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Educational Resources
Gene therapy is an experimental technique for treating disease by altering the patient's genetic material.
gene therapygene therapy is an experimental technique for treating disease by …
News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… in understanding the elevated cancer risk associated with gene therapy . they conducted research on mice with a rare disease …
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 …
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.
… of health (nih) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single …
News Release
NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.
… and protect the body from infection. ecd has no standard therapy , although consensus guidelines for clinical … were available for molecular testing, half had braf v600e gene mutations, which is sometimes seen in colon cancer, lung …
News Release
NIH awards more than $64 million to for a database of human cellular responses called the Library of Integrated Network-based Cellular Signatures or LINCS.
… a rational approach to drug discovery and personalizing therapy . oregon health and science university (ohsu), … on cellular phenotypes, protein make-up and gene expression readouts in cell lines. broad institute of …
News Release
A team from the NIH has surmounted a major obstacle to testing potential drug therapies for a rare, genetic condition called Gaucher disease.
… is an inherited condition caused by alterations in the gba gene . it affects one in every 50,000 to 100,000 people, but … called gaucher disease. ​ … gaucher disease, drug therapy for gaucher disease, ellen sidransky ph.d., gba gene
Fact Sheets
Genetics refers to the study of genes and their roles in inheritance. Genomics refers to the study of all of a person's genes (the genome).
… … understanding more about diseases caused by a single gene (using genetics) and complex diseases caused by multiple … of pharmacogenetics is to create an individualized drug therapy that allows for the best choice and dose of drugs. …
Policy Issues
Scientific institutions are developing recommendations to support the responsible use of human gene-editing research.
… genome-editing research. … in light of recent advances in gene -editing research and technologies, the national … of gene editing, germline modifications, somatic cell therapy , societal implications, international perspectives, …
News Release
Researchers have discovered that FGF21 is elevated in mice with liver disease that mimics the same condition in patients with methylmalonic acidemia.
… also allowed them to study the response to liver-directed gene therapy and to compare the findings in patients after liver …