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News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice … by researchers who have designed gene therapies using an adeno-associated virus (AAV) as a vector to deliver the corrected gene to a …
Staff
Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… Kazazian’s lab, where Dr. Chandler’s research focused on adeno-associated virus (AAV) gene therapy for the treatment of hemophilia A, a …
News Release
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
… on the brain. To do this, they used a non-disease-causing virus called the adeno-associated virus serotype 9 (AAV9) to transfer functioning …
Fact Sheet
Understanding gene therapy for sickle cell disease
… Now, researchers are developing new and emerging gene therapies to treat sickle cell disease. To help navigate … for the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with …
News Release
Scientists are using an exciting gene editing tool called CRISPR/Cas9 to protect plants from harmful DNA viruses, strengthening the plant's protections.
… Julie Coursen … Scientists are using an exciting gene editing tool called CRISPR/Cas9 to protect plants from … species. Tobacco plants are susceptible to a destructive virus called tomato yellow leaf curl virus, which leads to stunted growth, abnormal leaves, and …
Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project utilizes a deliberative stakeholder-engagement approach to develop patient educational materials for sickle cell disease gene therapy.
… that patients with sickle cell disease who are considering gene therapy fully understand the meaning and limitations of … welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or …
Educational Resources
A virus is an infectious agent that occupies a place near the boundary between the living and the nonliving.
… Virus … A virus is an infectious agent that occupies a place near the … Acid (DNA), Enzyme, Protein, Ribonucleic Acid (RNA) … A virus is an infectious microbe consisting of a segment of … acid (either DNA or RNA) surrounded by a protein coat. A virus cannot replicate alone; instead, it must infect cells …
Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… Step 4: Preparation for stem cell collection (~90 days)  Gene therapy requires stem cells to be collected from your bone … cell collection. The process differs based on the type of gene therapy approach. The process does not always work. If …
Educational Resources
Gene therapy is an experimental technique for treating disease by altering the patient's genetic material.
… Gene Therapy … Gene therapy is an experimental technique for treating disease by …
News Release
It’s teatime and doctors, researchers and patients are at the table. They celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating disease, GM1 gangliosidosis.
… celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating … in creating the gene therapy vector -- an unwitting virus that would deliver functioning copies of the defective …