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Carlos Ferreira, M.D., staff clinician in NHGRI's Metabolic Medicine Branch, knows the catalogue of rare skeletal disorders well. With hundreds of known rare bone disorders and more to be discovered, Dr. Ferreira breaks down the challenges of skeletal genomics.
… and approaches currently being developed, such as gene therapy and the use of small molecules. By improving our …
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Comente estas preguntas con su médico o su equipo del estudio clÃnico.
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… . . . . . . . . . . . . . . . . . . . . . . . 61 3.9 Gene therapy . . . . . . . . . . . . . . . . . . . . . . . . . . . … plementary) molecule called messenger ribonucleic acid (mRNA), which moves from the nucleus to the cytoplasm of a … the appropri- ate order, as directed by the codons of the mRNA (Figure 2.2). Some newly synthesized proteins undergo a …
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 …
Clinical Research
The goal of this study is to learn more about the genetic contributions to the severity of disease of COVID-19.
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… Navigating_sickle_cell_disease_gene_therapy.pdf …
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 …