Select a journal to view their latest articles or click here for a view at a glance of the latest gene therapy papers. 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. In systemic therapy, a therapeutic gene must be delivered efficiently to its target tissues without side effects. All successful, preclinical and clinical studies to date have utilized recombinant adenoassociated viral aav vectors for factor viii or ix hepatocyte transduction. Pdf gene therapy is the use of genes to heal diseases at the genetic. Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease and describing important advances in cell. Ethical and regulatory aspects of genome editing blood. Gene therapy clinical trials worldwide database journal of tissue. 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. Several databases pubmed, cochrane library, and web of science were searched for. The emphasis of the journal is on full research papers of any length required for concise presentation and discussion of the data. Advances and challenges for hemophilia gene therapy. The european medicines agency ema defines that a gene therapy medicinal product is a biological medicinal product which fulfils the following two characteristics. 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.
Sightrestoring therapy for the visually impaired and blind is a major unmet medical need. Human genetics presents original and timely articles on all aspects of human genetics. 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 strives to be a very diverse journal and topics in all fields will be considered for publication. Reducing risks and delays in the translation of cell and. Researchers developed the hierarchical shotgun method to achieve the human genome project.
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. Led by shashikant kulkarni, our experienced editorial board members are all active researchers in their field. This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with rpe65lca. Here you can find the latest updates in the field of genetics gene therapy and all that the various. 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. 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. The journal is adopted by the european and british societies of gene and cell therapy as their official journal. 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. The journal focus on the application of genetic syndrome, human gene. 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. Institute of scientific and technical information of china. The journal publishes indepth coverage of dna, rna, and cell therapies by delivering the latest breakthroughs in research and technologies. 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.
Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease. A randomized, doubleblind, placebocontrolled phase ii study of hepatocyte growth factor in the treatment of critical limb ischemia. 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. This is the sixth consecutive year that molecular therapys impact factor has increased. The field of gene therapy is striving more than ever to define a path to the clinic and the market. 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. 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. Updated list of high journal impact factor gene therapy journals. Asgct journal molecular therapy sees boost in impact factor. Adenoassociated virus vectors and neurological gene therapy.
Learn about approaches to and issues surrounding gene therapy. Gene therapy has strong potential for treating a variety of genetic disorders, as demonstrated in recent clinical trials. The effect of human gene therapy for rpe65 associated. Their gut microbiotas have a higher abundance of resistant starchdegrading microbes and drive higher adiposity when transferred to germfree mice. Of the various challenges involved in the process, one of the most significant is. At the forefront of medicine, gene therapy brings you the latest research into genetic and cellbased technologies. Highimpact journals are those considered to be highly influential in their respective fields. Although promising, gene therapy is currently being tested only for diseases that have no other cures soleimani et al. 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. Alzheimer disease ad is the most common neurodegenerative disorder and lacks effective diseasemodifying therapies. 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. Frontiers gene therapy using plasmid dna encoding vascular. View table of contents for the journal of gene medicine volume 22 issue 5.
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. Cell therapy manuscripts can also include application in nongenetic diseases when cells have been genetically. Human molecular genetics publishes papers of excellence, wide interest and broad significance in all aspects of human molecular genetics. 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. 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.
We injected the plasmid dna encoding two therapeutic speciesspecific growth factors. These advances raise great hope to treat devastating rare and inherited diseases as well as incurable illnesses. Several databases pubmed, cochrane library, and web of science were searched. Update on clinical gene therapy for hemophilia blood. Molecular genetics and gene therapy open access journals. Cell therapy manuscripts can also include application in diseases when cells have been genetically modified.
Nathalie cartier, md, and pierre cordelier, phdeditorialfull access. Human genetics is the study of the heriditory in humans. Human gene therapy rg journal impact rankings 2018 and 2019. Clinical development read 8 articles with impact on researchgate, the professional network for scientists. An update to date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. In 1989, the concept of human gene therapies has emerged with.
According to the journal citation report from 2011, the journals impact factor is rated at 4. Adenoassociated virus vectors for human gene therapy. 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. Human gene therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. However, in recent years gene therapy has gained more attention due. Till now human genome project sequenced thousands of human genomes. 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. Genetic syndromes and gene therapy open access journals. Nucleic acidbased therapy has become an increasingly important strategy for treating a variety of human diseases. Human gene therapy has been attempted on somatic body cells for diseases such as cystic fibrosis and cancer. Japanese science and technology agency jst journal citation reportsscience edition. 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. Citescore values are based on citation counts in a given year e.
We have developed a novel gene therapy approach for rat liver cirrhosis by muscledirected gene transfer of hepatocyte growth factor hgf. In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease. The journal publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans. 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. In press, journal preproof, available online 31 october 2019.
Cancer genetics is a part of elsevier s oncology journal network. Pdf human gene therapy the future of health care researchgate. Development and clinical translation of approved gene. British society for gene therapy is pleased to continue its partnership with mary ann. Succinct and carefully prepared papers are favoured in terms of impact as. Individuals with high amy1cn have an increased number of oral porphyromonas, which is linked to periodontitis. Gene therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. Human gene therapy vol 27, no 2 mary ann liebert, inc. Advances and challenges for hemophilia gene therapy human. 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. 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.
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. Perspectives on best practices for gene therapy programs. Human genome project started in the 1990 with the funding from nih and department of energy. The effect of human gene therapy for rpe65 associated leber. Human gene therapy vol 26, no 8 mary ann liebert, inc. The most common form of gene therapy involves using dna that encodes a functional, therapeutic gene to replace a mutated gene. Gene therapy clinical trials worldwide, provided by the journal of gene.
This contribution outlines the possibilities and limits of gene therapy in man from a medicotechnical viewpoint. Journal of genetics and gene therapy high impact factor. Gene therapy has many benefits to humans but there are also some negative aspects of gene therapy. The journal primarily focuses on publishing investigations on the molecular bases and experimental therapeutics of human diseases. 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. Updated list of high journal impact factor gene therapy. 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. To view the recent articles you need to login or create an account. Unfortunately, each one of these rare genetic diseases may require their own proprietary approach, and the impact needs to be evaluated longterm. Journal of genetics and gene therapy high impact factor journal. Human gene therapy journal british society for gene and cell.
The impact of coronavirus on the cancer gene therapy market 2020 projected to witness robust development by 2026 3w market news reports 3rd watch. These products are commonly discovered and developed in academic laboratories or small biotechnology companies with expertise in science and innovation but limited experience in bringing. 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. 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. The two years line is equivalent to journal impact factor thomson reuters metric. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide. Cell therapy manuscripts can also include application in nongenetic diseases when cells have been genetically modified.
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