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Educational Resources
Genetic engineering is the process of using recombinant DNA (rDNA) technology to alter the genetic makeup of an organism.
Educational Resources
Antisense is the non-coding DNA strand of a gene.
… DNA serves as the template for producing messenger RNA (mRNA), which directs the synthesis of a protein. … Antisense …
File
… Sickle_cell_disease_gene_therapy_FAQ.pdf …
Event
On February 16, 2023, the National Human Genome Research Institute (NHGRI) held a follow-up working group consultation after the 14th Genomic Medicine meeting, Genomic Medicine XIV: Genomic Learning Healthcare Systems.
… who has a special clinic setup for difficult cases of drug therapy adverse events and consults for PGx. In our Oncology …
Educational Resources
An exon is the portion of a gene that codes for amino acids.
… … An exon is a region of the genome that ends up within an mRNA molecule. Some exons are coding, in that they contain …
Staff
Dr. Peter McGuire is an investigator in NHGRI's Center for Precision Health Research.
… perturbations are demonstrated using metabolomics, mRNA profiling, proteomics, enzymology and in vivo metabolic …
Event
On November 1-5, 2023, the National Human Genome Research Institute (NHGRI) will participate in the ASHG Annual Meeting - the world's largest human genetics and genomics meeting and exposition.  The annual meeting provides a forum for the presentation and discussion of cutting-edge science in all areas of human genetics. 
… Sickle Cell Disease PB3447 Vence Bonham Democratizing Gene Therapy Education for Patient Communities. PB2369 David Adams … Syndrome: Exploring the therapeutic effect of gene therapy for HPS-1 pulmonary fibrosis PB2027 Zo Bly The …
Staff
Dr. Charles Venditti is a chief and senior investigator of NHGRI's Metabolic Medicine Branch.
… mitochondrial dysfunction and prove the efficacy of gene therapy as a treatment for both methylmalonic acidemia and … Investigator from the American Society of Gene and Cell Therapy in 2010 and election into the American Society of … fields of inborn errors of metabolism, genetics and gene therapy and serves on the medical advisory board of the …
Educational Resources
An intron is a portion of a gene that does not code for amino acids.
… within a gene but does not remain in the final mature mRNA molecule following transcription of that gene and does …
News Release
Investigators with The Cancer Genome Atlas Research Network have identified new potential therapeutic targets for a major form of bladder cancer.
… therapies - second choices for treatments when the initial therapy does not work - and no approved targeted agents for … molecular targets identified may help us to personalize therapy based on the characteristics of each patient's … been treated with chemotherapy, radiation or any type of therapy. The scientists found recurrent mutations in 32 …