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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 …
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).
… of pharmacogenetics is to create an individualized drug therapy that allows for the best choice and dose of drugs. … is the breast cancer drug trastuzumab (Herceptin). This therapy works only for women whose tumors have a particular … Asked Questions about Pharmacogenomics ). Stem Cell Therapy Stem cells have two important characteristics. First, …
Educational Resources
A ribosome is a cellular particle made of RNA and protein that serves as the site for protein synthesis in the cell.
… cell. … Amino Acids, Cell, Genetic Code, Messenger RNA (mRNA), Protein, Ribonucleic Acid (RNA) … A ribosome is an … in the cell. The ribosome reads the messenger RNA (mRNA) sequence and translates that genetic code into a …
Educational Resources
A promoter is a sequence of DNA needed to turn a gene on or off.
… Gene, Gene Expression, Gene Regulation, Messenger RNA (mRNA), Transcription … A promoter, as related to genomics, is … resulting transcription produces an RNA molecule (such as mRNA). … A promoter is a sequence of DNA needed to turn a …
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 …
Policy Issues
Scientific institutions are developing recommendations to support the responsible use of human gene-editing research.
… of gene editing, germline modifications, somatic cell therapy, societal implications, international perspectives, … the use of genome editing in basic research and somatic therapy trials, there are still safety, technical, and … the wide application of this technology in germline therapy trials. The committee recommended a set of very …
Educational Resources
Transfer RNA (tRNA) is a small RNA molecule that participates in protein synthesis.
… Anticodon, Base Pair, Codon, Genetic Code, Messenger RNA (mRNA), Protein, Ribosome, Translation, Amino Acids … Transfer … serves as a link (or adaptor) between the messenger RNA (mRNA) molecule and the growing chain of amino acids that make … specific tRNA pairs with its complementary sequence on the mRNA molecule, ensuring that the appropriate amino acid is …
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, …
Educational Resources
The nucleolus is a region found within the cell nucleus that is concerned with producing and assembling the cell's ribosomes.
… and assembling the cell's ribosomes. … Messenger RNA (mRNA), Ribonucleic Acid (RNA), Translation, Cell, Cytoplasm, …
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 …