, animal testing is also an option. This comprehensive text provides a background for understanding the techniques involved. An intimate history from the pulitzer prizewinning author of the emperor of all maladiesa fascinating history of the gene and a magisterial account of how human minds have laboriously, ingeniously picked apart what makes us tick elle. The concepts of gene therapy arose initially during the 1960s and early 1970s whilst the development of genetically marked cells lines and the clarification. Ii study of gene therapy has been undertaken in 74 patients with hiv1 infection.
The death of an 18yearold man participating in a gene therapy trial. The science, the history and the future of gene therapy. This difference is of importance, since current legislation allows gene therapy only on somatic cells. Ricki lewis takes you through the good, bad and ugly history of gene therapy by telling the stories of the individuals who have been treated for various inherited diseases using this technology. Gene therapy was first used in humans in 1990 to treat a child with adenosine deaminase deficiency, a rare hereditary immune disorder see immunity. Another reason to insist upon the creation of a serious ethical framework for guiding germline research is the history of dubious behavior with respect to pioneering gene editing. Discover book depositorys huge selection of gene therapy books online. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Key features it provides an excellent overview on a series of topics on gene therapy for the serious investigator.
The two basic methods are called in vivo and ex vivo gene therapy. Culver, anderson, and blaese with gene therapy patients. The concept of gene therapy was first introduced in 1972, and the first trial in a human patient occurred nearly two decades later in 1990. In addition to tracing the history of sparks treatment, the film brings attention to gene therapies for cystic. Kenneth culver, novarti pharmaceuticals corporation. Gene therapy is emerging as a new class of therapeutics for the treatment of inherited and acquired diseases.
Getting serious about the challenge of regulating germline. Virtanen institute, biotechnology and molecular medicine unit, univ. Investigators are advancing with cautious optimism that effective, durable, and safe therapies will provide benefit to patientsnot only those with single gene disorders but those with complex acquired diseases as well. Zolgensma, a gene therapy for spinal muscular atrophy, was approved by the fda in 2019. Gene therapy office of history, national institutes of health. Despite the setbacks gene therapy has faced, success stories have increasingly emerged. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the. Only open studies, recruiting or not yet recruiting, are shown found by a search using the keyword. It has finally happened, we now have a gene therapy approved in the us with a pricetag over 1 millionactually, well over 1 million.
Thus it may be used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favourably modify the clinical course of a condition. Gene therapy a brief overview of the past, present, and future roland scollay genteric, alameda, california 94501, usa abstract. History of gene therapy timeline created by pentakillace. The european medicines agency ema defines that a gene therapy medicinal product is a biological medicinal product which fulfils the following two characteristics. Currently, gene therapy refers to the transfer of a gene that encodes a functional protein into a cell or the transfer of an entity that will alter the expression of an endogenous gene in a cell. Gene therapy gene therapy is a methodology for correcting defective genes responsible for disease development. The first suitable retroviral vector for gene therapy was developed by richard mulligan, a researcher at massachusetts institute of technology and former doctoral student of paul berg, a key pioneer in. The first retroviralmediated gene therapy clinical trials started in 1990, and the first stem cell gene therapy clinical trial ada deficiency, also retrovirally mediated, was initiated in 1994. Pbss documentary the gene doctors arrives amid a gene therapy boom. Sid mukherjee has the uncanny ability to bring together. However, poor cellular uptake and instability of dna in the physiological milieu limits its therapeutic potential, hence a vector which can protect and efficiently transport dna to the target cells must be developed. Singlegene disorders are generally inherited autosomal dominantly shown below in blue figure, autosomal recessively shown below in pink figure, or through xlinked inheritance defective gene on the xlinked sex chromosome. History, vectors, technologies and application article pdf available in world journal of pharmacy and pharmaceutical sciences 510 january 2018 with,373 reads.
List of books and articles about gene therapy online. Jan 18, 2014 gene therapy had its start in the 1970s, including the creation of a regulatory body for gene therapy, the recombinant dna advisory committee rac. Human gene therapy has been attempted on somatic body cells for diseases such as cystic fibrosis and cancer. The european commission has approved this method for one particular treatment.
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