Pdf human gene therapy the future of health care researchgate. Development and clinical translation of approved gene. Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease. Pdf gene therapy is the use of genes to heal diseases at the genetic. Researchers developed the hierarchical shotgun method to achieve the human genome project. The emphasis of the journal is on full research papers of any length required for concise presentation and discussion of the data. Updated list of high journal impact factor gene therapy. Reducing risks and delays in the translation of cell and. Sightrestoring therapy for the visually impaired and blind is a major unmet medical need.
Human molecular genetics publishes papers of excellence, wide interest and broad significance in all aspects of human molecular genetics. Adenoassociated virus vectors and neurological gene therapy. Perspectives on best practices for gene therapy programs. Human gene therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. Institute of scientific and technical information of china. Human gene therapy vol 26, no 8 mary ann liebert, inc. Cell therapy manuscripts can also include application in nongenetic diseases when cells have been genetically modified. This cell science journal with highest impact factor offers an open access platform to the authors in academia and industry to publish their novel research.
Gene strives to be a very diverse journal and topics in all fields will be considered for publication. In 2001, we initiated a clinical trial of nerve growth factor ngf gene therapy in ad, the first effort at gene delivery in an adult neurodegenerative disorder. The open access journal by sci forschen for molecular genetics and gene therapy issn 24714968 aims to be the single largest compendium for cutting edge research work and high quality articles on the topic of genetics gene therapy. Alzheimer disease ad is the most common neurodegenerative disorder and lacks effective diseasemodifying therapies. Citescore values are based on citation counts in a given year e. Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease and describing important advances in cell. Human gene therapy journal british society for gene and cell. The field of gene therapy is striving more than ever to define a path to the clinic and the market. Several databases pubmed, cochrane library, and web of science were searched. This contribution outlines the possibilities and limits of gene therapy in man from a medicotechnical viewpoint. Cell therapy manuscripts can also include application in nongenetic diseases when cells have been genetically. Gene therapy clinical trials worldwide database journal of tissue.
All successful, preclinical and clinical studies to date have utilized recombinant adenoassociated viral aav vectors for factor viii or ix hepatocyte transduction. Nathalie cartier, md, and pierre cordelier, phdeditorialfull access. Learn about approaches to and issues surrounding gene therapy. According to the journal citation report from 2011, the journals impact factor is rated at 4. Aav vector liver directed gene therapy in hemophilia animal models induces immune tolerance to fviii and fix through induction of factorspecific regulatory t cells treg. Gene publishes papers that focus on the regulation, expression, function and evolution of genes in all biological contexts, including all prokaryotic and eukaryotic organisms, as well as viruses. The most common form of gene therapy involves using dna that encodes a functional, therapeutic gene to replace a mutated gene. To view the recent articles you need to login or create an account. Human gene therapy rg journal impact rankings 2018 and 2019. Ethical and regulatory aspects of genome editing blood. Gene therapy has strong potential for treating a variety of genetic disorders, as demonstrated in recent clinical trials. Hemophilia provides an attractive target for gene therapy studies, due to the monogenic nature of these disorders and easily measurable endpoints factor levels and bleed rates. We injected the plasmid dna encoding two therapeutic speciesspecific growth factors.
The effect of human gene therapy for rpe65 associated. We have developed a novel gene therapy approach for rat liver cirrhosis by muscledirected gene transfer of hepatocyte growth factor hgf. Update on clinical gene therapy for hemophilia blood. Human genome project started in the 1990 with the funding from nih and department of energy.
Here you can find the latest updates in the field of genetics gene therapy and all that the various. Of the various challenges involved in the process, one of the most significant is. Introduction cell, gene, and tissuebased advanced therapeutic products, referred to in this paper as cell and gene therapy cgt products, have the potential to address many unmet medical needs. British society for gene therapy is pleased to continue its partnership with mary ann. At the forefront of medicine, gene therapy brings you the latest research into genetic and cellbased technologies. With the approval of first adenoassociated virus aavbased gene therapy drug, glybera, to treat lipoprotein lipase deficiency lpld by the european union on november 2, 2012 1, 2, human gene therapy entered a new era. In human genetics different types of fields that includes classical or formal genetics, clinical genetics, genetic counseling, cancer genetics, cytogenetics, biochemical genetics, pharmacogenetics, molecular genetics, immunogenetics, behavioral genetics, population genetics, reproductive genetics, developmental genetics, ecogenetics and. Molecular genetics and gene therapy open access journals. Select a journal to view their latest articles or click here for a view at a glance of the latest gene therapy papers. The journal focus on the application of genetic syndrome, human gene. A randomized, doubleblind, placebocontrolled phase ii study of hepatocyte growth factor in the treatment of critical limb ischemia. Updated list of high journal impact factor gene therapy journals.
Genetic syndromes and gene therapy open access journals. Cancer genetics is a part of elsevier s oncology journal network. Several databases pubmed, cochrane library, and web of science were searched for. Human gene therapy has been attempted on somatic body cells for diseases such as cystic fibrosis and cancer. There is unfortunately no scarcity of disease targets, and the grand challenge in this field has instead been the development of safe and efficient gene delivery platforms. To date, only four subjects from all those enrolled in the 9 pd gene therapy trials have died and donated their brains for study bartus et al.
The ethical issues for unintended modification of germ cells in the course of an in vivo geneediting therapy are not different from those for therapeutic approaches that would use gene addition. Gene therapy has many benefits to humans but there are also some negative aspects of gene therapy. The journal publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans. Ocular gene therapy is a rational choice for restoring vision or preventing the loss of vision because most blinding diseases originate in cellular components of the eye, a compartment that is optimally suited for the delivery of genes, and many of these diseases have a genetic origin or genetic. It has been a long march from the first discovery of the aav in the 1960s, to the final approval of the first aavbased gene therapy drug. Nucleic acidbased therapy has become an increasingly important strategy for treating a variety of human diseases. Unfortunately, each one of these rare genetic diseases may require their own proprietary approach, and the impact needs to be evaluated longterm. The effect of human gene therapy for rpe65 associated leber. This is the sixth consecutive year that molecular therapys impact factor has increased. It has been a founding tenet of the field of human gene therapy that the human germ line in patients should not be perturbed genetically. Other forms involve directly correcting a mutation, or using dna that encodes a therapeutic protein drug rather than a natural human gene to provide treatment. These advances raise great hope to treat devastating rare and inherited diseases as well as incurable illnesses.
This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with rpe65lca. However, in recent years gene therapy has gained more attention due. Stem cell therapy is already being used in specific contexts, and its efficacy and safety being evaluated, and gene therapy trials in mouse models show clear benefits. Rpe65associated lca rpe65lca is an inherited retinal degeneration caused by the mutations of rpe65 gene and gene therapy has been developed to be a promising treatment. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. Dec 20, 2017 for the past 25 years, the development of gene therapy for hemophilia has fueled technological innovations and led to emerging insights that benefited the field at large. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide. In press, journal preproof, available online 31 october 2019.
Human genetics is the study of the heriditory in humans. An update to date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Dec 15, 20 the impact of coronavirus on the cancer gene therapy market 2020 projected to witness robust development by 2026 3w market news reports 3rd watch. The aim of cancer genetics is to publish high quality scientific papers on the cellular, genetic and molecular aspects of cancer, including cancer predisposition and. Gene therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. Oct 10, 2017 in this clinical study, for the first time we used the direct gene therapy to restore severe injuries of the suspensory ligament branch and superficial digital flexor tendon in horses equus caballus. For the past 25 years, the development of gene therapy for hemophilia has fueled technological innovations and led to emerging insights that benefited the field at large. The european medicines agency ema defines that a gene therapy medicinal product is a biological medicinal product which fulfils the following two characteristics. Mar 01, 2015 regulatory approval and oversight of gene therapy projects is also adapting to new technologies and has led to renewed optimism for gene therapy. Gene delivery to the cns involves both considerable promise and unique challenges, and better aav vectors are thus needed to translate cns gene therapy approaches to the clinic. Although promising, gene therapy is currently being tested only for diseases that have no other cures soleimani et al. In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease. Gene therapy has the potential to be able to cure genetic diseases such as cystic fibrosis, hemophilia, muscular dystrophy and sickle cell anemia, all of which cause by the mutation of a single gene 26. Advances and challenges for hemophilia gene therapy human.
Their gut microbiotas have a higher abundance of resistant starchdegrading microbes and drive higher adiposity when transferred to germfree mice. Succinct and carefully prepared papers are favoured in terms of impact as. Clinical development read 8 articles with impact on researchgate, the professional network for scientists. Here you can find the latest updates in the field of genetics gene therapy and all that the various researchers in this field are working on. Till now human genome project sequenced thousands of human genomes. In 1989, the concept of human gene therapies has emerged with. The early promises and subsequent disappointments during the first decade of human gene therapy. The journal is adopted by the european and british societies of gene and cell therapy as their official journal.
Led by shashikant kulkarni, our experienced editorial board members are all active researchers in their field. It was established in 1976 and is published by elsevier according to the journal citation reports, the journal has a 2015 impact factor of 2. Gene therapy provides new options for medicine, and implies new responsibilities for making decisions relating to the limitations which reason and morality impose upon manipulation of the human genome for the benefit both of patients and of society. Asgct journal molecular therapy sees boost in impact factor. Selection of scientific journals involved in gene therapy laboratory and clinical research. Journal of genetics and gene therapy high impact factor journal. Individuals with high amy1cn have an increased number of oral porphyromonas, which is linked to periodontitis. The application of recombinant dna technology and gene cloning which started in the 1980s and the resulting increase in genomics data during the 1990s have contributed to define some diseasecausing genetic factors and to explore the potential of new therapies based on engineered genes and. Human genetics presents original and timely articles on all aspects of human genetics. The journal primarily focuses on publishing investigations on the molecular bases and experimental therapeutics of human diseases. Human gene therapy vol 27, no 2 mary ann liebert, inc. Journal of genetics and gene therapy high impact factor. View table of contents for the journal of gene medicine volume 22 issue 5. Japanese science and technology agency jst journal citation reportsscience edition.
In systemic therapy, a therapeutic gene must be delivered efficiently to its target tissues without side effects. Highimpact journals are those considered to be highly influential in their respective fields. Advances and challenges for hemophilia gene therapy. The journal publishes indepth coverage of dna, rna, and cell therapies by delivering the latest breakthroughs in research and technologies.
Adenoassociated virus vectors for human gene therapy. Frontiers gene therapy using plasmid dna encoding vascular. The impact of coronavirus on the cancer gene therapy market 2020 projected to witness robust development by 2026 3w market news reports 3rd watch. Gene therapy clinical trials worldwide, provided by the journal of gene. Jul 23, 2019 hemophilia provides an attractive target for gene therapy studies, due to the monogenic nature of these disorders and easily measurable endpoints factor levels and bleed rates.
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