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News Release
Researchers are asking if the precision medicine approach will reduce or eliminate the role that race plays in prescribing drugs and in health care overall.
… overall. ​ … Precision medicine, drug prescribing, drug therapy, Vence Bonham, longitudinal drug studies, drug …
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 …
Educational Resources
Ribonucleic acid (RNA) is a molecule similar to DNA. Unlike DNA, RNA is single-stranded.
… Uracil, Adenine, Cytosine, Guanine, Messenger RNA (mRNA), Ribosome … Ribonucleic acid (abbreviated RNA) is a … (G). Different types of RNA exist in cells: messenger RNA (mRNA), ribosomal RNA (rRNA) and transfer RNA (tRNA). In …
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.
… 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 …
Educational Resources
Transcription is the process of making an RNA copy of a gene sequence.
… Acid (DNA), Gene, Gene Expression, Messenger RNA (mRNA), Nuclear Membrane, Nucleus, Ribosome, Translation, … of a gene’s DNA sequence. This copy, called messenger RNA (mRNA), carries the gene’s protein information encoded in DNA. In humans and other complex organisms, mRNA moves from the cell nucleus to the cell cytoplasm …
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 …
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, …
Policy Issues
Patients with genetic disorders and members of the public have diverse about germline genome editing.
… … Germline Genome Editing, Gene Editing, DNA Editing, Gene Therapy, Genetic Disorder, Genetic Disease, CRISPR-Cas9, …
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 …