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Translational Science Reviews—A New JAMA Review

Cette étude passe en revue les données portant sur l'efficacité des immunothérapies utilisant les lymphocytes CAR-T autorisés par la FDA pour traiter 6 types de tumeurs hématologiques

Recent discoveries in basic research have major implications for improving diagnosis and treatment of human diseases. For example, scientists recently identified the molecular cause of a T-cell phenotype imbalance associated with the development of lupus and demonstrated that the interferon-blocking drug anifrolumab can reverse this T-cell abnormality, with potential implications for curing lupus. In a separate study, an antisense oligonucleotide, a therapy affecting RNA processing and protein expression, repaired human brain neurons damaged in Timothy syndrome, a rare genetic disorder. Results have potential implications for treating other incurable brain diseases such as schizophrenia. To help clinicians keep up with ongoing basic and translational science discoveries that affect the diagnosis and treatment of human disease, JAMA has launched a new series, “Translational Science Reviews.” These new article types are succinct and informative summaries of important basic science advances that are transforming diagnosis and treatment of human disease.

JAMA 2023

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