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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.
… 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
Your mental and physical health are deeply connected. Mental health is important for your health and quality of life. 
…  There are many unknowns about what to expect from a gene therapy clinical trial. Your thoughts, feelings, beliefs and … for your mental and physical health as you consider a gene therapy clinical trial.  Focus on your thoughts and … of participation Check out the sickle cell disease gene therapy FAQ Cost What costs does the clinical trial cover? …
Fact Sheets
Genomics is the study of all of a person's genes (the genome), including interactions of those genes with each other and with the person's environment.
… DNA into a molecule called messenger ribonucleic acid (mRNA). The mRNA travels out of the nucleus and into the cell's … though recent genome-driven efforts in lipid-lowering therapy have considerably shortened that interval. According …
Staff
Dr. David Bodine is the chief of and senior investigator in NHGRI's Genetics and Molecular Biology Branch.
… Hematology. The American Society of Gene and Cell Therapy has elected Dr. Bodine to serve as President, … Gallagher PG, Bodine DM. Long term expression of g-globin mRNA in mouse erythrocytes from retrovirus vectors containing … Jordan CT, Anderson SM, Cline AP, Bodine DM. The level of mRNA encoding amphotropic retrovirus receptor in mouse and …
Event
A joint NHGRI-NCATS hosted meeting bringing together researchers, clinicians, families and patient advocates from around the world to discuss the natural history, disease pathophysiology, genetics and treatments for rare inborn metabolic errors.
… A. Chapman, M.D., Ph.D. 3:10 - 3:30 p.m. Systemic AAV gene therapy for propionic acidemia (PA) Randy Chandler, Ph.D. … David S. Rosenblatt, M.D. 10:30 - 10:45 a.m. High-Dose B12 Therapy for cblC Deficiency Irini Manoli, M.D., Ph.D. 10:45 - … p.m. Treatment of CBS Deficient Mice by AAV-Mediated Gene Therapy Warren Kruger, Ph.D. 2:30 - 3:00 p.m. Break  Session …