Targeting a blood stem cell subset shows lasting gene editing
Scientists report how editing a portion of stem cells with CRISPR-Cas9 is sufficient for long-term reactivation of therapeutic haemoglobin.
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Scientists report how editing a portion of stem cells with CRISPR-Cas9 is sufficient for long-term reactivation of therapeutic haemoglobin.
Researchers have newly found that protein CD24 acts as a defensive signal and is used by cancer cells to protect themselves.
A study has discovered that reducing expression of a key gene in neuroblastoma tumourigenesis is an important drug target for the condition.
New study sheds light on how the TP53 gene becomes mutated and how those mutations can help predict clinical outlooks for cancer.
Researchers have discovered a new combination therapy to treat drug-resistant acute lymphoblastic leukaemia, tested in pre-clinical trials.
Researchers have discovered a non-coding RNA which could be used as a biomarker and in new therapies for prostate cancer.
New discovery makes it possible to design new therapies to replace alpha-synuclein's function in people with Parkinson's disease.
Salk scientists discover a pair of enzymes that drive non-small-cell lung cancer by promoting inflammation which could inform the development of new therapies.
Newly developed HDO-antimiR could lead to new ways to treat diseases which are caused by malfunctioning miRNAs.
Researchers have successfully used a no-cut CRISPR gene editing technique as a therapy for muscular dystrophy in mice models.
Researchers used screening to discover genes that protect the body against the Zika virus, which could inform potential therapies against the virus.
Researchers have identified a key process behind senescence, or why cells age, which could be used to improve treatments for cancer.
Researchers have extended the life of heart segments from 24 hours to six days, enabling pre-clinical trials to experiment on these new models for longer periods of time.
A novel antibody-enzyme fusion therapeutic strategy for Lafora disease has shown effectiveness in pre-clinical study mice models.
Scientists have created the first completely artificial protein switch that can be ‘programmed’ to modify gene expression.