Researchers used antisense oligonucleotides to block defective genes responsible for producing hemoglobin, restoring correct production of beta-globin and enabling cells to produce more hemoglobin. The correction could last for months and may be a simpler therapy than gene therapy.
The article describes how novel human genes can be found using data mining techniques on the Internet. Researchers identified putative human genes related to peptide transporter proteins, which are essential for drug absorption and distribution.
Researchers at UNC-CH have successfully repaired a genetic problem causing cystic fibrosis, offering new hope for patients. The technique involves correcting a mutation involving unnecessary information inside a gene, resulting in the production of normal messenger RNA.
Victor Velculescu, a cancer researcher, develops SAGE technique to rapidly analyze gene expression in cells. The method provides a snapshot of all active genes and has potential applications in disease research.
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Researchers at Chromos Molecular Systems have successfully passed an artificial chromosome to their offspring, paving the way for practical germline gene therapy. The technology could enable genetic changes to be inherited without disrupting other genes, offering a safer alternative to current methods.
Researchers have discovered a novel function of the Survival Motor Neuron (SMN) protein essential for mRNA production in all cells. This finding links SMN deficiency to spinal muscular atrophy, a leading genetic cause of infant death, and paves the way for potential therapeutic interventions.
Researchers at Brandeis University have induced a form of chronic myelogenous leukemia (CML) in mice, allowing them to study its molecular mechanisms and potential treatments. The breakthrough could lead to the development of new therapies for CML, which affects one-fifth of all leukemia patients.
Myuanan Long, a University of Chicago researcher, has received the David and Lucile Packard Fellowship to study gene evolution. He believes that new genes arise through shuffling and mixing of existing genes or gene fragments, with 'junk' DNA playing a crucial role in this process.
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Researchers discovered a genetic defect in ALL1 gene duplication that signals poor prognosis and shorter survival time in AML patients. Patients with this defect require aggressive treatment, including allogenic bone marrow transplant, to improve survival rates.
Researchers at Johns Hopkins Medicine found that the TSG101 gene, previously identified as a tumor suppressor, was consistently normal and undamaged in human breast cancer cells. The cells exhibited abnormal RNA splicing, which may be a result of cancer cells trying to activate normal cell behaviors in an abnormal way.