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022 _a1476-5462
024 7 _a10.1038/41434.1476-5462
_2doi
210 1 0 _aGene Ther
245 1 0 _aGene Therapy
_h[electronic resource] /
_cedited by Janine Scholefield.
264 1 _aLondon :
_bNature Publishing Group UK :
_bImprint: Springer Nature.
300 _bonline resource.
520 _aGene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine. Gene Therapy covers all aspects of gene therapy as applied to human disease, including: preliminary studies which reveal novel genetic targets or strategies that improve a disease phenotype (Note: studies showing the mechanistic role of genetic elements alone, will be considered if significant therapeutic benefit or novelty is shown) the development of novel techniques for gene therapy research and improvements on established tools preclinical animal model systems and validation studies gene-based vaccine development and applications genetically engineered cellular models that can advance the validation of novel therapeutic targets, and drug discovery especially those using high-throughput applications and 3D modelling cell-based therapies including all aspects of stem cells and genetically modified cellular approaches (especially CRISPR and other gene editing strategies), including biobanking studies clinical trial reports which have significant impact for the field novel genetic biomarkers of disease susceptibility or disease progression with extensive scientific validation Industry sector pipeline profiles or commercial implications of gene therapies Regulatory landscapes and legislative issues pertaining to different global regions.
650 0 _aMedicine—Research.
650 0 _aBiology—Research.
650 0 _aMedical genetics.
650 0 _aCytology.
650 0 _aNanotechnology.
650 0 _aMedical genetics.
650 0 _aMolecular genetics.
650 1 4 _aBiomedical Research.
650 2 4 _aMedical Genetics.
650 2 4 _aCell Biology.
650 2 4 _aNanotechnology.
650 2 4 _aClinical Genetics.
650 2 4 _aMolecular Genetics.
700 1 _aScholefield, Janine.
_eeditor.
_4edt
_4http://id.loc.gov/vocabulary/relators/edt
710 2 _aSpringerLink (Online service)
776 0 8 _iPrinted version:
_x0969-7128
856 4 0 _uhttps://doi.org/10.1038/41434.1476-5462
_zHybrid
999 _c1918
_d1918