Institute of scientific and technical information of china. Selection of scientific journals involved in gene therapy laboratory and clinical research. This contribution outlines the possibilities and limits of gene therapy in man from a medicotechnical viewpoint. Updated list of high journal impact factor gene therapy journals. A randomized, doubleblind, placebocontrolled phase ii study of hepatocyte growth factor in the treatment of critical limb ischemia.
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. Till now human genome project sequenced thousands of human genomes. 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. 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. Gene therapy has strong potential for treating a variety of genetic disorders, as demonstrated in recent clinical trials. Alzheimer disease ad is the most common neurodegenerative disorder and lacks effective diseasemodifying therapies. The journal focus on the application of genetic syndrome, human gene. Reducing risks and delays in the translation of cell and.
Citescore values are based on citation counts in a given year e. Japanese science and technology agency jst journal citation reportsscience edition. In press, journal preproof, available online 31 october 2019. Gene therapy clinical trials worldwide database journal of tissue.
Researchers developed the hierarchical shotgun method to achieve the human genome project. Gene therapy clinical trials worldwide, provided by the journal of gene. 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. Although promising, gene therapy is currently being tested only for diseases that have no other cures soleimani et al. In systemic therapy, a therapeutic gene must be delivered efficiently to its target tissues without side effects. Nathalie cartier, md, and pierre cordelier, phdeditorialfull access. The journal is adopted by the european and british societies of gene and cell therapy as their official journal. Pdf gene therapy is the use of genes to heal diseases at the genetic. Perspectives on best practices for gene therapy programs. 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. 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.
Journal of genetics and gene therapy high impact factor journal. The two years line is equivalent to journal impact factor thomson reuters metric. However, in recent years gene therapy has gained more attention due. Human genetics presents original and timely articles on all aspects of human genetics. Human genetics is the study of the heriditory in 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. Asgct journal molecular therapy sees boost in impact factor. Development and clinical translation of approved gene. Studies evaluating the potency of experimental cell and gene therapies in clinically relevant animal models of disease. The field of gene therapy is striving more than ever to define a path to the clinic and the market.
Nucleic acidbased therapy has become an increasingly important strategy for treating a variety of human diseases. 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. The european medicines agency ema defines that a gene therapy medicinal product is a biological medicinal product which fulfils the following two characteristics. These advances raise great hope to treat devastating rare and inherited diseases as well as incurable illnesses. 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. Human gene therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. Advances and challenges for hemophilia gene therapy. 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.
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. Individuals with high amy1cn have an increased number of oral porphyromonas, which is linked to periodontitis. Ethical and regulatory aspects of genome editing blood. 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. This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with rpe65lca. 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. View table of contents for the journal of gene medicine volume 22 issue 5. Adenoassociated virus vectors and neurological gene therapy. 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.
At the forefront of medicine, gene therapy brings you the latest research into genetic and cellbased technologies. 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. 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. Adenoassociated virus vectors for human gene therapy. 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. To view the recent articles you need to login or create an account. 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. We injected the plasmid dna encoding two therapeutic speciesspecific growth factors. 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. We have developed a novel gene therapy approach for rat liver cirrhosis by muscledirected gene transfer of hepatocyte growth factor hgf. 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 most common form of gene therapy involves using dna that encodes a functional, therapeutic gene to replace a mutated gene. 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. Gene therapy has many benefits to humans but there are also some negative aspects of gene therapy.
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. Unfortunately, each one of these rare genetic diseases may require their own proprietary approach, and the impact needs to be evaluated longterm. 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. In gene therapy, a normal gene is inserted into the genome to replace an abnormal gene responsible for causing a certain disease. Genetic syndromes and gene therapy open access journals. Clinical development read 8 articles with impact on researchgate, the professional network for scientists. Highimpact journals are those considered to be highly influential in their respective fields. Several databases pubmed, cochrane library, and web of science were searched for. Sightrestoring therapy for the visually impaired and blind is a major unmet medical need. Of the various challenges involved in the process, one of the most significant is.
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. Cell therapy manuscripts can also include application in nongenetic diseases when cells have been genetically modified. The effect of human gene therapy for rpe65 associated leber. Human gene therapy vol 27, no 2 mary ann liebert, inc. The effect of human gene therapy for rpe65 associated. According to the journal citation report from 2011, the journals impact factor is rated at 4. The journal primarily focuses on publishing investigations on the molecular bases and experimental therapeutics of human diseases. The journal publishes scientific papers on original investigations into the transfer and expression of genes in mammals, including humans. Select a journal to view their latest articles or click here for a view at a glance of the latest gene therapy papers. Human genome project started in the 1990 with the funding from nih and department of energy. Advances and challenges for hemophilia gene therapy human. Updated list of high journal impact factor gene therapy. 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.
Frontiers gene therapy using plasmid dna encoding vascular. In 1989, the concept of human gene therapies has emerged with. An update to date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Led by shashikant kulkarni, our experienced editorial board members are all active researchers in their field. All successful, preclinical and clinical studies to date have utilized recombinant adenoassociated viral aav vectors for factor viii or ix hepatocyte transduction. 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. The impact of coronavirus on the cancer gene therapy market 2020 projected to witness robust development by 2026 3w market news reports 3rd watch. Update on clinical gene therapy for hemophilia blood. Several databases pubmed, cochrane library, and web of science were searched. 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.
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. Human gene therapy journal british society for gene and cell. Succinct and carefully prepared papers are favoured in terms of impact as. Journal of genetics and gene therapy high impact factor. Gene strives to be a very diverse journal and topics in all fields will be considered for publication. British society for gene therapy is pleased to continue its partnership with mary ann.
Cell therapy manuscripts can also include application in diseases when cells have been genetically modified. 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. Human gene therapy has been attempted on somatic body cells for diseases such as cystic fibrosis and cancer. 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. Learn about approaches to and issues surrounding gene therapy. The emphasis of the journal is on full research papers of any length required for concise presentation and discussion of the data. Their gut microbiotas have a higher abundance of resistant starchdegrading microbes and drive higher adiposity when transferred to germfree mice. Human gene therapy rg journal impact rankings 2018 and 2019. Molecular genetics and gene therapy open access journals. Gene therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. Twenty gene therapy products have already been approved and over two thousand human gene therapy clinical trials have been reported worldwide. Human gene therapy vol 26, no 8 mary ann liebert, inc. Cancer genetics is a part of elsevier s oncology journal network.
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 early promises and subsequent disappointments during the first decade of human gene therapy. This is the sixth consecutive year that molecular therapys impact factor has increased. 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. Here you can find the latest updates in the field of genetics gene therapy and all that the various.
89 200 55 645 1368 1502 208 285 635 918 1028 22 628 303 131 1112 464 956 1370 181 318 958 577 1378 1353 1137 1379 321 258 230 569 230