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DNA Analysis Finds New Target for Diabetes Drugs

NIH Director's Blog: Drug Development

Credit: Jane Ades, National Human Genome Research Institute, NIH Type 2 diabetes (T2D) tends to run in families, and over the last five years the application of genomic technologies has led to discovery of more than 60 specific DNA variants that contribute to risk. 2010 Jan-Feb;2(1):49-50. [7] J Clin Invest.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. Liviana was born in January 2008 in Omaha, Nebraska, and diagnosed with MLD in December 2010. Giovanni was born in January 2010. That would enable identification of children before symptoms begin.

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Not all neoantigens are created equal

Drug Target Review

During the process of transformation from a normal cell into a cancer cell, a cell acquires a series of changes, or mutations, in its DNA. But DNA mutations can also result in changes to the proteins that are displayed on the surface of the cancer cell.

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New insight into BRCA2 gene mutations and cancer risk

Drug Target Review

Researchers from the University of California, Davis delved deeper into the mechanisms of DNA repair and cancer development by studying the BRCA2 gene at the molecular level. The study’s findings provide new insights into the origins of cancer and DNA repair mechanisms.

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Afimetoran

New Drug Approvals

11:373-384 (2010)). 10:89-102 (2010)). TLR7-9 are among the set that are endosomally located and respond to single-stranded RNA (TLR7and TLR8) or unmethylated single-stranded DNA containing cytosine-phosphate-guanine (CpG) motifs (TLR9). Nature Immunol., With the exception of TLR3, all TLRs recruit the adaptor molecule MyD88.

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The Dangers of Mirrored Life

Codon

DNA and RNA molecules are also built from exclusively right-handed nucleic acids. Across the tree of life, organisms strictly require exactly one of the two chiral forms of their molecular building blocks — amino acids, nucleotides of RNA and DNA. 4 As far as we know, right-handed proteins never occur naturally.

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Experimental Myotonic Dystrophy Treatment Teams Monoclonal Antibody and siRNA

PLOS: DNA Science

In these 50 or so conditions, symptoms may appear earlier and worsen from generation to generation, as the mutant gene grows, adding copies of a 3- or 4-base DNA sequence. The duo is designed to correct the mutation, the MAb targeting the siRNA to the gene behind MD1, where it snips out DNA repeats.