Search Results
Facet (Page)
Refine Your Search
Audiences
Content Formats
Sections
Staff
Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… research focused on adeno-associated virus (AAV) gene therapy for the treatment of hemophilia A, a genetically … Chandler’s research has focused on the efficacy of gene therapy as a treatment for rare genetic disorders, such as … have helped enable two clinical trials: one uses mRNA therapy and the other, AAV-mediated nuclease-free genome …
Staff
Dr. Julie Segre is chief and a senior investigator in the Translational and Functional Genomics Branch at the National Human Genome Research Institute.
​
… tools to track hospital-acquired infections of multi-drug resistant organisms, including the NIH's recent … reconstructed a polymicrobial outbreak of multi-drug resistant Acinetobacter baumannii that swept through the … overall fitness. MGS's long-term goal is to create a drug strategy that capitalizes on the bacterium's loss of …
Profile
After major advances in hematology, David Bodine retires with an eye towards mentorship and a garden
David Bodine, founder of the Hematopoiesis Section and chief of the Genetics and Molecular Biology Branch, advanced the fields of gene therapy and hematology in his 38 years at the NIH. Now, he plans to continue his contributions to science as an emeritus and through training the next generation of scientists.
… you see as the most exciting future opportunities in gene therapy?  Bodine: When we founded the American Society of Gene Therapy, we wanted to make these therapies low cost and widely available. Now, 20 years later, gene therapy has been very successful for treating any number of …
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).
… account a person's genetic information regarding specific drug receptors and how drugs are transported and metabolized … goal of pharmacogenetics is to create an individualized drug therapy that allows for the best choice and dose of drugs. …
Staff
Dr. Rongling Li is an epidemiologist and program director in the Division of Genomic Medicine at the National Human Genome Research Institute.
​
… medical records, gene-environment interactions, gene-drug interactions … Dr. Rongling Li is an epidemiologist and …
News Release
Researchers have discovered that FGF21 is elevated in mice with liver disease that mimics the same condition in patients with methylmalonic acidemia.
… allowed them to study the response to liver-directed gene therapy and to compare the findings in patients after liver … "Our new markers can accurately predict how effective a therapy, whether cellular or genomic, might be for the … acidemia, medical genomics, genetic disorders, gene therapy, Charles Venditti … Researchers have discovered that …
News Release
Receiving a cancer diagnosis can feel like losing control of everything.
… control of everything. … cancer, cancer diagnosis, cancer therapy, genome sequencing, Precision medicine, epigenome, …
Policy Issues
Most ethical discussions about genome editing center on human germline editing because changes are passed down to future generations.
… at this time, but that studies that would make gene therapy  safe and effective should continue. 1 , 2  Most … it is impossible to obtain informed consent for germline therapy because the patients affected by the edits are the … from prospective parents as long as the risks of germline therapy are unknown. 10 Justice and Equity As with many new …
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 …
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 …