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News Release
NIH scientists proposed a new approach for finding ways to impede or halt cancer's spread: transgenic zebrafish as a live animal model of metastasis.
… Such high-throughput screening has identified thousands of drug candidates over the past 10 years but has resulted in … anti-cancer compounds and identify genetic targets for therapy that current screening methods might miss. They … metastasis. ​ … Zebrafish, cancer and metastasis, targeted therapy against cancer, Viviana Gallardo Ph.D., Shawn Burgess …
Policy Issues
Scientific institutions are developing recommendations to support the responsible use of human gene-editing research.
… of gene editing, germline modifications, somatic cell therapy, societal implications, international perspectives, … the use of genome editing in basic research and somatic therapy trials, there are still safety, technical, and … the wide application of this technology in germline therapy trials. The committee recommended a set of very …
Staff
Sangeetha Raghavan is a Senior Licensing and Patenting Manager in NHGRI's Technology Transfer Office.
… in wide ranging areas of biologics, diagnostics, drug delivery, therapeutics, medical devices, plant … Patented Technology, Licensing, biologics, diagnostics, drug delivery, therapeutics, medical devices, plant …
Policy Issues
Genome editing is currently being applied to research on cancer, mental health, rare diseases, and many other disease areas.
… targets. Although excitement about the potential for gene therapy has grown tremendously since the discovery of CRISPR, … might eventually be able to prescribe targeted gene therapy to make corrections to patient genomes and prevent, … are two different categories of gene therapies: germline  therapy and somatic  therapy. Germline therapies can alter …
Policy Issues
Genome editing is a method that lets scientists change the DNA of many organisms, including plants, bacteria, and animals.
… are two different categories of gene therapies: germline therapy and somatic therapy. Germline therapies change DNA in reproductive cells … these cells affect only the person who receives the gene therapy. In 2015, scientists successfully used  somatic gene …
Policy Issues
Scientists have edited genomes for many years, but CRISPR technology has improved the speed, cost, accuracy, and efficiency of genome editing
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… aid the development of new treatments, including gene therapy . … Scientists have had the knowledge and ability to … Genome Editing, Genomic Technology, Gene Editing, Gene Therapy, DNA Editing … Scientists have edited genomes for …
Policy Issues
Patients with genetic disorders and members of the public have diverse about germline genome editing.
… of 2016, passed in December 2015, prohibits the Food and Drug Administration (FDA) from reviewing research that … … Germline Genome Editing, Gene Editing, DNA Editing, Gene Therapy, Genetic Disorder, Genetic Disease, CRISPR-Cas9, …
For Patients and Families
The Genetic and Rare Diseases Information Center helps people find useful information about genetic and rare diseases.
… and rare diseases ​ … Genetic Disease, Rare Disease, Gene Therapy, Diagnosing A Disease, DNA Sequence, Genome Sequence, …
15 Ways Genomics Influences Our World
Advances in genomics are helping you access information about your genome from your home.
… you are interested in been approved by  the U.S. Food and Drug Administration (FDA) if it is about your health? How … Limitations To DTC Genomic Testing, Regulations, Food And Drug Administration (FDA) … Advances in genomics are helping …
Fact Sheet
Learn about the different types of hemoglobin.
… To understand sickle cell disease and gene therapy , you should first know about blood cells and the … welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to … are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use …