CRISPR genome editing corrects a Duchenne muscular dystrophy deletion mutation in mice
Appeared in BioNews 1093
Two genome editing approaches can correct a variety of mutations associated with Duchenne muscular dystrophy (DMD), researchers have shown.
DMD is a genetic disorder caused by mutations in the
dystrophingene, leading to a fatal decline in muscle integrity. For the first time, researchers have used two CRISPR-based genome editing techniques to correct multiple dystrophin mutations in mice. It is hoped this could pave the way for new genome editing therapies for DMD. Every cell in the human body has three billion letters of DNA sequence in its genome, and this method makes it possible to correct large deletions in the DMD gene by specifically swapping one of these letters, said Professor Eric Olson, of the University of Texas Southwestern Medical Centre in Dallas, and senior author of the study. That level of specificity and efficiency is remarkable, he added.
Study provides the most comprehensive look yet on COVID-19 hospital mortality
A new study of hospital deaths in the United States, published today in
JAMA Network Open, is the most comprehensive look yet at changes in hospital mortality during the pandemic. Researchers analyzed data on more than 20,000 patients admitted to hospitals for COVID-19, over nine full months, from March to November of 2020. They found that rates of in-hospital mortality among COVID patients fell 38% between March and May, but there was little further decline through November 2020.
The researchers aimed to uncover the most likely cause of the observed trend by controlling for patient age, sex, comorbidities, and severity of disease when the patient was admitted. However, they found that none of these factors fully explained the decline in mortality rates.
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The advent of next-generation proteasome inhibitors, immunomodulatory agents, and monoclonal antibodies over the last decade has revolutionized the way patients with multiple myeloma are managed.
However, the disease remains challenging, as relapse and disease progression remain common, even after complete remission. Myeloma often mutates and becomes more resistant to subsequent lines of therapy, leading to shorter responses and remissions.
Why CAR T-Cell Therapy?
Chimeric antigen receptor (CAR) T-cell therapy is one of the latest available therapeutic strategies in relapsed or refractory multiple myeloma (RRMM). Why is this a promising approach? The bigger question is why CAR T-cell therapy is a good strategy for blood cancer to begin with, said Saad Usmani, MD, of the Levine Cancer Institute in Charlotte, North Carolina.
Researchers analyzed data on more than 20,000 patients admitted to hospitals for COVID-19. After controlling for patient age, sex, comorbidities, and severity of disease when the patient was admitted, they found that none of these factors fully explained the decline in mortality rates.
January 2006
1. What is the STAR D study?
A. The Sequenced Treatment Alternatives to Relieve Depression (STAR D) Study, funded by the NIH National Institute of Mental Health, is a nationwide public health clinical trial. The purpose of the trial is to determine the effectiveness of different treatments for people with Major Depressive Disorder (MDD) who have not responded to initial treatment with an antidepressant. This is the largest and longest study ever done to evaluate depression treatment. Over a seven-year period, the study enrolled more than 4,000 outpatients, aged 18-75 years. The participants were people who came to their doctors for care and who had other psychiatric and medical conditions like those regularly seen in typical clinical practices (see Ref 1). Patients from both mental health (specialty care) and non-mental health (primary medical care) practices in diverse racial, ethnic, and socioeconomic populations were enrolled in the study.